113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details


臨床試験数 : 622 薬物数 : 494 - (DrugBank : 99) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 168

  
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PhaseCountries
1NCT05126758
(ClinicalTrials.gov)
March 15, 20224/11/2021A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular DystrophyMuscular Dystrophies;Muscular Dystrophy, Duchenne;Muscular Disorders, Atrophic;Muscular Diseases;Neuromuscular Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn;Nervous System DiseasesBiological: CAP-1002;Drug: PlaceboCapricor Inc.NULLNot yet recruiting10 YearsN/AMale68Phase 3United States
2NCT05185622
(ClinicalTrials.gov)
March 15, 20229/11/2021A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: VamoroloneReveraGen BioPharma, Inc.Santhera PharmaceuticalsNot yet recruiting2 Years17 YearsMale44Phase 2NULL
3EUCTR2018-001762-42-NO
(EUCTR)
04/03/202202/12/2021A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
154Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Poland;Romania;Germany;Norway;New Zealand;Sweden
4EUCTR2019-003374-91-ES
(EUCTR)
21/02/202220/10/2021Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular DystrophyA Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: SRP-9001
INN or Proposed INN: Not available
Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 3France;United States;Taiwan;Hong Kong;Belgium;Spain;Germany;United Kingdom;Italy;Japan
5EUCTR2021-004276-33-DK
(EUCTR)
11/02/202201/10/2021GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophyA phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I) FKRP-related limb-girdle muscular dystrophy (LGMD R9)
MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: rAAV9-hFKRPco_miR-208a
Product Code: GNT0006
INN or Proposed INN: not yet available
Other descriptive name: Adeno-associated virus serotype 9 carrying the human fukutin-related protein and target sequence of the miR-208a
Atamyo TherapeuticsNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
39Phase 1;Phase 2France;United States;Canada;Denmark;Norway;Germany;United Kingdom;Sweden
6EUCTR2018-001762-42-GR
(EUCTR)
01/02/202201/12/2021A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
154Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden
7NCT04336826
(ClinicalTrials.gov)
December 29, 20213/4/2020A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular DystrophyNonsene Mutation Duchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLRecruiting6 Months2 YearsMale10Phase 2United States
8NCT05195775
(ClinicalTrials.gov)
December 14, 20214/1/2022Tadalafil as Adjuvant Therapy for DMDTadalafil as an Adjuvant to Therapy for Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: TadalafilUniversity of FloridaNULLRecruiting7 Years13 YearsMale25Phase 2/Phase 3United States
9NCT05160415
(ClinicalTrials.gov)
December 20212/12/2021A Study of EDG-5506 in Adult Males With Becker Muscular DystrophyA Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular DystrophyBecker Muscular DystrophyDrug: EDG-5506Edgewise Therapeutics, Inc.Medpace, Inc.Recruiting18 Years55 YearsMale8Phase 1United States
10EUCTR2021-000122-10-GR
(EUCTR)
25/11/202113/10/2021A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of
11NCT05027269
(ClinicalTrials.gov)
October 28, 202124/8/2021Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) PatientsA Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) PatientsDM1;Myotonic Dystrophy 1;Myotonic Dystrophy;Myotonic Dystrophy Type 1 (DM1);Dystrophy Myotonic;Myotonic Disorders;Steinert Disease;Myotonic Muscular DystrophyDrug: AOC 1001;Drug: PlaceboAvidity Biosciences, Inc.NULLRecruiting18 Years65 YearsAll44Phase 1/Phase 2United States
12NCT04906460
(ClinicalTrials.gov)
September 28, 202124/5/2021Open-label Study of WVE-N531 in Patients With Duchenne Muscular DystrophyAn Open-label Phase 1b/2a Study of WVE-N531 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: WVE-N531Wave Life Sciences Ltd.NULLRecruiting5 Years12 YearsMale15Phase 1/Phase 2Canada;United Kingdom
13EUCTR2019-002076-13-NO
(EUCTR)
08/09/202118/02/2021This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Netherlands;Norway;China;Japan;New Zealand;Sweden;Korea, Republic of
14JPRN-jRCT2031210252
06/09/202118/08/2021A phase 1 study in patients with Fukuyama-type congenital muscular dystrophyA multicenter phase 1 study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy Fukuyama-type congenital muscular dystrophyThis study consists of the following 4 cohorts. The study will start with cohort 1 and only D-mannitol will be administered once during the premedication phase, followed by 12 simultaneous doses of NS-035 and D-mannitol once weekly during the treatment phase. The dose of D-mannitol was fixed at 500 mg / kg in all cohorts, and NS-035 was gradually increased from cohort 1 to cohort 4 (1.6 mg/kg, 6.0 mg/kg, 20 mg/kg and 40 mg/kg, respectively).Toda TatsushiNULLRecruiting>= 5age old<= 10age oldBoth12Phase 1Japan
15NCT04624750
(ClinicalTrials.gov)
September 3, 202120/10/2020Open Label Study in Adolescents and Children With Myotonic DisordersAn Open-label, Non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children With Myotonic DisordersMyotonic DystrophyDrug: MexiletineLupin Ltd.NULLRecruiting6 Years18 YearsAll14Phase 3France
16NCT05004129
(ClinicalTrials.gov)
August 23, 20215/8/2021Safety and Efficacy of Tideglusib in Congenital Myotonic DystrophyA 52-Week, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 (REACH CDM X)Congenital Myotonic DystrophyDrug: TideglusibAMO Pharma LimitedNULLEnrolling by invitation6 Years17 YearsAll56Phase 2/Phase 3United States;Australia;Canada;New Zealand
17EUCTR2019-000601-77-DE
(EUCTR)
17/08/202131/03/2020A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
60Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy
18EUCTR2020-003653-30-ES
(EUCTR)
26/07/202115/12/2021A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
20Phase 2United States;Spain;Turkey;Russian Federation;Italy;China
19NCT04700046
(ClinicalTrials.gov)
July 6, 20215/1/2021Study to Investigate the Efficacy and Safety of Mexiletine in Patients With Myotonic Dystrophy Type 1 and Type 2A Randomized, Double-blind, Placebo-controlled, Multi-center Study to Investigate the Efficacy and Safety of Mexiletine During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 [The MIND Study]Myotonic Dystrophy Type 1 and Type 2Drug: Mexiletine 167 mg;Drug: PlaceboLupin Ltd.NULLNot yet recruiting18 YearsN/AAll158Phase 3NULL
20NCT04956289
(ClinicalTrials.gov)
July 1, 202130/6/2021Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMDA Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History ControlsDuchenne Muscular DystrophyDrug: ViltolarsenNS Pharma, Inc.Nippon Shinyaku Co., Ltd.Recruiting8 YearsN/AMale20Phase 2United States;Italy;Russian Federation;Spain;Turkey
21EUCTR2021-000122-10-NL
(EUCTR)
30/06/202108/04/2021A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of
22NCT04886518
(ClinicalTrials.gov)
June 28, 202110/5/2021Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label ExtensionMyotonic Dystrophy 1;Excessive Daytime SleepinessDrug: Pitolisant Oral Tablet;Drug: Placebo oral tabletHarmony Biosciences, LLCNULLRecruiting18 Years65 YearsAll135Phase 2United States
23EUCTR2020-000699-39-NL
(EUCTR)
28/06/202109/02/2021Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected by Duchenne Muscular Dystrophy and just Treated with CorticosteroidsA Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) Ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: FG-3019
INN or Proposed INN: PAMREVLUMAB
FibroGen, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
70Phase 3United States;France;Canada;Spain;Belgium;Austria;Australia;Netherlands;United Kingdom;Italy
24NCT05135663
(ClinicalTrials.gov)
June 23, 20215/10/2021Extension Study of NS-089/NCNP-02 in DMDA Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: NS-089/NCNP-02Nippon Shinyaku Co., Ltd.NULLActive, not recruitingN/AN/AMale6Phase 2Japan
25EUCTR2019-002921-31-BE
(EUCTR)
18/06/202107/09/2020Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY DUCHENNE MUSCULAR DYSTROPHY (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PF-06939926
Product Code: PF-06939926
INN or Proposed INN: PF-06939926
Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
Pfizer Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of
26EUCTR2021-000122-10-ES
(EUCTR)
15/06/202130/07/2021A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of
27NCT04687020
(ClinicalTrials.gov)
June 10, 202116/12/2020Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)Duchenne Muscular DystrophyDrug: ViltolarsenNS Pharma, Inc.NULLEnrolling by invitationN/AN/AMale16Phase 4United States;Canada
28EUCTR2020-003653-30-IT
(EUCTR)
08/06/202108/06/2021This is an Open-label Study to evaluate the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: [NS-065/NCNP-01]
INN or Proposed INN: VILTOLARSEN
NS Pharma, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
20Phase 2United States;Spain;Turkey;Russian Federation;China;Italy
29EUCTR2020-004901-29-PL
(EUCTR)
01/06/202120/01/2021Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD).The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-16 years. A randomized, double-blind, placebo controlledstudy. - MeDMD Duchenne muscular dystrophyCardiomyopathyTachycardia
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Betaloc ZOK 25, 23,75 mg
INN or Proposed INN: Metoprolol
Other descriptive name: METOPROLOL SUCCINATE
Trade Name: Betaloc ZOK 100, 95 mg
INN or Proposed INN: Metoprolol
Other descriptive name: METOPROLOL SUCCINATE
Medical University of GdanskNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
144Phase 3Poland
30EUCTR2020-000699-39-AT
(EUCTR)
26/04/202129/01/2021Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected Duchenne Muscular Dystrophy and just Treated with CorticosteroidsA Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) Ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: FG-3019
INN or Proposed INN: PAMREVLUMAB
FibroGen, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
70Phase 3United States;France;Canada;Spain;Belgium;Australia;Austria;Netherlands;United Kingdom;Italy
31EUCTR2021-000122-10-IT
(EUCTR)
26/04/202108/06/2021A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: [NS-065/NCNP-01]
INN or Proposed INN: VILTOLARSEN
NS Pharma, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of
32EUCTR2020-000699-39-IT
(EUCTR)
19/04/202104/06/2021Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected by Duchenne Muscular Dystrophy and just Treated with CorticosteroidsA Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) - na Ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: [FG-3019]
INN or Proposed INN: PAMREVLUMAB
FIBROGENNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
70Phase 3France;United States;Czechia;Canada;Spain;Belgium;Austria;Australia;Netherlands;United Kingdom;Switzerland;Italy
33NCT04768062
(ClinicalTrials.gov)
April 13, 202119/2/2021Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: ViltolarsenNS Pharma, Inc.Nippon Shinyaku Co., Ltd.Enrolling by invitationN/AN/AMale74Phase 3Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Russian Federation;Spain;Turkey
34NCT04821063
(ClinicalTrials.gov)
April 13, 202126/3/2021Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC IntervalA Randomized, Partially Double-Blind, Four-Period, Four-Treatment, Crossover Study Investigating the Placebo-Corrected Effects of a Therapeutic Dose (100 mg) and a Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval in Healthy Male and Female SubjectsDuchenne and Becker Muscular Dystrophy;Polycytemia VeraDrug: ITF2357 10 mg/mL;Drug: Placebo;Drug: Moxifloxacin HydrochlorideItalfarmacoNULLCompleted18 Years55 YearsAll31Phase 1Canada
35EUCTR2019-002921-31-FR
(EUCTR)
15/03/202107/09/2020Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY DUCHENNE MUSCULAR DYSTROPHY (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PF-06939926
Product Code: PF-06939926
INN or Proposed INN: PF-06939926
Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
Pfizer Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of
36NCT03692312
(ClinicalTrials.gov)
March 3, 202116/3/2018Efficacy and Safety of Tideglusib in Congenital Myotonic DystrophyA Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)Congenital Myotonic DystrophyDrug: Tideglusib;Drug: PlaceboAMO Pharma LimitedNULLRecruiting6 Years16 YearsAll56Phase 2/Phase 3United States;Australia;Canada;New Zealand;United Kingdom
37NCT04184882
(ClinicalTrials.gov)
February 24, 20212/12/2019A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)A Randomized, Double Blind, Placebo-Controlled Phase 1b Study With Open-Label Extension to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Patients With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular Dystrophy (DMD)Drug: Bocidelpar;Drug: PlaceboAstellas Pharma IncNULLRecruiting8 Years16 YearsMale18Phase 1United States
38NCT04800874
(ClinicalTrials.gov)
February 18, 202125/1/2021Open Label Phase 2 Study of BBP-418 in Patients With LGMD2IAn Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003)LGMD2IDrug: BBP-418ML Bio Solutions, Inc.NULLEnrolling by invitation12 Years55 YearsAll16Phase 2United States
39EUCTR2020-000698-26-AT
(EUCTR)
02/02/202117/08/2020A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) Non-ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: FG-3019
INN or Proposed INN: PAMREVLUMAB
FibroGen, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
96Phase 3United States;Czechia;Spain;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands
40EUCTR2020-000698-26-NL
(EUCTR)
20/01/202108/09/2020Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with CorticosteroidsA Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) Non-ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: FG-3019
INN or Proposed INN: PAMREVLUMAB
FibroGen, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
90Phase 3United States;Czechia;Spain;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands
41JPRN-jRCT2041200087
07/01/202125/12/2020An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular DystrophyAn Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD Nonsense Mutation Duchenne Muscular DystrophySubjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening doses of
10 mg/kg, 10 mg/kg, and 20 mg/kg.
Penematsa VinayNULLRecruiting>= 5age oldNot applicableMale25Phase 3China;Japan
42EUCTR2020-000698-26-IT
(EUCTR)
28/12/202006/11/2020Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with CorticosteroidsA Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) - na Non-ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: [FG-3019]
INN or Proposed INN: PAMREVLUMAB
FIBROGENNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
90Phase 3United States;Czechia;Slovakia;Spain;Turkey;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands
43NCT04616807
(ClinicalTrials.gov)
December 17, 202023/10/2020An Observational Study in Adult Patients With Non-dystrophic Myotonic DisordersAn Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic DisordersMyotonic DystrophyDrug: MexiletineLupin Ltd.NULLRecruiting18 YearsN/AAll50France;Germany;United Kingdom
44EUCTR2020-000698-26-GB
(EUCTR)
16/12/202014/10/2020Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with CorticosteroidsA Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) Non-ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Pamrevlumab
Product Code: FG-3019
INN or Proposed INN: PAMREVLUMAB
FibroGen, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
90Phase 3United States;Slovakia;Spain;Turkey;Austria;Israel;United Kingdom;Italy;Switzerland;France;Czech Republic;Canada;Belgium;Australia;Netherlands
45NCT04632940
(ClinicalTrials.gov)
December 11, 202012/11/2020A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Pamrevlumab;Drug: PlaceboFibroGenNULLRecruiting6 Years11 YearsMale70Phase 3United States;Australia;Austria;Belgium;Canada;China;France;Italy;Netherlands;Spain;United Kingdom;Switzerland
46EUCTR2015-002069-52-IE
(EUCTR)
11/12/202021/05/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
47EUCTR2019-002921-31-IT
(EUCTR)
04/12/202006/11/2020Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY - - DUCHENNE MUSCULAR DYSTROPHY (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PF-06939926
Product Code: [PF-06939926]
INN or Proposed INN: PF-06939926
PFIZER INCNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of
48EUCTR2017-004625-32-BG
(EUCTR)
03/12/202028/10/2020A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
150Phase 3United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden
49EUCTR2020-002093-27-FR
(EUCTR)
30/11/202024/06/2020A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1).After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2).In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3).Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular DystrophyA phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys. - Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: rAAV8-hMD1
Product Code: GNT0004
INN or Proposed INN: To be determined
Other descriptive name: Adeno-associated viral vector serotype 8 containing the human MD1 gene
GenethonNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
51Phase 1;Phase 2;Phase 3United States;France;Israel;United Kingdom
50EUCTR2019-002076-13-GR
(EUCTR)
12/11/202024/09/2020This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of
51NCT04634682
(ClinicalTrials.gov)
November 9, 202025/9/2020Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1Assessing the Effect of the MYODM Food Supplement on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1Myotonic Dystrophy 1Dietary Supplement: MYODMMyogem Health Company, S.L.NULLRecruiting18 YearsN/AAll30N/ASpain
52NCT04587908
(ClinicalTrials.gov)
November 1, 202013/10/2020A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy(REACH-DMD)A Phase 3, Randomized, Placebo-controlled, Double-blind and Open-label, Extension Study of TAS-205 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: TAS-205;Drug: PlaceboTaiho Pharmaceutical Co., Ltd.NULLRecruiting5 YearsN/AMale80Phase 3Japan
53NCT04708314
(ClinicalTrials.gov)
October 31, 202021/9/2020An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular DystrophyAn Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53]Rare Disease Research, LLCSarepta Therapeutics, Inc.Terminated7 YearsN/AMale2Phase 4United States
54EUCTR2018-001762-42-DE
(EUCTR)
29/10/202001/10/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
154Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Poland;Belgium;Romania;Norway;Germany;New Zealand;Sweden
55NCT03340675
(ClinicalTrials.gov)
October 19, 20203/11/2017Oral Ifetroban in Subjects With Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study With an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy Cardiomyopathy;Cardiomyopathy, DilatedDrug: Ifetroban;Drug: PlaceboCumberland PharmaceuticalsVanderbilt University Medical CenterRecruiting7 YearsN/AMale48Phase 2United States
56NCT04585464
(ClinicalTrials.gov)
October 12, 20202/10/2020A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy AdultsA Phase 1, Randomized, Placebo-Controlled, Double-blind, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and PK of EDG-5506 in Adult Healthy Volunteers and Adults With Becker Muscular DystrophyHealthy Volunteer;Becker Muscular DystrophyDrug: EDG-5506;Drug: PlaceboEdgewise Therapeutics, Inc.Worldwide Clinical TrialsCompleted18 Years55 YearsAll128Phase 1United States
57NCT05154851
(ClinicalTrials.gov)
October 1, 20209/12/2021Individual Patient Expanded Access IND for the Treatment of Spinal Cord Injury, and Congenital Muscular Dystrophy.Individual Patient Expanded Access IND for the Treatment of Spinal Cord Injury, and Congenital Muscular Dystrophy.Spinal Cord Injuries;Congenital Muscular Dystrophy Due to Lamin A/C MutationBiological: HB-adMSCsHope Biosciences Stem Cell Research FoundationNULLNo longer availableN/AN/AMaleUnited States
58EUCTR2019-002921-31-GB
(EUCTR)
29/09/202002/07/2020Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy.A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY DUCHENNE MUSCULAR DYSTROPHY (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PF-06939926
Product Code: PF-06939926
INN or Proposed INN: PF-06939926
Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene
Pfizer Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of
59NCT04322357
(ClinicalTrials.gov)
July 30, 202024/3/2020Weekend Steroids and Exercise as Therapy for DMDImpact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle FunctionDuchenne Muscular Dystrophy (DMD)Drug: Prednisone;Behavioral: In-home Exercise Training;Drug: Prednisone with daily edasalonexent;Drug: Prednisone plus exerciseUniversity of FloridaU.S. Army Medical Research and Development Command;Catabasis PharmaceuticalsRecruiting5 Years8 YearsMale89Phase 2United States
60NCT04371666
(ClinicalTrials.gov)
July 30, 202029/4/2020A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Pamrevlumab;Drug: PlaceboFibroGenNULLActive, not recruiting12 YearsN/AMale90Phase 3United States;Australia;Austria;Belgium;Canada;China;Czechia;France;Israel;Italy;Netherlands;Spain;Switzerland;United Kingdom
61NCT04428476
(ClinicalTrials.gov)
July 20, 20209/6/2020Open-label Extension of the HOPE-2 TrialOpen-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy TrialDuchenne Muscular DystrophyBiological: CAP-1002Capricor Inc.NULLActive, not recruiting10 YearsN/AAll13Phase 2United States
62EUCTR2018-001762-42-NL
(EUCTR)
20/07/202021/07/2020A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne MuscularDystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
152Phase 3Korea, Republic of;Sweden;New Zealand;Netherlands;Germany;Norway;Australia;Denmark;Belgium;Poland;Canada;Mexico;France;Switzerland;United Kingdom;Colombia;Russian Federation;Turkey;Ireland;Spain;Taiwan;Czechia;United States
63NCT04386304
(ClinicalTrials.gov)
July 13, 20201/5/2020Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular DystrophyA Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of Orally Administered (+)-Epicatechin in Patients With Becker or Becker-like Muscular Dystrophy With Continued Ambulation Past 16 Years of AgeBecker Muscular DystrophyDrug: (+)-EpicatechinEpirium Bio Inc.NULLActive, not recruiting16 Years59 YearsMale20Phase 1United States
64NCT03992430
(ClinicalTrials.gov)
July 13, 202018/6/2019A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 SkippingMuscular Dystrophy, DuchenneDrug: EteplirsenSarepta Therapeutics, Inc.NULLActive, not recruiting7 Years13 YearsMale152Phase 3United States;Canada;Korea, Republic of;Taiwan
65EUCTR2019-004426-24-IT
(EUCTR)
06/07/202015/06/2021Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers.Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers. - TREAT-LMNA Congenital Muscolar distrophy LMNA related
MedDRA version: 20.0;Level: LLT;Classification code 10003718;Term: Atrophy skeletal muscle;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Deflazacort
Product Code: [Deflazacort]
INN or Proposed INN: deflazacort
Trade Name: Lucen
Product Name: Lucen
Product Code: [-]
INN or Proposed INN: esomeprazolo
Other descriptive name: esomeprazole
AZIENDA OSPEDALIERO-UNIVERSITARIA PISANANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2Italy
66NCT04433234
(ClinicalTrials.gov)
June 30, 202012/6/2020Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular DystrophyA Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: DS-5141bDaiichi Sankyo Co., Ltd.NULLActive, not recruiting5 YearsN/AMale8Phase 2Japan
67EUCTR2019-003563-22-IE
(EUCTR)
23/06/202021/01/2020A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular DystrophyAn Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: EDASALONEXENT
Catabasis Pharmaceuticals Inc.NULLNot RecruitingFemale: no
Male: yes
140Phase 3United States;Canada;Ireland;Australia;Germany;United Kingdom;Sweden
68EUCTR2017-004625-32-SE
(EUCTR)
17/06/202009/08/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
260Phase 3United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden
69NCT03895528
(ClinicalTrials.gov)
June 8, 202027/3/2019Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid LaminopathyA Treatment IND (Investigational New Drug) Protocol for EAP (Expanded Access Program) for the Use of Lonafarnib in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) or Progeroid LaminopathyProgeria;HGPSDrug: LonafarnibEiger BioPharmaceuticalsNULLApproved for marketing12 MonthsN/AAllNULL
70ChiCTR2000032525
2020-05-012020-05-01Early intervention for subclinical myocardial prevention of ACEI and beta-blocker in patients with Duchenne muscular dystrophy: a prospective multicenter randomized controlled clinical trialEarly intervention for subclinical myocardial prevention of ACEI and beta-blocker in patients with Duchenne muscular dystrophy: a prospective multicenter randomized controlled clinical trial Duchenne muscular dystrophyGroup A:Perindopril;Group B:metoprolol;Group C:Perindopril and metoprolol;West China Second University Hospital, Sichuan UniversityNULLRecruiting118MaleGroup A:106;Group B:106;Group C:106;Phase 4China
71EUCTR2018-001762-42-SE
(EUCTR)
27/04/202026/09/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
152Phase 3United States;Taiwan;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;United Kingdom;Italy;France;Canada;Argentina;Belgium;Peru;Australia;Denmark;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of
72EUCTR2019-003563-22-GB
(EUCTR)
23/04/202013/01/2020A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular DystrophyAn Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy - An Open-Label Extension Study of Edasalonexent in DMD Patients Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: EDASALONEXENT
Catabasis Pharmaceuticals Inc.NULLNot RecruitingFemale: no
Male: yes
140Phase 3United States;Canada;Ireland;Australia;Germany;United Kingdom;Sweden
73EUCTR2019-003563-22-DE
(EUCTR)
16/04/202031/01/2020A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular DystrophyAn Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: EDASALONEXENT
Catabasis Pharmaceuticals Inc.NULLNot RecruitingFemale: no
Male: yes
140Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
74EUCTR2019-003563-22-SE
(EUCTR)
15/04/202003/02/2020A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular DystrophyAn Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: EDASALONEXENT
Catabasis Pharmaceuticals Inc.NULLNot RecruitingFemale: no
Male: yes
140Phase 3United States;Canada;Ireland;Australia;Germany;United Kingdom;Sweden
75NCT04060199
(ClinicalTrials.gov)
April 14, 202015/8/2019Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Viltolarsen;Drug: PlaceboNS Pharma, Inc.Nippon Shinyaku Co., Ltd.Recruiting4 Years7 YearsMale74Phase 3United States;Australia;Canada;Chile;Greece;Hong Kong;Italy;Japan;Korea, Republic of;Mexico;Netherlands;New Zealand;Norway;Russian Federation;Spain;Taiwan;Turkey;Ukraine;United Kingdom;Sweden
76EUCTR2019-000601-77-NL
(EUCTR)
10/04/202025/11/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
60Phase 2United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy
77EUCTR2015-002069-52-NO
(EUCTR)
07/04/202030/09/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
222Phase 3Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Germany;Norway;Sweden
78EUCTR2018-001762-42-IE
(EUCTR)
03/04/202001/07/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
154Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden
79EUCTR2019-004602-94-BE
(EUCTR)
27/03/202003/02/2020NAA 3-part, Randomized, Double Blind, Adaptive Seamless Phase 1-3 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO101 in Non-Ambulatory Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy and Evidence of Respiratory Deterioration - Adaptive Seamless Phase 1-3 Study of Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO Duchenne Muscular Dystrophy regardless the genotype
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: BIO101
INN or Proposed INN: BIO101
Other descriptive name: BIO101
Product Code: BIO101
INN or Proposed INN: BIO101
Other descriptive name: BIO101
Product Code: BIO101
INN or Proposed INN: BIO101
Other descriptive name: BIO101
BiophytisNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
260Phase 1Belgium
80EUCTR2017-004554-42-FR
(EUCTR)
26/03/202017/08/2018The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Tamoxifen 20mg Hexal® Filmtabletten
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE
University of Basel Children's Hospital, Division of NeuropediatricsNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
100Phase 3France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
81EUCTR2016-000602-10-BG
(EUCTR)
10/03/202007/01/2020A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
82EUCTR2019-002076-13-NL
(EUCTR)
03/03/202025/11/2019This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of
83EUCTR2019-002076-13-GB
(EUCTR)
02/03/202016/09/2019This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3Korea, Republic of;Sweden;Japan;Netherlands;Australia;Poland;Brazil;Belgium;Canada;France;Italy;United Kingdom;Russian Federation;Chile;Turkey;Spain;Taiwan;United States
84NCT04254172
(ClinicalTrials.gov)
February 19, 202031/1/2020A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular DystrophyA SINGLE-SITE, PROSPECTIVE, NATURAL HISTORY LOW INTERVENTIONAL STUDY TO ESTABLISH NORMATIVE DATA OF REAL-WORLD ACTIVITY MEASURES USING WEARABLE SENSORS IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY (DMD)Duchenne Muscular Dystrophy (DMD)Device: Activity MonitorPfizerNULLTerminated4 Years12 YearsMale2United States
85NCT04179409
(ClinicalTrials.gov)
February 18, 202012/8/2019A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD DuplicationsA 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD DuplicationsDuchenne Muscular DystrophyDrug: Casimersen;Drug: Eteplirsen;Drug: GolodirsenKevin FlaniganSarepta Therapeutics, Inc.Active, not recruiting6 MonthsN/AMale3Phase 2United States
86NCT04264442
(ClinicalTrials.gov)
February 13, 202030/1/2020Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)Facioscapulohumeral Muscular Dystrophy (FSHD)Drug: LosmapimodFulcrum TherapeuticsNULLEnrolling by invitation18 Years65 YearsAll76Phase 2United States;Canada;France;Spain;Germany
87EUCTR2019-002076-13-IT
(EUCTR)
27/01/202005/01/2021This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: [NS-065/NCNP-01]
INN or Proposed INN: VILTOLARSEN
NS Pharma, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
74Phase 3United States;Taiwan;Greece;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Brazil;Belgium;Australia;Netherlands;Japan;Sweden;Korea, Republic of
88EUCTR2017-004279-30-NL
(EUCTR)
23/01/202016/05/2018A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;France;Spain;Belgium;Austria;Israel;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden
89EUCTR2017-000397-10-GB
(EUCTR)
20/01/202015/03/2019Study in which all the patients take the same investigational drug with theaim of evaluate the long-term safety, tolerability, and efficacy ofGIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophywho have been already treated in one of the GIVINOSTAT studies in thepastOpen label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies. - Givinostat DMD long term study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GIVINOSTAT (hyrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
185Phase 3France;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom
90EUCTR2019-000601-77-ES
(EUCTR)
15/01/202005/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
24Phase 2United States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;United Kingdom;Italy
91NCT04240314
(ClinicalTrials.gov)
January 15, 202022/1/2020AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: scAAV9.U7.ACCAMegan WaldropAudentes TherapeuticsEnrolling by invitation6 Months13 YearsMale3Phase 1/Phase 2United States
92EUCTR2018-001762-42-DK
(EUCTR)
18/12/201930/10/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
154Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden
93EUCTR2019-002076-13-ES
(EUCTR)
16/12/201906/11/2019This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Viltolarsen
Product Code: NS-065/NCNP-01
INN or Proposed INN: VILTOLARSEN
NS Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
74 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Taiwan;Spain;Turkey;Russian Federation;Chile;United Kingdom;Italy;France;Canada;Poland;Belgium;Brazil;Australia;Netherlands;Japan;Sweden;Korea, Republic of
94EUCTR2019-000601-77-IE
(EUCTR)
11/12/201926/06/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
24Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy
95NCT04129294
(ClinicalTrials.gov)
December 2, 201915/10/2019Exploratory Study of NS-089/NCNP-02 in DMDExploratory Study of NS-089/NCNP-02 in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: NS-089/NCNP-02National Center of Neurology and Psychiatry, JapanNippon Shinyaku Co., Ltd.Enrolling by invitation4 Years17 YearsMale6Phase 1/Phase 2Japan
96EUCTR2015-002069-52-GR
(EUCTR)
29/11/201911/09/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
97EUCTR2015-002069-52-DK
(EUCTR)
26/11/201925/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
98NCT02878694
(ClinicalTrials.gov)
November 14, 20198/3/2016Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous GraftTreatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous GraftMuscular Dystrophy, Oculopharyngeal;PtosisBiological: Myoblast autologous graftUniversity Hospital, CaenUniversity Hospital, Rouen;Rennes University Hospital;Centre Hospitalier Universitaire, Amiens;University Hospital, Lille;University Hospital, Brest;Groupe Hospitalier Pitie-SalpetriereTerminated18 Years75 YearsAll1Phase 2/Phase 3France
99EUCTR2019-000601-77-GB
(EUCTR)
04/11/201912/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-5051
Product Code: SRP-5051
INN or Proposed INN: SRP-5051
Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
70Phase 2United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;Italy;United Kingdom
100EUCTR2018-001762-42-ES
(EUCTR)
29/10/201909/08/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
152 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noTaiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;Korea, Republic of
101EUCTR2018-004740-36-DK
(EUCTR)
29/10/201925/06/2019A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2IA MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
PTC Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;France;Canada;Denmark;Russian Federation;Norway;Germany;Sweden
102EUCTR2019-001181-15-ES
(EUCTR)
29/10/201929/10/2019Safety and Efficacy of Losmapimod in Patients with FSHDA Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Losmapimod
Product Code: FTX-1821
INN or Proposed INN: LOSMAPIMOD
Fulcrum Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
66 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Spain;Germany
103EUCTR2018-004740-36-DE
(EUCTR)
14/10/201929/04/2019A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2IA MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
PTC Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden
104EUCTR2017-002704-27-NL
(EUCTR)
10/10/201925/02/2019A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
ReveraGen BioPharma, Inc.NULLNot RecruitingFemale: no
Male: yes
120Phase 2United States;Czechia;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden
105EUCTR2017-000397-10-NL
(EUCTR)
01/10/201906/11/2019Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the pastOpen label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3France;United States;Serbia;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy
106EUCTR2018-004009-22-CZ
(EUCTR)
25/09/201926/06/2019Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular DystrophyA Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Other descriptive name: suvodirsen
Wave Life Sciences UK LimitedNULLNot RecruitingFemale: no
Male: yes
150Phase 2;Phase 3United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Netherlands;Japan;Sweden
107EUCTR2019-001825-28-GB
(EUCTR)
24/09/201929/07/2019 Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy: a phase I/IIa study / DMD04Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open label, phase I/IIa study. - Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Lentiviral Vector Trans-Skip gene modified Autologous MesoangioblastsThe University of ManchesterNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
5 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
108EUCTR2019-001181-15-FR
(EUCTR)
17/09/201929/05/2019Safety and Efficacy of Losmapimod in Patients with FSHDA Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Losmapimod
Product Code: FTX-1821
INN or Proposed INN: LOSMAPIMOD
Fulcrum Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
66 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Spain;Germany
109EUCTR2018-001762-42-GB
(EUCTR)
17/09/201904/07/2019A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophyA Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
122Phase 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;Switzerland;United Kingdom;France;Mexico;Canada;Poland;Belgium;Australia;Denmark;Norway;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
110NCT03907072
(ClinicalTrials.gov)
September 4, 20195/4/2019Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular DystrophyA Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51)Duchenne Muscular DystrophyDrug: WVE-210201 (suvodirsen);Drug: PlaceboWave Life Sciences Ltd.NULLTerminated5 Years12 YearsMale6Phase 2/Phase 3United States;Belgium;Canada;Czechia;France;Italy;Sweden;United Kingdom
111NCT04004000
(ClinicalTrials.gov)
August 23, 201925/6/2019Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With ExtensionAn Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) With ExtensionFacioscapulohumeral Muscular Dystrophy 1Drug: LosmapimodFulcrum TherapeuticsNULLActive, not recruiting18 Years65 YearsAll14Phase 2Netherlands
112EUCTR2018-004740-36-FR
(EUCTR)
19/08/201904/06/2019A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2IA MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
Trade Name: EMFLAZA® (DEFLAZACORT)
Product Name: EMFLAZA® (deflazacort)
Product Code: EMFLAZA® (deflazacort)
INN or Proposed INN: Deflazacort
Other descriptive name: DEFLAZACORT
PTC Therapeutics, Inc.NULLNot RecruitingFemale: yes
Male: yes
100Phase 3United States;France;Canada;Denmark;Norway;Germany;Sweden
113EUCTR2017-002704-27-ES
(EUCTR)
13/08/201911/06/2019A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
ReveraGen BioPharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
114NCT04003974
(ClinicalTrials.gov)
August 9, 201925/6/2019Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)Facioscapulohumeral Muscular Dystrophy (FSHD)Drug: Losmapimod;Drug: Placebo oral tabletFulcrum TherapeuticsNULLCompleted18 Years65 YearsAll80Phase 2United States;Canada;France;Spain;Germany
115EUCTR2017-002704-27-GR
(EUCTR)
01/08/201910/06/2019A Study to Assess the efficacy and the safety of Vamorolone in Boys withDuchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- andActive-controlled Study with Double-Blind Extension to Assess the Efficacyand Safety of Vamorolone in Ambulant Boys with Duchenne MuscularDystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
ReveraGen BioPharma, IncNULLNot RecruitingFemale: no
Male: yes
120Phase 2Czech Republic;Canada;Greece;Spain;Belgium;Australia;Israel;Netherlands;Germany;United Kingdom;Italy;Sweden
116EUCTR2018-004009-22-SE
(EUCTR)
20/07/201905/04/2019Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular DystrophyA Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Other descriptive name: suvodirsen
Wave Life Sciences UK LimitedNULLNot Recruiting Female: no
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden
117EUCTR2018-000692-32-IT
(EUCTR)
17/07/201914/01/2020Trial to evaluate the efficacy of the drug metformin on the muscles in patients with myotonic distrophy type 1 EFFICACY OF METFORMIN ON MOTILITY AND STRENGTH IN MYOTONIC DYSTROPHY TYPE 1. A randomized, double blind, placebo-controlled, multicenter clinical trial. - MetMyd Myotonic Distrophy type 1, confirmed by genetic testing, with a CTG expansion size >100
MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Level: LLT;Classification code 10013987;Term: Dystrophia myotonica;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Trade Name: METFORMINA - 500 30 COMPRESSE 500 MG
Product Name: Metformina
INN or Proposed INN: Metformina
DIP. MEDICINA DEI SISTEMI UNIVERSITà DEGLI STUDI DI ROMA TOR VERGATANULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
194 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noItaly
118EUCTR2018-004009-22-IT
(EUCTR)
09/07/201905/11/2020Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular DystrophyA Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy - - Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: [WVE-210201]
Other descriptive name: suvodirsen
Wave Life Sciences UK LimitedNULLNot RecruitingFemale: no
Male: yes
150Phase 2;Phase 3United States;Czechia;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Netherlands;Japan;Sweden
119EUCTR2017-002704-27-BE
(EUCTR)
06/07/201921/02/2018A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
ReveraGen BioPharma, Inc.NULLNot RecruitingFemale: no
Male: yes
120Phase 2United States;Czechia;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
120NCT04054375
(ClinicalTrials.gov)
July 1, 20197/8/2019Weekly Steroids in Muscular DystrophyOpen Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular DystrophyLimb-girdle Muscular Dystrophy;Becker Muscular DystrophyDrug: PrednisoneNorthwestern UniversityNULLActive, not recruiting18 Years65 YearsAll20Phase 2United States
121EUCTR2019-000337-39-GB
(EUCTR)
28/06/201923/04/2019A research study of a new investigational medicinal product for thetreatment of Duchenne Muscular Dystrophy patients who have completedstudy 4658-102An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen inPatients with Duchenne Muscular Dystrophy Who Have Completed Study4658-102 Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;United Kingdom
122EUCTR2018-004009-22-GB
(EUCTR)
27/06/201928/03/2019Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular DystrophyA Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Other descriptive name: suvodirsen
Wave Life Sciences UK LimitedNULLNot RecruitingFemale: no
Male: yes
150Phase 2;Phase 3United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden
123NCT04004065
(ClinicalTrials.gov)
June 26, 201927/6/2019Two-Part Study for Dose Determination of SRP-5051 (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentA Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping TreatmentDuchenne Muscular DystrophyDrug: SRP-5051Sarepta Therapeutics, Inc.NULLRecruiting7 Years21 YearsMale60Phase 2United States;Belgium;Canada;Spain;United Kingdom
124NCT03985878
(ClinicalTrials.gov)
June 26, 201911/6/2019A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular DystrophyAn Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102Duchenne Muscular DystrophyDrug: EteplirsenSarepta Therapeutics, Inc.NULLEnrolling by invitation2 Years5 YearsMale15Phase 2Belgium;France;Italy;United Kingdom
125EUCTR2017-002704-27-CZ
(EUCTR)
19/06/201922/03/2019A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
ReveraGen BioPharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
126NCT03959189
(ClinicalTrials.gov)
June 17, 201916/5/2019Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1Myotonic Dystrophy, Type 1 (DM1);Myotonic DystrophyDrug: ERX-963;Drug: PlaceboExpansion Therapeutics, Inc.NULLCompleted18 Years65 YearsAll12Phase 1United States
127EUCTR2019-000337-39-IT
(EUCTR)
13/06/201901/02/2021A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 - Not applicable Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: [AVI-4658]
INN or Proposed INN: ETEPLIRSEN
SAREPTA THERAPEUTICS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;United Kingdom;Italy
128EUCTR2017-000397-10-BE
(EUCTR)
12/06/201912/02/2019Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the pastOpen label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
185Phase 3United States;France;Canada;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy
129EUCTR2018-004009-22-BE
(EUCTR)
07/06/201921/05/2019Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular DystrophyA Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Other descriptive name: suvodirsen
Wave Life Sciences UK LimitedNULLNot RecruitingFemale: no
Male: yes
150Phase 2;Phase 3United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Netherlands;Japan;Sweden
130NCT03789734
(ClinicalTrials.gov)
June 4, 201912/12/2018Safety Study of BLS-M22 in Healthy VolunteersA Dose Block-randomized, Double-blind, Placebo-controlled and Dose-escalation Phase I Clinical Trial to Evaluate Safety of BLS-M22 Following Single/Multiple Oral Administration in Healthy Adult VolunteersMuscular Dystrophy, DuchenneBiological: BLS-M22;Other: PlaceboBioLeaders CorporationNULLCompleted19 Years55 YearsAll37Phase 1Korea, Republic of
131EUCTR2019-000337-39-BE
(EUCTR)
03/06/201908/04/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: ETEPLIRSEN
Sarepta Therapeutics, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;United Kingdom
132EUCTR2017-000397-10-ES
(EUCTR)
16/05/201911/04/2019Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the pastOpen label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD study long term Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
185 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom
133NCT03783923
(ClinicalTrials.gov)
May 15, 201919/12/2018A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)A Multicenter Open Label Study on the Safety and Efficacy of Deflazacort (Emflaza®) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)Limb-Girdle Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLCompleted18 YearsN/AAll30Phase 3United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden
134EUCTR2019-000305-79-ES
(EUCTR)
14/05/201912/04/2019An extension study to the A083-02 and A083-03 studies that evaluate the long-term effect of the ACE-083 investigational product in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) previously participated in study A083-02 and also in patients with Charcot-Marie Tooth (CMT) disease type 1 and X who previously participated in study A083-03.An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03 Facioscapulohumeral Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ACE-083
Product Code: ACE-083
INN or Proposed INN: pending
Other descriptive name: ACE-083
Acceleron Pharma Inc.NULLNot Recruiting Female: yes
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Spain
135NCT03943290
(ClinicalTrials.gov)
May 10, 201917/4/2019Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients With Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03Facioscapulohumeral Muscular Dystrophy;Charcot-Marie-Tooth DiseaseDrug: ACE-083Acceleron Pharma, Inc.NULLTerminated18 YearsN/AAll62Phase 2United States;Canada;Spain
136NCT03936894
(ClinicalTrials.gov)
May 1, 201929/4/2019Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular DystrophyA Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) Assessing Safety and Biomarker Changes in Boys With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Canakinumab Injection [Ilaris]Children's National Research InstituteFoundation to Eradicate DuchenneRecruiting2 YearsN/AMale6Phase 1/Phase 2United States
137NCT04999735
(ClinicalTrials.gov)
April 14, 20192/7/2019Digital Biotyping of FSHD Patients and ControlsAn Exploratory, Non-interventional Study to Biotype Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Controls Using Digital TechnologiesMuscular Dystrophy, FacioscapulohumeralBehavioral: CHDR Monitoring Remotely (MORE);Behavioral: Withings Steel HR;Behavioral: Withings Body+ scale;Behavioral: Withings Blood Pressure MonitorCentre for Human Drug Research, NetherlandsFacio TherapeuticsCompleted16 YearsN/AAll58Netherlands
138NCT03796637
(ClinicalTrials.gov)
April 11, 20194/1/2019A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With AtalurenPhase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for =9 MonthsDuchenne Muscular DystrophyOther: Dystrophin levels;Drug: AtalurenPTC TherapeuticsNULLCompletedN/AN/AMale6Phase 2United States
139NCT03917719
(ClinicalTrials.gov)
March 14, 201912/4/2019An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular DystrophyAn Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: EdasalonexentCatabasis PharmaceuticalsNULLTerminated4 Years12 YearsMale130Phase 3United States;Australia;Canada;Germany;Sweden;United Kingdom
140EUCTR2015-001192-48-FR
(EUCTR)
04/03/201911/09/2015Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes.Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes. - TTT-PT-DOP Patients atteints d’une DMOP et présentant un ptosis
MedDRA version: 18.0;Level: LLT;Classification code 10010498;Term: Congenital hereditary muscular dystrophy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Myoblastes autologuesCHU CAENNULLNot RecruitingFemale: yes
Male: yes
10Phase 2France
141NCT02592941
(ClinicalTrials.gov)
March 1, 201929/10/2015Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular DystrophyAn Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsParexel;Dohmen Life Science ServicesApproved for marketing5 YearsN/AAllUnited States
142EUCTR2017-004279-30-AT
(EUCTR)
15/02/201904/01/2019A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
143EUCTR2018-000975-34-NL
(EUCTR)
06/02/201916/08/2018A Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Wave Life Sciences Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
40 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Canada;Belgium;Netherlands;Italy;United Kingdom
144EUCTR2017-004625-32-FR
(EUCTR)
05/02/201923/07/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
260 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
145EUCTR2018-000464-29-DE
(EUCTR)
31/01/201924/09/2018A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophyA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMD Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: EDASALONEXENT
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: EDASALONEXENT
Catabasis Pharmaceuticals, Inc.NULLNot RecruitingFemale: no
Male: yes
126Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
146EUCTR2017-004554-42-GB
(EUCTR)
29/01/201903/08/2018The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open labelextension of a multicentre, randomised, double-blind, placebo-controlled,phase 3 safety and efficacy trial Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Tamoxifen 20mg Hexal® Filmtabletten
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE
University of Basel Children's Hospital, Division of NeuropediatricsNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
100Phase 3France;Belgium;Spain;Netherlands;Germany;Switzerland;United Kingdom
147NCT03863119
(ClinicalTrials.gov)
January 28, 201921/2/2019Expanded Access Protocol for Boys With Duchenne Muscular DystrophyAn Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 StudiesDuchenne Muscular DystrophyDrug: VamoroloneReveraGen BioPharma, Inc.NULLAvailableN/AN/AMaleUnited States;Canada;Israel
148EUCTR2017-004554-42-ES
(EUCTR)
22/01/201917/09/2018The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. Tamoxifen in Duchenne muscular dystrophy - TAMDMD A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: TAMOX
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE
University of Basel Children's Hospital, Division of NeuropediatricsNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
149EUCTR2018-000464-29-IE
(EUCTR)
16/01/201902/10/2018A study to compare edasalonexent with placebo in pediatric patients with Duchenne Muscular dystrophy.A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OFEDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - Phase 3 Study of Edasalonexent in Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: Edasalonexent
Catabasis Pharmaceuticals, Inc.NULLNot RecruitingFemale: no
Male: yes
126Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
150EUCTR2017-004554-42-NL
(EUCTR)
16/01/201902/10/2018The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease.Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: TAMOXIFEN
INN or Proposed INN: Tamoxifen
Other descriptive name: TAMOXIFEN CITRATE
University of Basel Children's Hospital, Division of NeuropediatricsNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
100Phase 3France;Belgium;Spain;Turkey;Germany;Netherlands;United Kingdom;Switzerland
151NCT03779646
(ClinicalTrials.gov)
January 16, 201914/12/2018Bisoprolol in DMD Early CardiomyopathyBisoprolol for Early Cardiomyopathy in Duchenne Muscular Dystrophy: a Randomized, Controlled TrialDuchenne Muscular Dystrophy;Cardiomyopathy, DilatedDrug: Bisoprolol FumaratePeking Union Medical College HospitalNational Natural Science Foundation of China;Chinese Academy of Medical SciencesRecruiting7 YearsN/AMale42Phase 2/Phase 3China
152EUCTR2017-002213-60-GB
(EUCTR)
08/01/201911/12/2018A study to investigate the safety, tolerability, and efficacy of SGT-001 in male adolescents and children with Duchenne muscular dystrophyA randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SGT-001
Product Code: rAAV9-CK8-h-µD5
INN or Proposed INN: SGT-001
Other descriptive name: rAAV9-CK8-h-µD5
Solid Biosciences Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
153NCT03648827
(ClinicalTrials.gov)
December 21, 201824/8/2018A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With AtalurenDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLCompleted2 Years7 YearsMale20Phase 2United States
154EUCTR2017-004279-30-ES
(EUCTR)
21/12/201816/11/2018A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;France;Belgium;Spain;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
155EUCTR2017-004625-32-BE
(EUCTR)
20/12/201810/09/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
150 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSweden;Germany;Australia;Belgium;Poland;Canada;Czech Republic;France;United Kingdom;Italy;Israel;Ireland;Spain;Finland;United States
156NCT03675126
(ClinicalTrials.gov)
December 19, 20186/8/2018An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051Muscular Dystrophy, DuchenneDrug: SRP-5051Sarepta Therapeutics, Inc.NULLTerminated4 YearsN/AMale15Phase 1/Phase 2United States;Canada
157JPRN-jRCTs031180038
18/12/201812/11/2018Tranilast-MDA multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD muscular dystrophy
muscular dystrophy ,heart failure ,tranilast;muscular dystrophies
Tranilast 300 mg / day is administered three times per minute.
Treatment for 28 weeks (in principle, outpatient administration). As of the 28th week, reconfirmation of consent regarding continuation of administration is confirmed, and if confirmation is obtained, further treatment for 116 weeks is carried out.
Matsumura TsuyoshiNULLNot Recruiting>= 13age oldNot applicableBoth20N/AJapan
158EUCTR2016-000602-10-HU
(EUCTR)
18/12/201805/11/2018A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
159JPRN-UMIN000031965
2018/12/1430/03/2018A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophyA multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD Heart failure patients with muscular dystrophy who showed brain natriuretic peptide (BNP) of 100 pg/mL or more in spite of myocardial protection treatmentTranilast 300 mg/day is administered three times per day as the study treatment.
The start date of study treatment is defined as the medication start date, and 28 weeks of study treatment will be performed (in principle, by outpatient administration). We reconfirm consent for continuation of administration at 28 weeks, and if consent is obtained, further treatment for 116 weeks will be carried out.
As long as there are no particular problems, the outpatient visit during the observation period is in 4 weeks (21-35 days), 12 weeks (77-91 days), 20 weeks (134-147 days), 24 weeks (161-175 days), and 28 weeks (190-203 days) after starting medication. A prescription until the next outpatient visit will be issued at the consultation. The outpatient visit after consent reconfirmation is done at 12-weeks intervals after 36 weeks, and the prescription until the next outpatient visit will be issued until the next 144 weeks after starting medication at the consultation.
Evaluation of various examination findings including clinical findings, cardiac functions, respiratory functions, motor function, QOL Questionnaire (MDQoL-60, SF-12), and adverse events at the designated timing.
In order to confirm TRPV2 inhibitory effect by tranilast and to assess its effectiveness as a biomarker, central laboratory tests for the TRPV2 expression analysis will be performed.
National Hospital Organization Toneyama National HospitalNULLComplete: follow-up continuing13years-oldNot applicableMale and Female20Not selectedJapan
160EUCTR2018-000464-29-SE
(EUCTR)
11/12/201824/09/2018A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy.A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OFEDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMD Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Edasalonexent
Product Code: CAT-1004
INN or Proposed INN: Edasalonexent
Catabasis Pharmaceuticals, Inc.NULLNot RecruitingFemale: no
Male: yes
126Phase 3Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden;United States
161EUCTR2015-002069-52-PL
(EUCTR)
11/12/201813/09/2018A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
162NCT03777319
(ClinicalTrials.gov)
December 5, 201810/12/2018Spironolactone Versus Prednisolone in DMDA Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMDMuscular Dystrophy, DuchenneDrug: Spironolactone;Drug: PrednisoloneKevin FlaniganMuscular Dystrophy AssociationTerminated4 Years7 YearsMale2Phase 1United States
163EUCTR2016-004623-23-GB
(EUCTR)
19/11/201808/02/2018A Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic DystrophyA Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy - Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy Treatment of child and adolescent congenital myotonic dystrophy.
MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Tideglusib
INN or Proposed INN: Tideglusib
Other descriptive name: 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione
AMO Pharma LtdNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
56 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Canada;United Kingdom
164EUCTR2017-001629-41-NL
(EUCTR)
15/11/201831/05/2018Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular DystrophyA randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy ofgivinostat in patients with Becker Muscular Dystrophy - NA Distrofia Muscolare di Becker (DMB)
MedDRA version: 20.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.P.A.NULLNot RecruitingFemale: no
Male: yes
48Phase 2Netherlands;Italy
165EUCTR2018-000975-34-BE
(EUCTR)
13/11/201821/09/2018A Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001A Multicenter, Open-Label Extension Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001 Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Wave Life Sciences Ltd.NULLNot RecruitingFemale: no
Male: yes
40Phase 1France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom
166NCT03642145
(ClinicalTrials.gov)
October 31, 201810/7/2018A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)A 52-Week Phase 3B Randomized Open-Label Study Evaluating the Safety and Pharmacokinetics of Emflaza® (Deflazacort) Compared to a Comparable Natural History Control Group in Males Aged =2 to <5 Years With Duchenne Muscular Dystrophy (DMD) Followed by a 52-Week Extension PeriodDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLWithdrawn2 Years4 YearsMale0Phase 3United States
167EUCTR2017-004279-30-GB
(EUCTR)
26/10/201809/05/2018A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden
168EUCTR2018-000975-34-GB
(EUCTR)
25/10/201826/06/2018A Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001A Multicenter, Open-Label Extension Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001 Duchenne muscular dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Wave Life Sciences Ltd.NULLNot RecruitingFemale: no
Male: yes
40Phase 1France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom
169NCT03703882
(ClinicalTrials.gov)
October 2, 20188/10/2018Phase III Study of Edasalonexent in Boys With Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Edasalonexent;Drug: PlaceboCatabasis PharmaceuticalsNULLCompleted4 Years7 YearsMale131Phase 3United States;Australia;Canada;Germany;Ireland;Israel;Sweden;United Kingdom
170EUCTR2017-002704-27-GB
(EUCTR)
21/09/201825/06/2019A Study to Assess the efficacy and the safety of Vamorolone in comparison to Prednisone in Boys with Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
ReveraGen BioPharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
171ChiCTR1800018340
2018-09-122018-09-12Clinical study for cardiac MRI in evaluating early myocardial lesions in patients with muscular dystrophyClinical study for cardiac MRI in evaluating early myocardial lesions in patients with muscular dystrophy Muscular dystrophyGold Standard:Genomic sequencing, muscle biopsy;Index test:Myocardial strain, T1/T2mapping value, late delayed enhancement (LGE), myocardial first pass perfusion;Department of Radiology, West China Second Hospital, Sichuan UniversityNULLRecruiting418MaleTarget condition:200;Difficult condition:30China
172EUCTR2017-004625-32-ES
(EUCTR)
07/09/201810/09/2018A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
150Phase 3United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
173EUCTR2017-004139-35-IT
(EUCTR)
31/08/201817/06/2021Clinical Trial in Male Subjects with Muscular DiseaseA Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy - Clinical Trial in Male Subjects with Muscular Disease Duchenne Muscular Distrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Cosintropina acetato
Product Code: [MNK-1411]
INN or Proposed INN: TETRACOSACTIDE ESACETATO
Other descriptive name: Cosyntropin acetate
Trade Name: Synacthen Ampoules 250 mcg
Product Name: Synacthen Ampoules 250 microgrammi
Product Code: [Synacthen Ampoules 250 microgrammi]
INN or Proposed INN: TETRACOSACTIDE
MALLINCKRODT ARD INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
132Phase 2Serbia;United States;Spain;Costa Rica;Ukraine;Ireland;Turkey;Israel;Russian Federation;United Kingdom;Italy;India;Mexico;Canada;Puerto Rico;Poland;Bulgaria
174EUCTR2017-004279-30-IT
(EUCTR)
29/08/201822/01/2021A clinical study to assess the long-term safety and efficacy of idebenonetreatment in patients with Duchenne Muscular Dystrophy (DMD) whocompleted the SIDEROS study.A Phase III Open-Label Extension Study to Assess the Long-Term Safetyand Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy(DMD) who completed the SIDEROS study - SIDEROS-E Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
Product Code: [Idebenone]
INN or Proposed INN: IDEBENONE
SANTHERA PHARMACEUTICALSNULLNot RecruitingFemale: no
Male: yes
266Phase 3France;United States;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
175EUCTR2017-004139-35-BG
(EUCTR)
28/08/201828/06/2018Clinical Trial in Male Subjects with Muscular DiseaseA Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy Duchenne Muscular Distrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Cosyntropin acetate
Product Code: MNK-1411
INN or Proposed INN: TETRACOSACTIDE HEXAACETATE
Other descriptive name: Cosyntropin acetate
Mallinckrodt ARD Inc.NULLNot Recruiting Female: no
Male: yes
132 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noSerbia;United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Bulgaria;Germany;Netherlands
176EUCTR2017-002704-27-SE
(EUCTR)
15/08/201812/03/2018A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: VAMOROLONE
Product Name: Prednisone
INN or Proposed INN: PREDNISONE
Other descriptive name: PREDNISONE
ReveraGen BioPharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2United States;Czechia;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
177NCT03558958
(ClinicalTrials.gov)
August 8, 20185/6/2018Safety and Efficacy of P-188 NF in DMD PatientsAn Exploratory, Open-label Study to Assess the Effect of P-188 NF (Carmeseal-MD) on Safety, on Respiratory and Cardiac Dysfunction and on Upper Limb Strength in Non-ambulatory Patients With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: P-188 NFPhrixus Pharmaceuticals, Inc.Charley's FundSuspended12 Years25 YearsMale10Phase 2United States
178EUCTR2015-002069-52-BG
(EUCTR)
06/08/201811/06/2018A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Denmark;Australia;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Brazil;Poland;Georgia;Bulgaria;Norway;Germany;Sweden
179NCT03532542
(ClinicalTrials.gov)
August 2, 201810/5/2018An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular DystrophyLong-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or GolodirsenDuchenne Muscular DystrophyDrug: Casimersen;Drug: GolodirsenSarepta Therapeutics, Inc.NULLEnrolling by invitation7 Years23 YearsMale260Phase 3United States;Belgium;Bulgaria;Canada;Czechia;France;Germany;Israel;Italy;Poland;Spain;Sweden;United Kingdom
180EUCTR2017-004625-32-GB
(EUCTR)
26/07/201823/01/2019A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsLong-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: CASIMERSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: GOLODIRSEN
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
260Phase 3United States;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Sweden
181NCT03400852
(ClinicalTrials.gov)
July 17, 20189/1/2018A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular DystrophyA Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Participants 4 to 8 Years of Age With Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Cosyntropin;Other: PlaceboMallinckrodt ARD LLCNULLTerminated4 Years8 YearsMale44Phase 2United States;Bulgaria;Israel;Italy;Mexico;Serbia;Spain;Turkey
182EUCTR2016-001615-21-BG
(EUCTR)
06/07/201812/04/2018An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 yearsA Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Pfizer Inc. 235 East 42nd Street, New York, NY 10017NULLNot Recruiting Female: no
Male: yes
105 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom
183NCT03603288
(ClinicalTrials.gov)
July 4, 201831/5/2018Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS StudyDuchenne Muscular DystrophyDrug: idebenone 150 mg film-coated tabletsSanthera PharmaceuticalsNULLTerminated11 YearsN/AMale161Phase 3United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom
184NCT03603171
(ClinicalTrials.gov)
July 1, 201817/7/2018Clinical Outcome Measures in Myotonic Dystrophy Type 2Observational Trial in Myotonic Dystrophy Type 2 to Define Specific Clinical Outcome MeasuresMyotonic Dystrophy Type 2Diagnostic Test: DM1-ActivC;Diagnostic Test: R-PAct;Diagnostic Test: Beck depression inventory;Diagnostic Test: McGill pain questionnaire;Diagnostic Test: Brief Pain Inventory Short-Form;Diagnostic Test: Fatigue and Daytime Sleepiness Scale;Diagnostic Test: Myotonia Behaviour scale;Diagnostic Test: Hand opening time;Diagnostic Test: Pressure pain threshold;Diagnostic Test: Manual muscle testing;Diagnostic Test: Quantitative muscle testing;Diagnostic Test: Scale for Assessment and Rating of Ataxia;Diagnostic Test: Berg balance scale;Diagnostic Test: Quick motor function test;Diagnostic Test: GSGC;Diagnostic Test: 30 seconds sit to stand test;Diagnostic Test: Functional Index-2;Diagnostic Test: Six minute walking test;Diagnostic Test: Myotonia (from Individualised Neuromuscular Quality of Life Questionnaire)Prof. Dr. Benedikt SchoserNULLCompleted18 Years90 YearsAll60Germany
185EUCTR2017-002686-21-NL
(EUCTR)
26/06/201819/06/2018A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular DystrophyA Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Wave Life Sciences Ltd.NULLNot RecruitingFemale: no
Male: yes
32Phase 1France;United States;Czech Republic;Belgium;Spain;Ireland;Denmark;Netherlands;Italy;United Kingdom
186NCT03439670
(ClinicalTrials.gov)
June 19, 20189/1/2018A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Vamorolone;Drug: Prednisone;Other: PlaceboReveraGen BioPharma, Inc.European Union;Cooperative International Neuromuscular Research Group;Newcastle University;University of PittsburghCompleted4 Years7 YearsMale121Phase 2United States;Australia;Belgium;Canada;Czechia;Greece;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy
187EUCTR2017-004139-35-BE
(EUCTR)
18/06/201803/04/2018Clinical Trial in Male Subjects with Muscular DiseaseA Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy Duchenne Muscular Distrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Cosyntropin acetate
Product Code: MNK-1411
INN or Proposed INN: TETRACOSACTIDE HEXAACETATE
Other descriptive name: Cosyntropin acetate
Mallinckrodt ARD Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
132Phase 2United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Germany;Netherlands
188EUCTR2017-004279-30-BE
(EUCTR)
14/06/201804/05/2018A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study.A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
189EUCTR2017-004139-35-ES
(EUCTR)
12/06/201819/03/2018Clinical Trial in Male Subjects with Muscular DiseaseA Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy Duchenne Muscular Distrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Cosyntropin acetate
Product Code: MNK-1411
INN or Proposed INN: TETRACOSACTIDE HEXAACETATE
Other descriptive name: Cosyntropin acetate
Mallinckrodt ARD Inc.NULLNot Recruiting Female: no
Male: yes
132 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Bulgaria;Germany;Netherlands
190NCT03354039
(ClinicalTrials.gov)
June 12, 201810/10/2017Tamoxifen in Duchenne Muscular DystrophyTamoxifen in Duchenne Muscular Dystrophy: A Multicenter, Randomised, Double-blind, Placebo-controlled, Phase 3 Safety and Efficacy 48-week TrialDuchenne Muscular DystrophyDrug: Tamoxifen;Drug: Matching placeboUniversity Hospital, Basel, SwitzerlandNULLActive, not recruiting78 Months16 YearsMale93Phase 3France;Germany;Netherlands;Spain;Switzerland;United Kingdom
191NCT03424460
(ClinicalTrials.gov)
June 11, 20187/12/2017Venous Thromboembolism in Myotonic Dystrophy Type 1Venous Thromboembolism in Myotonic Dystrophy Type 1Myotonic Dystrophy 1Biological: Haemostasis tests;Biological: Monocytes and megacaryocytes culture and RNA extraction;Genetic: RNA extractionAssistance Publique - Hôpitaux de ParisAFM-Téléthon (Funding);Recherche Clinique Paris Descartes Necker Cochin Sainte AnneRecruiting18 YearsN/AAll130N/AFrance
192EUCTR2017-002686-21-BE
(EUCTR)
28/05/201812/03/2018A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular DystrophyA Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Wave Life Sciences Ltd.NULLNot Recruiting Female: no
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Canada;Belgium;Netherlands;Italy;United Kingdom
193EUCTR2017-002686-21-IT
(EUCTR)
25/05/201805/11/2020A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular DystrophyA Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy - - Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: [WVE-210201]
INN or Proposed INN: WVE-210201
WAVE LIFE SCIENCES USA INCNULLNot RecruitingFemale: no
Male: yes
32Phase 1United States;France;Canada;Belgium;Netherlands;United Kingdom;Italy
194EUCTR2016-003257-15-ES
(EUCTR)
12/04/201804/01/2018A Phase 2 clinical trial that is randomized and controlled by a placebo (similar to the product under investigation but does not have any therapeutic effect) of ACE-083 in Patients with muscular dystrophy (on face, around shoulder blades, in upper arms). The study is a double-blind study, this means that neither you nor your doctor will know if you receive study drug or placeboA Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy Facioscapulohumeral Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ACE-083
Product Code: ACE-083
INN or Proposed INN: pending
Other descriptive name: ACE-083
Acceleron Pharma Inc.NULLNot Recruiting Female: yes
Male: yes
92 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Spain
195EUCTR2017-002686-21-GB
(EUCTR)
11/04/201827/12/2017A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular DystrophyA Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: WVE-210201
Product Code: WVE-210201
INN or Proposed INN: WVE-210201
Wave Life Sciences Ltd.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Canada;Belgium;Netherlands;Italy;United Kingdom
196NCT03406780
(ClinicalTrials.gov)
March 4, 201815/1/2018A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular DystrophyA Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular DystrophyMuscular Dystrophies;Muscular Dystrophy, Duchenne;Muscular Disorders, Atrophic;Muscular Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornBiological: CAP-1002;Drug: PlaceboCapricor Inc.NULLCompleted10 YearsN/AMale18Phase 2United States
197NCT03375255
(ClinicalTrials.gov)
February 5, 201812/12/2017A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping TreatmentMuscular Dystrophy, DuchenneDrug: SRP-5051Sarepta Therapeutics, Inc.NULLCompleted12 YearsN/AMale15Phase 1United States;Canada
198NCT03508947
(ClinicalTrials.gov)
January 24, 201816/4/2018Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular DystrophyA Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: WVE-210201;Drug: PlaceboWave Life Sciences Ltd.NULLCompleted5 Years18 YearsMale36Phase 1United States;Belgium;Canada;France;Italy;Netherlands;United Kingdom
199EUCTR2017-001223-49-BG
(EUCTR)
16/01/201811/10/2017A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular DystrophyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension Nonsense Mutation Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Translarna 125 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ATALUREN
PTC Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
340Phase 3Korea, Republic of;Japan;China;Bulgaria;Australia;Poland;Malaysia;Brazil;Argentina;Jordan;Canada;Mexico;India;Russian Federation;Turkey;Thailand;Hong Kong;Taiwan;United States
200EUCTR2017-003568-10-GB
(EUCTR)
05/01/201817/05/2018An Extension Study to Assess the Long-term Safety and Efficacy ofVamorolone in Boys With Duchenne Muscular Dystrophy(DMD)A 24-month Phase II Open-label, Multicenter Long-term Extension Study toAssess the Long-term Safety and Efficacy of Vamorolone in Boys withDuchenne Muscular Dystrophy (DMD) - VBP15-LTE Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
INN or Proposed INN: Vamorolone
Other descriptive name: EV substance code: SUB188638
Product Name: Vamorolone
INN or Proposed INN: Vamorolone
Other descriptive name: EV substance code: SUB188638
Product Name: Vamorolone
INN or Proposed INN: Vamorolone
Other descriptive name: EV substance code: SUB188638
Product Name: Vamorolone
INN or Proposed INN: Vamorolone
Other descriptive name: EV substance code: SUB188638
ReveraGen BioPharma Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
201NCT03123913
(ClinicalTrials.gov)
December 18, 201712/4/2017Study of Testosterone and rHGH in FSHDStudy of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept StudyFacioscapulohumeral Muscular DystrophyDrug: Testosterone Enanthate;Drug: SomatropinUniversity of RochesterNULLActive, not recruiting18 Years65 YearsMale20Phase 1United States
202NCT03238235
(ClinicalTrials.gov)
December 12, 201712/7/2017Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular DystrophyA Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD)Becker Muscular DystrophyDrug: givinostat;Drug: placeboItalfarmacoNULLActive, not recruiting18 Years65 YearsMale51Phase 2Italy;Netherlands
203EUCTR2017-003568-10-SE
(EUCTR)
23/11/201705/10/2017An Extension Study to Assess the Long-term Safety and Efficacy ofVamorolone in Boys With Duchenne Muscular Dystrophy(DMD)A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamorolone
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
INN or Proposed INN: VAMOROLONE
Product Name: Vamorolone
INN or Proposed INN: VAMOROLONE
ReveraGen BioPharma Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
204EUCTR2016-001654-18-DE
(EUCTR)
13/11/201723/06/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyA randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: no
Male: yes
159Phase 3United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
205EUCTR2016-001654-18-BE
(EUCTR)
10/11/201726/06/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular DystrophyA randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 (BMS-986089) in ambulatory boys with Duchenne Muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMS-986089-01 (RO7239361) Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: RO7239361 (BMS-986089)
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01 (RO7239361) , anti-myostatin
Product Name: BMS-986089-01 (RO7239361) Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: RO7239361 (BMS-986089)
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01 (RO7239361), anti-myostatin
Product Name: BMS-986089-01 (RO7239361) Injections, 35 mg/Syringe (50 mg/mL)
Product Code: RO7239361 (BMS-986089)
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01 (RO7239361), anti-myostatin
Product Name: BMS-986089-01 (RO7239361) Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: RO7239361 (BMS-986089)
INN or Proposed INN: Anti-myostati
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: no
Male: yes
159Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
206EUCTR2016-001654-18-FR
(EUCTR)
10/11/201730/10/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular DystrophyA Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Bristol-Myers Squibb International CorporationNULLNot RecruitingFemale: no
Male: yes
160Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
207NCT03333590
(ClinicalTrials.gov)
November 6, 20171/11/2017Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular DystrophyPhase I/IIa Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2Duchenne Muscular DystrophyBiological: rAAVrh74.MCK.GALGT2Kevin FlaniganNULLActive, not recruiting4 YearsN/AMale6Phase 1/Phase 2United States
208NCT02653833
(ClinicalTrials.gov)
November 1, 201714/12/2015The Study of Skeletal Muscle Blood Flow in Becker Muscular DystrophySkeletal Muscle Blood Flow in Becker Muscular DystrophyMuscular DystrophyDrug: Tadalafil 20 MG;Other: beetroot juice extractCedars-Sinai Medical CenterNULLTerminated18 Years45 YearsMale6Early Phase 1United States
209EUCTR2016-001654-18-GB
(EUCTR)
01/11/201727/06/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyA Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, Anti-myostatin
Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: no
Male: yes
159Phase 3United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
210EUCTR2016-001654-18-NL
(EUCTR)
26/10/201712/07/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyA Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, Anti-myostatin
Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: no
Male: yes
159Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Netherlands;Germany;Japan;Sweden
211NCT03373968
(ClinicalTrials.gov)
October 24, 20174/12/2017Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability StudyOpen Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT StudiesDuchenne Muscular DystrophyDrug: GivinostatItalfarmacoCromsourceEnrolling by invitation7 YearsN/AMale206Phase 2/Phase 3United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;United Kingdom
212EUCTR2016-001654-18-IT
(EUCTR)
19/09/201707/02/2018A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy - - A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy - - Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATIONNULLNot RecruitingFemale: no
Male: yes
160Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
213EUCTR2015-002069-52-CZ
(EUCTR)
11/09/201714/03/2017A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
214EUCTR2017-001629-41-IT
(EUCTR)
08/09/201715/01/2021Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy.A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy - NA Becker Muscular Dystrophy (BMD)
MedDRA version: 20.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.P.A.NULLNot RecruitingFemale: no
Male: yes
55Phase 2Netherlands;Italy
215EUCTR2016-001654-18-ES
(EUCTR)
04/09/201704/07/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular DystrophyA Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: BMS-986089
INN or Proposed INN: Anti-Myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Bristol-Myers Squibb International CorporationNULLNot Recruiting Female: no
Male: yes
160 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
216NCT03218995
(ClinicalTrials.gov)
August 16, 20179/7/2017Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingAn Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingDuchenne Muscular DystrophyDrug: EteplirsenSarepta Therapeutics, Inc.NULLCompleted6 Months48 MonthsMale15Phase 2Belgium;France;Italy;United Kingdom;Germany
217EUCTR2016-001654-18-SE
(EUCTR)
12/08/201715/06/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyA randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, Anti-myostatin
Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL)
Product Code: RO7239361
INN or Proposed INN: Anti-myostatin Adnectin
Other descriptive name: BMS-986089-01, anti-myostatin
F. Hoffmann-La Roche LtdNULLNot RecruitingFemale: no
Male: yes
159Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
218EUCTR2017-000397-10-IT
(EUCTR)
31/07/201730/01/2018Studio nel quale tutti i pazienti prendono lo stesso farmaco sperimentale con lo scopo di valutare a lungo termine la sicurezza, la tollerabilità e l'efficacia del GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in uno studio con GIVINOSTAT.Studio in aperto a lungo termine volto a valutare la sicurezza, la tollerabilità e l'efficacia di GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in studio con GIVINOSTAT. - Studio con Givinostat in DMD a lungo termine Distrofia Muscolare di Duchenne (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (idrocloruro monoidrato)
Product Code: ITF2357
INN or Proposed INN: Givinostat (idrocloruro monoidrato)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
100 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy
219EUCTR2016-000951-29-DE
(EUCTR)
13/07/201713/04/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
12 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Belgium;Germany;Italy;United Kingdom
220NCT03179631
(ClinicalTrials.gov)
July 6, 20171/6/2017Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyA Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label ExtensionMuscular Diseases;Musculoskeletal Disease;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn;Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, AtrophicDrug: PLACEBO;Drug: AtalurenPTC TherapeuticsNULLActive, not recruiting5 YearsN/AMale360Phase 3United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Taiwan;Thailand;Turkey;Argentina;Chile;Jordan;Sri Lanka
221NCT03039686
(ClinicalTrials.gov)
July 6, 201727/1/2017Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyA Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: RO7239361;Drug: Placebo for RO7239361Hoffmann-La RocheNULLTerminated6 Years11 YearsMale166Phase 2/Phase 3United States;Argentina;Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;Sweden;United Kingdom
222NCT03167255
(ClinicalTrials.gov)
July 6, 201722/5/2017Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: NS-065/NCNP-01NS Pharma, Inc.Nippon Shinyaku Co., Ltd.;Cooperative International Neuromuscular Research Group;Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)Completed4 Years10 YearsMale16Phase 2United States;Canada
223EUCTR2016-000401-36-NL
(EUCTR)
28/06/201714/12/2016Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
169Phase 3France;United States;Serbia;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy
224EUCTR2015-002069-52-SE
(EUCTR)
26/06/201720/09/2016A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
222Phase 3United States;Serbia;Portugal;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Chile;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
225EUCTR2016-000951-29-GB
(EUCTR)
22/06/201704/04/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;Germany;Italy;United Kingdom
226NCT04226924
(ClinicalTrials.gov)
June 15, 201715/7/2019Treatment of Oculopharyngeal Muscular Dystrophy With TrehaloseA Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment ofOculopharyngeal Muscular DystrophyDrug: TrehaloseBioblast Pharma Ltd.NULLWithdrawn50 Years70 YearsAll0Phase 2Canada
227EUCTR2016-000951-29-IT
(EUCTR)
13/06/201707/02/2018A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping - N/A Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
SAREPTA THERAPEUTICS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
12Phase 2France;Belgium;Germany;United Kingdom;Italy
228EUCTR2016-000951-29-FR
(EUCTR)
01/06/201717/07/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
12Phase 1France;Belgium;Germany;Italy;United Kingdom
229NCT02851797
(ClinicalTrials.gov)
June 1, 201727/7/2016Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular DystrophyRandomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: givinostat;Drug: placeboItalfarmacoSyneos HealthActive, not recruiting6 Years17 YearsMale179Phase 3United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;United Kingdom
230EUCTR2016-000951-29-BE
(EUCTR)
29/05/201711/04/2017A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patientsAn Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Eteplirsen
Product Code: AVI-4658
INN or Proposed INN: eteplirsen
Other descriptive name: ETEPLIRSEN
Sarepta Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
15Phase 2France;Belgium;Germany;Italy;United Kingdom
231EUCTR2016-000401-36-IT
(EUCTR)
24/05/201708/02/2017Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
192Phase 3France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
232EUCTR2016-000401-36-DE
(EUCTR)
15/05/201728/10/2016Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLNot RecruitingFemale: no
Male: yes
169Phase 3United States;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
233EUCTR2015-002069-52-ES
(EUCTR)
11/05/201710/03/2017A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3United Kingdom;Germany;Israel;Spain;Canada;United States
234EUCTR2015-002069-52-BE
(EUCTR)
28/04/201705/09/2016A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Denmark;Australia;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Georgia;Norway;Germany;Sweden
235EUCTR2016-000602-10-NL
(EUCTR)
26/04/201720/09/2016A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden
236EUCTR2016-000401-36-GB
(EUCTR)
07/03/201726/10/2016Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
169Phase 3United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom
237EUCTR2016-004462-26-GB
(EUCTR)
16/02/201723/12/2016 A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy (DMD)
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
ReveraGen BioPharma, Inc.NULLNot Recruiting Female: no
Male: yes
48 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Australia;Israel;United Kingdom
238EUCTR2016-004262-26-GB
(EUCTR)
16/02/201721/12/2020A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD)VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability,Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne MuscularDystrophy (DMD) Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
ReveraGen BioPharma, Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
239EUCTR2016-004262-26-SE
(EUCTR)
13/02/201716/12/2016A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
INN or Proposed INN: vamorolone
Product Name: Vamorolone
INN or Proposed INN: vamorolone
Product Name: Vamorolone
INN or Proposed INN: vamorolone
Product Name: Vamorolone
INN or Proposed INN: vamorolone
ReveraGen BioPharma Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
240EUCTR2016-004263-38-SE
(EUCTR)
13/02/201716/12/2016An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD)A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
INN or Proposed INN: vamorolone
Product Name: Vamorolone
INN or Proposed INN: vamorolone
Product Name: Vamorolone
INN or Proposed INN: vamorolone
Product Name: Vamorolone
INN or Proposed INN: vamorolone
ReveraGen BioPharma Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
241EUCTR2016-004263-38-GB
(EUCTR)
10/02/201708/02/2019 An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) A Phase II Open-label, Multicenter Extension Study to Assess the Longterm Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
Product Name: Vamorolone
Product Code: VBP15
INN or Proposed INN: vamorolone
ReveraGen BioPharma, Inc.NULLNot Recruiting Female: no
Male: yes
48 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Australia;Israel;United Kingdom;Sweden
242EUCTR2016-000401-36-ES
(EUCTR)
10/02/201710/02/2017Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
192Phase 3France;United States;Canada;Belgium;Spain;Israel;Netherlands;Germany;Italy;United Kingdom
243EUCTR2015-002069-52-DE
(EUCTR)
07/02/201724/10/2016A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Casimersen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Golodirsen
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden
244EUCTR2016-001615-21-IT
(EUCTR)
06/02/201709/01/2017An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 yearsA Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Pfizer Inc. 235 East 42nd Street, New York, NY 10017NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
95Phase 2United States;Canada;Poland;Australia;Bulgaria;Japan;United Kingdom;Italy
245NCT03038399
(ClinicalTrials.gov)
February 2, 201730/1/2017Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Vamorolone 0.25 mg/day/day;Drug: Vamorolone 0.75 mg/day/day;Drug: Vamorolone 2.0 mg/day/day;Drug: Vamorolone 6.0 mg/day/dayReveraGen BioPharma, Inc.University of Pittsburgh;Cooperative International Neuromuscular Research GroupCompleted4 Years7 YearsMale46Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
246EUCTR2016-000401-36-BE
(EUCTR)
27/01/201727/10/2016Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy.Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (hydrochloride monohydrate)
Product Code: ITF2357
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
Other descriptive name: GIVINOSTAT
ITALFARMACO S.p.A.NULLNot RecruitingFemale: no
Male: yes
242Phase 3United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy
247EUCTR2015-002069-52-GB
(EUCTR)
23/01/201705/09/2016A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not yet available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
248EUCTR2015-002530-50-IT
(EUCTR)
19/01/201722/08/2018A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of rimeporide in patients with Duchenne Muscular Dystrophy (DMD)A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of rimeporide in patients with Duchenne Muscular Dystrophy (DMD) - RIM4DMD Distrofia Muscolare di Duchenne (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: rimeporide
Product Code: EMD 87580
ESPERARE FOUNDATIONNULLNot Recruiting Female: no
Male: yes
20 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Spain;United Kingdom;Italy
249EUCTR2015-002069-52-IT
(EUCTR)
19/01/201707/02/2018A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4045
Product Code: SRP-4045
Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 45
Product Name: SRP-4053
Product Code: SRP-4053
Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 53
SAREPTA THERAPEUTICS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
99Phase 3France;Czech Republic;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden
250EUCTR2014-002072-92-BG
(EUCTR)
29/12/201618/10/2016 A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Pfizer Inc. 235 East 42nd Street, New York, NY 10017NULLNot Recruiting Female: no
Male: yes
105 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom
251EUCTR2016-000602-10-GB
(EUCTR)
28/12/201623/09/2016A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden
252EUCTR2016-000602-10-AT
(EUCTR)
21/12/201601/12/2016A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
253EUCTR2016-000602-10-ES
(EUCTR)
20/12/201618/11/2016A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
254EUCTR2016-001615-21-GB
(EUCTR)
12/12/201621/10/2016An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 yearsA Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Pfizer Inc.NULLNot RecruitingFemale: no
Male: yes
105Phase 2United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom
255NCT02740972
(ClinicalTrials.gov)
December 201623/3/2016Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: NS-065/NCNP-01;Drug: PlaceboNS Pharma, Inc.Nippon Shinyaku Co., Ltd.;Cooperative International Neuromuscular Research Group;Therapeutic Research in Neuromuscular Disorders SolutionsCompleted4 Years9 YearsMale16Phase 2United States;Canada
256EUCTR2016-000602-10-FR
(EUCTR)
25/11/201612/01/2017A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
257EUCTR2016-000602-10-DE
(EUCTR)
17/11/201630/05/2016A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
258NCT02964377
(ClinicalTrials.gov)
November 20167/11/2016Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory AdolescentsA Single Center Dose Ranging Pilot Study of (+)-Epicatechin in Non-ambulatory Adolescents With Duchenne Muscular Dystrophy and Pre-symptomatic Cardiac DysfunctionDuchenne Muscular DystrophyDrug: (+)- EpicatechinCraig McDonald, MDCardero Therapeutics, Inc.Completed8 Years17 YearsMale15Phase 1/Phase 2United States
259NCT03236662
(ClinicalTrials.gov)
November 20167/11/2016(-)- Epicatechin Becker Muscular DystrophyUCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular DystrophyBecker Muscular DystrophyDrug: (-)-EpicatechinCraig McDonald, MDCardero Therapeutics, Inc.Completed18 Years70 YearsMale2Phase 2United States
260NCT02927080
(ClinicalTrials.gov)
November 20165/10/2016Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Facioscapulohumeral Muscular DystrophyFacioscapulohumeral Muscular DystrophyDrug: ACE-083;Drug: ACE-083 or placeboAcceleron Pharma, Inc.NULLTerminated18 YearsN/AAll95Phase 2United States;Canada;Spain
261NCT02835079
(ClinicalTrials.gov)
November 201622/6/2016Treatment Effect of Tamoxifen on Patients With DMDTreatment Effect of Tamoxifen on Patients With DMDDuchenne Muscular DystrophyDrug: TamoxifenHadassah Medical OrganizationNULLUnknown status5 Years16 YearsMale19Phase 1NULL
262EUCTR2016-000602-10-IT
(EUCTR)
20/10/201606/02/2018A clinical study to assess how effective and safe is idebenone treatment inpatients with Duchenne Muscular Dystrophy (DMD) who are currentlyreceiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Studyassessing the Efficacy, Safety and Tolerability of Idebenone inPatients with Duchenne Muscular Dystrophy Receiving GlucocorticoidSteroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
SANTHERA PHARMACEUTICALSNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
266Phase 3France;United States;Belgium;Spain;Austria;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden
263EUCTR2013-005489-20-FR
(EUCTR)
18/10/201618/06/2015Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
264NCT02907619
(ClinicalTrials.gov)
October 13, 201614/9/2016An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular DystrophyA MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHYDuchenne Muscular DystrophyBiological: PF-06252616PfizerNULLTerminated6 Years18 YearsMale59Phase 2United States;Canada;Italy;Japan;United Kingdom
265NCT02500381
(ClinicalTrials.gov)
September 28, 201614/7/2015Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD)A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: SRP-4045;Drug: SRP-4053;Drug: PlaceboSarepta Therapeutics, Inc.NULLRecruiting6 Years13 YearsMale222Phase 3United States;Australia;Belgium;Bulgaria;Canada;Czechia;Denmark;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Mexico;Poland;Russian Federation;Serbia;Spain;Sweden;United Kingdom
266EUCTR2016-000602-10-BE
(EUCTR)
02/09/201619/07/2016A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS Duchenne Muscular Dystrophy (DMD)
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Raxone
Product Name: Idebenone
INN or Proposed INN: IDEBENONE
Other descriptive name: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
267NCT02814019
(ClinicalTrials.gov)
September 201617/6/2016A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid SteroidsA Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid SteroidsDuchenne Muscular Dystrophy (DMD)Drug: Idebenone 150 mg film-coated tablets;Drug: placeboSanthera PharmaceuticalsNULLTerminated10 YearsN/AMale255Phase 3United States;Austria;Belgium;Bulgaria;France;Germany;Hungary;Ireland;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom
268EUCTR2014-002072-92-PL
(EUCTR)
10/08/201614/07/2016 A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Pfizer Inc. 235 East 42nd Street, New York, NY 10017NULLNot Recruiting Female: no
Male: yes
120 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom
269NCT02760277
(ClinicalTrials.gov)
July 28, 201628/4/2016An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Vamorolone 0.25 mg/day/day;Drug: Vamorolone 0.75 mg/day/day;Drug: Vamorolone 2.0 mg/day/day;Drug: Vamorolone 6.0 mg/day/dayReveraGen BioPharma, Inc.University of Pittsburgh;National Institute of Neurological Disorders and Stroke (NINDS);Cooperative International Neuromuscular Research Group;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)Completed4 Years7 YearsMale48Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
270EUCTR2016-000601-36-IT
(EUCTR)
26/07/201608/06/2021Clinical trial on the efficacy of non invasive ventilation and modafinil on excessive daytime sleepiness in Myotonic Dystrophy type 1CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: AMULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1 - ModaNIV Myotonic Dystrophy type 1
MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: PROVIGIL - 100 MG COMPRESSE 30 COMPRESSEFONDAZIONE SERENA ONLUSNULLNot RecruitingFemale: yes
Male: yes
80Phase 3Italy
271NCT02858908
(ClinicalTrials.gov)
July 20, 20164/8/2016Study of Tideglusib in Adolescent and Adult Patients With Myotonic DystrophyA Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic DystrophyMyotonic Dystrophy 1Drug: TideglusibAMO Pharma LimitedNULLCompleted12 Years45 YearsAll16Phase 2United Kingdom
272NCT02836418
(ClinicalTrials.gov)
July 12, 201630/6/2016Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular DystrophyAn Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular DystrophyFacioscapulohumeral Muscular Dystrophy;Limb Girdle Muscular DystrophyDrug: ATYR1940aTyr Pharma, Inc.NULLCompleted16 Years25 YearsAll8Phase 1/Phase 2United States;Denmark;Italy
273EUCTR2014-002008-25-FR
(EUCTR)
06/07/201611/01/2017A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not Available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
48 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Italy;United Kingdom
274EUCTR2016-000624-25-DK
(EUCTR)
22/06/201619/04/2016An Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular DystrophyAn Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
MedDRA version: 19.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ATYR1940
Product Code: ATYR1940
INN or Proposed INN: Not available yet
Other descriptive name: ATYR1940
aTyr Pharma, Inc.NULLNot RecruitingFemale: yes
Male: yes
22Phase 1;Phase 2United States;Denmark;Italy
275NCT02819557
(ClinicalTrials.gov)
June 9, 201616/6/2016Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular DystrophyA Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation DystrophinopathyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLCompleted2 Years5 YearsMale14Phase 2United States
276NCT02760264
(ClinicalTrials.gov)
June 201628/4/2016A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)Duchenne Muscular DystrophyDrug: Vamorolone 0.25 mg/kg/day;Drug: Vamorolone 0.75 mg/kg/day;Drug: Vamorolone 2.0 mg/kg/day;Drug: Vamorolone 6.0 mg/kg/dayReveraGen BioPharma, Inc.University of Pittsburgh;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);National Institute of Neurological Disorders and Stroke (NINDS);Cooperative International Neuromuscular Research GroupCompleted4 Years6 YearsMale48Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
277NCT02858362
(ClinicalTrials.gov)
June 201627/7/2016Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)Phaseout DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With Ezutromid in Ambulatory Paediatric Male Subjects With Duchenne Muscular Dystrophy (SMT C11005)Duchenne Muscular DystrophyDrug: EzutromidSummit TherapeuticsNULLTerminated5 YearsN/AMale43Phase 2United States;United Kingdom
278NCT02808585
(ClinicalTrials.gov)
June 20168/6/2016Study to Assess the Safety, Tolerability and PK Response and Explore the PD Response Following 4 Weekly SC Injections of PB1046 in Subjects With Stable Heart Failure With Reduced Ejection Fraction (HFrEF)Phase 2a, Randomized, Double-blind, Placebo-controlled, Multiple-dose, 2-Part Study to Assess the Safety, Tolerability and Pharmacokinetic Response and Explore the Pharmacodynamic Response Following 4 Weeks of Once Weekly Subcutaneous Injections of PB1046 in Adult Subjects With Stable Heart Failure With Reduced Ejection Fraction (HFrEF) (Part 1) and in Subjects With Cardiac Dysfunction Secondary to Duchenne Muscular Dystrophy (Part 2)Heart FailureDrug: PB1046 Injection;Drug: Placebo InjectionPhaseBio Pharmaceuticals Inc.NULLCompleted18 YearsN/AAll29Phase 2United States
279EUCTR2014-005296-81-BE
(EUCTR)
02/05/201619/11/2015A clinical study to provide drisapersen (study medication) to patients withDuchenne disease (muscular disease) and to assess the efficacy, safety and tolerability.A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: Drisapersen
Other descriptive name: DRISAPERSEN SODIUM
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
104Phase 3United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden
280NCT02752048
(ClinicalTrials.gov)
May 20166/4/2016A Phase IIa Study of TAS-205 for Duchenne Muscular DystrophyA Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: TAS-205;Drug: PlaceboTaiho Pharmaceutical Co., Ltd.NULLCompleted5 YearsN/AMale36Phase 2Japan
281EUCTR2016-000067-16-GB
(EUCTR)
20/04/201613/05/2016A clinical trial to investigate how safe and effective tideglusib is, as treatment for adolescents and adults with myotonic dystrophy diagnosed before they were 12 years old.A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy
MedDRA version: 19.1;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Tideglusib
INN or Proposed INN: Tideglusib
Other descriptive name: 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione
AMO Pharma Ltd.NULLNot RecruitingFemale: yes
Male: yes
16Phase 2United Kingdom
282NCT02958202
(ClinicalTrials.gov)
April 201627/10/2016Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kgBioMarin PharmaceuticalNULLTerminated5 YearsN/AMale7Phase 2Belgium;Italy;Netherlands;Sweden
283NCT02704325
(ClinicalTrials.gov)
April 201623/12/2015Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2Duchenne Muscular DystrophyBiological: rAAVrh74.MCK.GALGT2;Other: PLACEBO (Saline)Kevin FlaniganNULLWithdrawn9 YearsN/AMale0Phase 1/Phase 2United States
284NCT02439216
(ClinicalTrials.gov)
April 201629/4/2015Phase 1/2 Study in Boys With Duchenne Muscular DystrophyA Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Edasalonexent;Drug: PlaceboCatabasis PharmaceuticalsNULLCompleted4 Years7 YearsMale31Phase 1/Phase 2United States
285NCT02603562
(ClinicalTrials.gov)
March 30, 20165/11/2015Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular DystrophyAn Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular DystrophyFacioscapulohumeral Muscular Dystrophy (FSHD)Biological: ATYR1940;Biological: PlaceboaTyr Pharma, Inc.NULLCompleted12 Years25 YearsAll8Phase 1/Phase 2United States;France;Italy
286NCT02710591
(ClinicalTrials.gov)
March 201626/1/2016Rimeporide in Patients With Duchenne Muscular DystrophyA Phase Ib, Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of Rimeporide in Patients With Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: RimeporideEspeRare FoundationNULLCompleted6 Years14 YearsMale20Phase 1France;Italy;Spain;United Kingdom
287EUCTR2015-004333-27-GB
(EUCTR)
19/01/201604/11/2015 A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular Dystrophy A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD Duchenne Muscular Dystrophy
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: SMT C1100
INN or Proposed INN: Pending
Summit (Oxford) LimitedNULLNot Recruiting Female: no
Male: yes
40 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
288JPRN-UMIN000020580
2016/01/1520/01/2016Pilot study of tranilast for cardiomyopathy of muscular dystrophy muscular dystrophyTranilast 300mg/day for three months (oral intake)National Hospital Organization Toneyama National HospitalNational Cerebral and Cardiovascular CenterComplete: follow-up complete20years-oldNot applicableMale and Female2Not applicableJapan
289EUCTR2015-001955-54-DE
(EUCTR)
13/01/201618/08/2015A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM
Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan
290EUCTR2015-003681-87-BE
(EUCTR)
12/01/201616/11/2015A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study)A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 044
Product Code: BMN 044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Product Name: BMN 044
Product Code: BMN 044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
50Phase 2United States;Belgium;Netherlands;Italy;Sweden
291EUCTR2015-001955-54-BE
(EUCTR)
06/01/201621/09/2015A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan
292NCT02485938
(ClinicalTrials.gov)
January 201619/6/2015HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular DystrophyDuchenne Muscular Dystrophy;CardiomyopathyDrug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)Capricor Inc.NULLCompleted12 YearsN/AMale25Phase 1/Phase 2United States
293EUCTR2015-002530-50-FR
(EUCTR)
18/12/201511/09/2015Rimeporide in patients with Duchenne Muscular DystrophyA phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy. Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Rimeporide
Product Code: EMD 87580
INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate
Other descriptive name: Rimeporide
INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate
Other descriptive name: Rimeporide
EspeRareNULLNot RecruitingFemale: no
Male: yes
20Phase 1France;Spain;Italy;United Kingdom;Switzerland
294EUCTR2015-001910-88-DK
(EUCTR)
17/12/201530/06/2015A Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular DystrophiesAn Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
MedDRA version: 19.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ATYR1940
Product Code: ATYR1940
INN or Proposed INN: Not available yet
aTyr Pharma, Inc.NULLNot RecruitingFemale: yes
Male: yes
16Phase 1;Phase 2France;United States;Denmark;Italy
295EUCTR2015-001955-54-NL
(EUCTR)
08/12/201509/07/2015A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Drisapersen
Product Code: Drisapersen
BioMarin Nederland BVNULLNot RecruitingFemale: no
Male: yes
220Phase 3Portugal;United States;Belarus;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan;Sweden
296NCT02515669
(ClinicalTrials.gov)
December 2, 201529/7/2015Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMDA Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular DystrophyMuscular Dystrophy (DMD)Drug: RO7239361;Drug: PlaceboHoffmann-La RocheNULLTerminated5 Years10 YearsMale43Phase 1/Phase 2United States;Canada
297NCT02606136
(ClinicalTrials.gov)
November 30, 20154/11/2015Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: pamrevlumab (FG-3019)FibroGenNULLActive, not recruiting12 YearsN/AMale21Phase 2United States
298NCT02636686
(ClinicalTrials.gov)
November 27, 20159/12/2015Extension Study of Drisapersen in DMD SubjectsAn Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy.Duchenne Muscular DystrophyDrug: DrisapersenBioMarin PharmaceuticalNULLNo longer available5 Years80 YearsMaleUnited States;Argentina;Australia;Belgium;Bulgaria;Czechia;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom;Czech Republic
299EUCTR2015-001955-54-ES
(EUCTR)
24/11/201505/10/2015A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term.An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Drisapersen
Product Code: Drisapersen
INN or Proposed INN: drisapersen
Other descriptive name: DRISAPERSEN SODIUM
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Belarus;Portugal;Taiwan;Spain;Ukraine;Chile;Israel;Russian Federation;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Norway;Japan
300EUCTR2015-003195-68-GB
(EUCTR)
11/11/201526/10/2015Testosterone therapy in DMDObservational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy. - Testosterone therapy in Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Trade Name: Sustanon 250
Product Name: Sustanon 250
Product Code: PRD2175434
INN or Proposed INN: testosterone propionate
INN or Proposed INN: testosterone phenylpropionate
INN or Proposed INN: testosterone isocaproate
INN or Proposed INN: testosterone decanoate
The Newcastle upon Tyne NHS Hospitals Foundation TrustNULLNot RecruitingFemale: no
Male: yes
20Phase 3United Kingdom
301NCT02579239
(ClinicalTrials.gov)
November 5, 201528/9/2015The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular DystrophiesAn Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular DystrophiesLimb-Girdle Muscular Dystrophies;Facioscapulohumeral Muscular DystrophyBiological: ATYR1940;Biological: PlaceboaTyr Pharma, Inc.NULLCompleted18 Years75 YearsAll18Phase 1/Phase 2United States;Denmark;France;Italy
302EUCTR2015-002530-50-ES
(EUCTR)
04/11/201525/08/2015Rimeporide in patients with Duchenne Muscular DystrophyA phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy. - Rim4DMD Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Rimeporide
Product Code: EMD 87580
INN or Proposed INN: N(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate
Other descriptive name: Rimeporide
EspeRareNULLNot RecruitingFemale: no
Male: yes
20Phase 1France;Spain;Italy;United Kingdom;Switzerland
303NCT02571205
(ClinicalTrials.gov)
November 201524/8/2015Testosterone Therapy for Pubertal Delay in Duchenne Muscular DystrophyObservational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Sustanon (testosterone)Newcastle-upon-Tyne Hospitals NHS TrustNULLCompleted12 Years17 YearsMale15United Kingdom
304EUCTR2015-002530-50-GB
(EUCTR)
29/10/201507/09/2015Rimeporide in patients with Duchenne Muscular DystrophyA phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy - RIM4DMD Duchenne Muscular Dystrophy (DMD)
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Rimeporide
Product Code: EMD 87580
INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate
Other descriptive name: Rimeporide
INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate
Other descriptive name: Rimeporide
EspeRareNULLNot RecruitingFemale: no
Male: yes
20Phase 1France;Spain;Italy;Switzerland;United Kingdom
305NCT02530905
(ClinicalTrials.gov)
October 14, 201510/8/2015Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) PatientsA Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 SkippingDuchenne Muscular DystrophyDrug: SRP-4045;Drug: PlaceboSarepta Therapeutics, Inc.NULLCompleted7 Years21 YearsMale12Phase 1United States
306NCT02667483
(ClinicalTrials.gov)
October 201526/1/2016Study of DS-5141b in Patients With Duchenne Muscular DystrophyPhase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: DS-5141bDaiichi Sankyo Co., Ltd.Orphan Disease Treatment Institute Co., Ltd.Active, not recruiting5 Years10 YearsMale7Phase 1/Phase 2Japan
307NCT02531217
(ClinicalTrials.gov)
September 201525/6/2015Safety, Tolerability, PK, and Activity of ATYR1940 in Patients With Muscular Dystrophy - Study ExtensionAn Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients With Fascioscapulohumeral Muscular Dystrophy (FSHD)Facioscapulohumeral Muscular DystrophyBiological: ATYR1940aTyr Pharma, Inc.NULLCompleted18 Years65 YearsAll9Phase 1/Phase 2United States;Italy;Netherlands
308NCT03067831
(ClinicalTrials.gov)
September 201525/2/2017Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular DystrophySafety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy.Duchenne Muscular DystrophyBiological: Stem CellsStem Cells ArabiaNULLRecruiting4 Years25 YearsAll20Phase 1/Phase 2Jordan
309EUCTR2013-005489-20-BG
(EUCTR)
25/08/201526/06/2015Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
310EUCTR2015-001912-36-NL
(EUCTR)
04/08/201522/06/2015A Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Exposure of ATYR1940 in Adult Patients with Genetic MyopathyAn Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Facioscapulohumeral muscular dystrophy
MedDRA version: 19.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ATYR1940
Product Code: ATYR1940
INN or Proposed INN: Not available yet
Other descriptive name: ATYR1940
aTyr Pharma, Inc.NULLNot RecruitingFemale: yes
Male: yes
32Phase 1;Phase 2United States;Netherlands;Italy
311EUCTR2015-001967-38-GB
(EUCTR)
03/08/201520/07/2015A research project for boys with Duchenne muscular dystrophy aged 5-9 years. It aims to find out how safe and well tolerated different doses of the experimental medicine are, when given by mouth after breakfast and evening meal.A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD) - SMTC11004 - Phase 1 study in healthy volunteers and DMD patients Duchenne muscular dystrophy (DMD)
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SMT C1100 (F5; nanosuspension)
Product Code: SMT C1100 (F5)
Product Name: SMT C1100 (F6; Powder for Oral Suspension)
Product Code: SMT C1100 (F6)
Summit (Oxford) LimitedNULLNot Recruiting Female: no
Male: yes
24 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
312NCT02420379
(ClinicalTrials.gov)
June 30, 201510/4/2015Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: eteplirsenSarepta Therapeutics, Inc.NULLCompleted4 Years6 YearsMale33Phase 2United States
313NCT02434627
(ClinicalTrials.gov)
June 201528/4/2015Sodium Nitrate for Muscular DystrophySodium Nitrate for Muscular DystrophyBecker Muscular DystrophyDrug: Sodium NitrateCedars-Sinai Medical CenterNULLCompleted15 Years45 YearsMale5Phase 1United States
314NCT02484560
(ClinicalTrials.gov)
June 201516/6/2015Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2Duchenne Muscular DystrophyDrug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem CellUniversity of GaziantepIstinye University, Cukurova University, Yildirim Beyazit University, Gaziantep Deva Hospital, Gaziantep Public HospitalActive, not recruiting8 Years14 YearsMale10Phase 1Turkey
315NCT02525302
(ClinicalTrials.gov)
May 201518/7/2015HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02Duchenne Muscular DystrophyDrug: HT-100Akashi TherapeuticsNULLTerminated6 Years20 YearsMale10Phase 2United States
316EUCTR2013-003605-26-NL
(EUCTR)
13/04/201519/11/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Prosensa Therapeutics B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
317NCT02354352
(ClinicalTrials.gov)
March 20, 201527/1/2015Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular DystrophyTherapeutic Potential for Aldosterone Inhibition in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Eplerenone;Drug: SpironolactoneOhio State UniversityUniversity of California, Los Angeles;University of Utah;University of Colorado, Denver;University of Kansas Medical Center;Vanderbilt University Medical CenterCompleted7 YearsN/AMale52Phase 3United States
318EUCTR2014-002072-92-IT
(EUCTR)
05/03/201512/01/2015A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular DystrophyA Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: -
Other descriptive name: PF-06252616
Pfizer Inc. 235 East 42nd Street, New York, NY 10017NULLNot RecruitingFemale: no
Male: yes
105Phase 2United States;Canada;Poland;Bulgaria;United Kingdom;Japan;Italy
319NCT02376816
(ClinicalTrials.gov)
March 201526/2/2015Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular DystrophyPhase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.Micro-dystrophinDuchenne Muscular DystrophyBiological: rAAVrh74.MCK.micro-DystrophinJerry R. MendellEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Completed7 YearsN/AMale2Phase 1United States
320NCT01976091
(ClinicalTrials.gov)
February 201524/7/2013Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCAPhase I/IIa Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCALimb Girdle Muscular Dystrophy Type 2D (LGMD2D)Drug: scAAVrh74.tMCK.hSGCAJerry R. MendellNULLCompleted7 YearsN/AAll6Phase 1/Phase 2United States
321NCT02383511
(ClinicalTrials.gov)
February 20154/2/2015Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced DietA Phase 1b Placebo-controlled, Multi-centre, Randomized, Double-blind Dose Escalation Study to Evaluate the Pharmacokinetics (PK) and Safety of SMT C1100 in Patients With Duchenne Muscular Dystrophy (DMD) Who Follow a Balanced DietMuscular Dystrophy, DuchenneDrug: SMT C1100Summit TherapeuticsNULLCompleted5 Years13 YearsMale12Phase 1United Kingdom
322EUCTR2013-003605-26-BE
(EUCTR)
23/01/201504/12/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Prosensa Therapeutics B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
323NCT02310906
(ClinicalTrials.gov)
January 13, 20153/12/2014Phase I/II Study of SRP-4053 in DMD PatientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 SkippingDuchenne Muscular DystrophyDrug: Placebo;Drug: SRP-4053Sarepta Therapeutics, Inc.Institut de Myologie, France;Consultants for Research in Imaging and Spectroscopy;Great Ormond Street Hospital for Children NHS Foundation Trust;Catholic University of the Sacred Heart;Royal Holloway University;SYSNAV;University College, London;University of Newcastle Upon-TyneCompleted6 Years15 YearsMale39Phase 1/Phase 2United States;France;Italy;United Kingdom
324EUCTR2013-005489-20-CZ
(EUCTR)
08/01/201524/10/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
325NCT02354781
(ClinicalTrials.gov)
January 201526/1/2015Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular DystrophyPhase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: rAAV1.CMV.huFollistin344Jerry R. MendellDuchenne Alliance;Milo TherapeuticsCompleted7 YearsN/AMale3Phase 1/Phase 2United States
326NCT02328482
(ClinicalTrials.gov)
January 201525/12/2014Continuation Protocol to Protocol BBCO-001A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001Muscular Dystrophy, Oculopharyngeal (OPMD)Drug: Tehalose 30grBioblast Pharma Ltd.NULLCompleted18 Years80 YearsAll9Phase 3Canada
327JPRN-UMIN000016092
2014/12/3131/12/2014Analysis of the effects of animal fat free diet and aspirin for prostaglandins in patients with myotonic dystrophy myotonic dystrophy1st stage (1w): animal fat free diet
2nd stage (1w): animal fat free diet and aspirin (330mg/day)
National Hospital Organization Toneyama National HospitalGraduate school of engineering Osaka UniversityComplete: follow-up completeNot applicableNot applicableMale and Female10Not applicableJapan
328EUCTR2014-002072-92-GB
(EUCTR)
30/12/201416/09/2014A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular DystrophyA Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy Duchenne's Muscular Dystrophy
MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Product Code: PF-06252616
INN or Proposed INN: PF-06252616
Pfizer Inc. 235 East 42nd Street, New York, NY 10017NULLNot RecruitingFemale: no
Male: yes
105Phase 2United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom
329EUCTR2013-003605-26-IT
(EUCTR)
02/12/201411/09/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
Prosensa Therapeutics B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Italy;Sweden
330EUCTR2014-003100-78-GB
(EUCTR)
01/12/201419/09/2014A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet. SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet. Duchenne Muscular Dystrophy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: SMT C1100
INN or Proposed INN: Not available
Other descriptive name: SMT C1100
Summit (Oxford) LimitedNULLNot Recruiting Female: no
Male: yes
12 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
331NCT02312011
(ClinicalTrials.gov)
December 20144/12/2014A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients With Myotonic Dystrophy Type 1Myotonic Dystrophy Type 1Drug: IONIS-DMPKRx;Drug: PlaceboIonis Pharmaceuticals, Inc.NULLCompleted20 Years55 YearsAll48Phase 1/Phase 2United States
332NCT01805024
(ClinicalTrials.gov)
December 20144/3/2013Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)Congenital Muscular DystrophyDrug: OmigapilSanthera PharmaceuticalsNULLCompleted5 Years16 YearsAll20Phase 1United States
333NCT02329769
(ClinicalTrials.gov)
December 201422/12/2014Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kgBioMarin PharmaceuticalNULLTerminated9 Years20 YearsMale15Phase 2Belgium;Italy;Netherlands;Sweden
334NCT02295748
(ClinicalTrials.gov)
December 201418/11/2014An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability DeflazacortAn Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLCompleted4 YearsN/AMale24Phase 1United States
335NCT02251600
(ClinicalTrials.gov)
December 201422/9/2014A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular DystrophyA Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: DeflazacortPTC TherapeuticsNULLCompleted4 Years16 YearsMale24Phase 1United States
336EUCTR2013-003605-26-SE
(EUCTR)
26/11/201426/09/2014A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01)A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO044
Product Code: PRO044
INN or Proposed INN: PS188 (company code)
Other descriptive name: PS188
BioMarin Nederland B.V.NULLNot RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
337NCT02310763
(ClinicalTrials.gov)
November 24, 20144/11/2014A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular DystrophyA PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHYDuchenne Muscular DystrophyBiological: PF-06252616;Drug: PlaceboPfizerNULLTerminated6 Years15 YearsMale121Phase 2United States;Australia;Bulgaria;Canada;Italy;Japan;Poland;United Kingdom
338NCT02255552
(ClinicalTrials.gov)
November 17, 201425/9/2014Study of Eteplirsen in DMD PatientsAn Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: eteplirsenSarepta Therapeutics, Inc.NULLCompleted7 Years16 YearsMale109Phase 3United States
339EUCTR2014-002008-25-IT
(EUCTR)
13/11/201410/09/2014A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not Available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
48Phase 1;Phase 2France;United Kingdom;Italy
340NCT02286947
(ClinicalTrials.gov)
November 201430/10/2014Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular DystrophyAn Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: EteplirsenSarepta Therapeutics, Inc.NULLCompleted7 Years21 YearsMale24Phase 2United States
341NCT02036463
(ClinicalTrials.gov)
November 20146/1/2014A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular DystrophyCINRG0513: A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: Prednisone;Drug: PlaceboAnn & Robert H Lurie Children's Hospital of ChicagoChildren's Research InstituteWithdrawn3 Years6 YearsMale0Phase 2United States
342EUCTR2014-002210-23-DK
(EUCTR)
14/10/201402/06/2014Can local botulinum toxin improve swallowing difficulties in persons with msucle disease?Can local intramuscular botulinum toxin improve dysphagia in patients with myopathic dysphagia and constriction of the cricoid muscle? Oculopharyngesl muscle dystrophy, inclusion body myositis
MedDRA version: 18.1;Level: LLT;Classification code 10019897;Term: Hereditary progressive muscular dystrophy;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Xeomin
Product Name: Xeomin
Other descriptive name: BOTULINUM TOXIN TYPE A
RigshospitaletNULLNot Recruiting Female: yes
Male: yes
15 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noDenmark
343EUCTR2013-004427-37-IT
(EUCTR)
01/10/201404/09/2014Efficacy and tolerability of ibuprofen and isosorbide dinitrate (20 mg, 40 mg, 60 mg, 80 mg)Multicentre, randomised, double-blind, dose titration design in patients with Duchenne muscular dystrophy to evaluate the efficacy and the tolerability of the combinations of Ibuprofen (200 mg bid) and Isosorbide Dinitrate (20 mg, 40 mg, 60 mg, 80 mg) - ESPP001 Restrictive respiratory syndrome with frequent pulmonary infections and cardiomyopathy.wasting of skeletal muscle, severe local inflammation and, at least initially, muscle regeneration.
MedDRA version: 17.0;Level: PT;Classification code 10064571;Term: Gene mutation;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Trade Name: Isosorbide Dinitrate 20 mg
Product Name: Isosorbide Dinitrate
INN or Proposed INN: ISOSORBIDE DINITRATE
Trade Name: IBUPROFEN 200MG
INN or Proposed INN: IBUPROFEN
Trade Name: Isosorbide Dinitrate 40 mg
Product Name: Isosorbide Dinitrate
INN or Proposed INN: ISOSORBIDE DINITRATE
Trade Name: Isosorbide Dinitrate 60 mg
Product Name: Isosorbide Dinitrate
INN or Proposed INN: ISOSORBIDE DINITRATE
Trade Name: Isosorbide Dinitrate 80 mg
Product Name: Isosorbide Dinitrate
INN or Proposed INN: ISOSORBIDE DINITRATE
PARENT PROJECT ONLUSNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
188Italy
344EUCTR2013-005489-20-DE
(EUCTR)
29/09/201428/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
345EUCTR2014-001753-17-NL
(EUCTR)
19/09/201403/06/2014A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic MyopathyA Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies Facioscapulohumeral muscular dystrophy
MedDRA version: 17.1;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ATYR1940
Product Code: ATYR1940
INN or Proposed INN: N/A
aTyr Pharma, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
44France;United States;Netherlands;Italy
346EUCTR2014-002008-25-GB
(EUCTR)
10/09/201408/09/2014A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsA 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4053
Product Code: SRP-4053
INN or Proposed INN: Not Available
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Sarepta Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
48 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Italy;United Kingdom
347EUCTR2013-005489-20-IT
(EUCTR)
10/09/201430/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
348EUCTR2013-005489-20-ES
(EUCTR)
01/09/201406/08/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
349NCT01834066
(ClinicalTrials.gov)
September 201426/2/2013Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy.Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical TrialMuscular Dystrophy;Duchenne Muscular Dystrophy,Biological: Stem CellChaitanya Hospital, PuneNULLRecruiting6 Years25 YearsBoth25Phase 1/Phase 2India
350NCT02235844
(ClinicalTrials.gov)
September 20148/9/2014Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)Duchenne's Muscular DystrophyBiological: Umbilical Cord Mesenchymal Stem CellsAllergy and Asthma Consultants, Wichita, KansasAidan Foundation;Neil H. Riordan PhDCompleted28 Years31 YearsMale1Phase 1United States
351NCT02246478
(ClinicalTrials.gov)
September 20149/9/2014A Study of TAS-205 for Duchenne Muscular DystrophyA Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: TAS-205;Drug: PlaceboTaiho Pharmaceutical Co., Ltd.NULLCompleted5 Years15 YearsMale21Phase 1Japan
352EUCTR2013-005489-20-BE
(EUCTR)
28/08/201411/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
353EUCTR2013-005489-20-SE
(EUCTR)
22/08/201411/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Sweden;Korea, Republic of
354EUCTR2013-005489-20-GB
(EUCTR)
22/08/201411/07/2014Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden
355NCT02239224
(ClinicalTrials.gov)
August 20147/9/2014Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients With Muscular DystrophyA Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defined Genetic Muscular DystrophiesFacioscapulohumeral Muscular Dystrophy (FSHD)Biological: Placebo;Biological: ATYR1940aTyr Pharma, Inc.NULLCompleted18 Years65 YearsAll20Phase 1/Phase 2United States;France;Italy;Netherlands
356NCT02251457
(ClinicalTrials.gov)
August 201425/9/2014Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1Myotonia Congenita;Paramyotonia Congenita;Myotonic Dystrophy 1Drug: RanolazineOhio State UniversityGilead SciencesCompleted18 Years100 YearsAll35Phase 1United States
357EUCTR2014-001753-17-IT
(EUCTR)
30/07/201413/06/2014A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic MyopathyA Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies Facioscapulohumeral muscular dystrophy
MedDRA version: 17.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ATYR1940
Product Code: ATYR1940
INN or Proposed INN: N/A
aTyr Pharma, Inc.NULLNot RecruitingFemale: yes
Male: yes
44Phase 1;Phase 2United States;France;Netherlands;Italy
358JPRN-UMIN000014836
2014/05/0112/08/2014A pilot study to evaluate efficacy and safety of resveratrol for muscular dystrophy. Duchenne muscular dystrophy, Becker muscular dystrophy, Fukuyama congenital muscular dystrophy500mg resveratrol is administrated daily for 8 weeks.
Subsequently, resveratrol is increased to 1500mg by 500mg every 8 weeks.
Sapporo Medical UniversityNULLComplete: follow-up complete12years-oldNot applicableMale and Female10Not selectedJapan
359NCT02196467
(ClinicalTrials.gov)
May 20149/5/2014Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) PatientsTransplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) PatientsDuchenne Muscular DystrophyBiological: Myoblast transplantation;Procedure: Saline injectionCHU de Quebec-Universite LavalNULLRecruiting16 YearsN/AMale10Phase 1/Phase 2Canada
360NCT02167217
(ClinicalTrials.gov)
April 17, 20143/2/2014Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular DystrophyPhase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PrednisoloneWashington University School of MedicineNationwide Children's Hospital;Feinberg School of Medicine, Northwestern University;University of Texas Southwestern Medical Center;University of California, Davis;Nemours Hospital, Orlando, FLCompleted1 Month30 MonthsMale25Phase 2United States
361NCT02090959
(ClinicalTrials.gov)
March 20, 201417/3/2014An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation DystrophinopathyA Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation DystrophinopathyMuscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornDrug: AtalurenPTC TherapeuticsNULLTerminated7 Years15 YearsMale219Phase 3United States;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic
362EUCTR2012-004527-20-PL
(EUCTR)
12/02/201427/11/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
363NCT02015481
(ClinicalTrials.gov)
February 20148/12/2013Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) PatientsMulti-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD PatientsOculopharyngeal Muscular DystrophyDrug: CabalettaBioblast Pharma Ltd.NULLCompleted18 Years80 YearsAll25Phase 2United States;Canada;Israel
364NCT01890798
(ClinicalTrials.gov)
January 201427/6/2013Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment ProtocolA Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen StudyMuscular DystrophiesDrug: DrisapersenGlaxoSmithKlineNULLWithdrawn5 YearsN/AMale0Phase 3NULL
365EUCTR2013-001194-25-NL
(EUCTR)
16/12/201317/07/2013A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Cialis 2.5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 10 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 20 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Eli Lilly and CompanyNULLNot RecruitingFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of
366NCT02195999
(ClinicalTrials.gov)
December 201317/7/2014Assessment of Cardiopulmonary Function in Duchenne Muscular DystrophyAssessment of Cardiopulmonary Function in Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneOther: Magnetic Resonance Imaging (MRI);Other: Pulmonary Function Testing (PFT);Other: Metabolic Exercise Testing using stationary bicycle;Other: EchocardiogramUniversity of FloridaCureDuchenneCompleted5 Years15 YearsMale9United States
367NCT02056808
(ClinicalTrials.gov)
November 201321/11/2013A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: SMT C1100Summit Corporation Plc.NULLCompleted5 Years11 YearsMale12Phase 1United Kingdom
368NCT02285673
(ClinicalTrials.gov)
November 20135/11/2014Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular DystrophyEfficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 StudyDuchenne Muscular DystrophyBiological: Umbilical Cord Mesenchymal Stem CellAcibadem UniversityNULLRecruiting7 Years20 YearsMale10Phase 1/Phase 2Turkey
369NCT01970735
(ClinicalTrials.gov)
October 201323/10/2013Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2Muscular Dystrophy, FacioscapulohumeralBiological: Blood testCentre Hospitalier Universitaire de NiceNULLRecruiting18 Years75 YearsBoth100N/AFrance
370NCT02147639
(ClinicalTrials.gov)
October 201325/3/2014Effects of Sodium Nitrate on Blood Flow in Becker Muscular DystrophyBecker Muscular DystrophyDietary Supplement: Sodium Nitrate;Dietary Supplement: Sodium Nitrate - double dose;Dietary Supplement: Placebo;Procedure: Increased exercise intensityCedars-Sinai Medical CenterNULLCompleted15 Years45 YearsMale19Phase 2/Phase 3United States
371NCT01978366
(ClinicalTrials.gov)
October 201331/10/2013Open Label Extension Study of HT-100 in Patients With DMDAn Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01Duchenne Muscular DystrophyDrug: HT-100Processa PharmaceuticalsNULLTerminated6 Years20 YearsMale17Phase 2United States
372NCT01995032
(ClinicalTrials.gov)
October 201320/11/2013L-citrulline and Metformin in Duchenne's Muscular DystrophyA Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne's Muscular DystrophyDuchenne's Muscular Dystrophy (DMD)Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.;Drug: PlaceboUniversity Hospital, Basel, SwitzerlandNULLCompleted78 Months10 YearsAll47Phase 3Switzerland
373NCT02847975
(ClinicalTrials.gov)
October 201330/4/2014Sodium Nitrate to Improve Blood FlowSodium Nitrate to Improve Blood FlowBecker Muscular DystrophyDietary Supplement: Sodium nitrateCedars-Sinai Medical CenterNULLCompleted15 Years55 YearsMale11Phase 1United States
374EUCTR2013-001194-25-GB
(EUCTR)
25/09/201305/06/2013A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Cialis 2.5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 10 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 20 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Eli Lilly and CompanyNULLNot RecruitingFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of
375EUCTR2013-001194-25-BE
(EUCTR)
19/09/201328/06/2013A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Cialis 2.5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 10 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 20 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Eli Lilly and CompanyNULLNot RecruitingFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of
376EUCTR2013-001732-21-FR
(EUCTR)
06/09/201318/02/2014A phase II study of metformin in myotonic dystrophy type 1 patientsA randomized, double blind, placebo-controlled phase II study of metformin in myotonic dystrophy type 1 patients - Myomet Myotonic dystrophy type 1 (DM1) also known as Steinert disease;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Trade Name: metformin 500mg
INN or Proposed INN: METFORMIN
Centre d'Etude des Cellules Souches (CECS)NULLNot RecruitingFemale: yes
Male: yes
30Phase 2France
377EUCTR2013-001194-25-DE
(EUCTR)
03/09/201313/06/2013A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Cialis 2.5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 10 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 20 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Eli Lilly and CompanyNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of
378NCT01865084
(ClinicalTrials.gov)
September 201324/5/2013A Study of Tadalafil for Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Tadalafil;Drug: PlaceboEli Lilly and CompanyNULLTerminated7 Years14 YearsMale331Phase 3United States;Argentina;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Puerto Rico;Russian Federation;Spain;Taiwan;Turkey;United Kingdom
379EUCTR2012-004527-20-SE
(EUCTR)
30/08/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Sweden;Korea, Republic of
380EUCTR2012-004527-20-CZ
(EUCTR)
30/08/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
381EUCTR2013-001194-25-ES
(EUCTR)
27/08/201326/06/2013A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Cialis 2.5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 10 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 20 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Lilly S.A.NULLNot RecruitingFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of
382EUCTR2011-005042-35-GB
(EUCTR)
23/08/201318/03/2013A study to test if BMN 053 is safe and effective in people who suffer from Duchenne muscular dystrophyA Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 053
Product Code: BMN 053
INN or Proposed INN: PS524
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: no
Male: yes
45 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
383EUCTR2013-002115-99-GB
(EUCTR)
23/08/201331/07/2013A Clinical Trial to investigate if the drug SMT C1100 is safe and well tolerated in children with DMD when given orally one to three times a day.SMT C1100 - A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: SMT C1100
INN or Proposed INN: 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
Summit Corporation plcNULLNot RecruitingFemale: no
Male: yes
Phase 1United Kingdom
384EUCTR2012-004527-20-DE
(EUCTR)
20/08/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
385JPRN-JMA-IIA00134
14/08/201305/08/2013Phase II study of nonsense readthrough compound NPC-14 (Arbekacin sulfate) to explore safety, tolerability, and efficacy in Duchenne muscular dystrophy patients (NORTH POLE DMD study)Phase II study of nonsense readthrough compound NPC-14 (Arbekacin sulfate) to explore safety, tolerability, and efficacy in Duchenne muscular dystrophy patients (NORTH POLE DMD study) Duchenne muscular dystrophyIntervention type:DRUG. Intervention1:NPC-14, Dose form:INJECTION, Route of administration:INTRAVENOUS DRIP, intended dose regimen:Not applicable. Control intervention1:Placebo, Dose form:INJECTION, Route of administration:INTRAVENOUS DRIP, Intended dose regimen:Not applicable.Yasuhiro Takeshima, MD, PhDHyogo College of Medicine Hospital, department of pediatricsNot applicableCompleted>=4 YEARSNo LimitMale21Phase 2Japan
386EUCTR2013-001194-25-IT
(EUCTR)
09/08/201311/06/2013A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children.A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Cialis 2.5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 5 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 10 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Trade Name: Cialis 20 mg film-coated tablets
INN or Proposed INN: TADALAFIL
Eli Lilly and CompanyNULLNot RecruitingFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of
387EUCTR2012-004527-20-GB
(EUCTR)
08/08/201324/06/2013 Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot Recruiting Female: no
Male: yes
220 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
388NCT01918384
(ClinicalTrials.gov)
August 20131/8/2013Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular DystrophyPhase II Study of Nonsense Readthrough Compound NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy Patients (NORTH POLE DMD Study)Muscular Dystrophy, DuchenneDrug: NPC-14;Drug: PlaceboKobe UniversityJapan Medical Association;NobelpharmaActive, not recruiting4 YearsN/AMale21Phase 2Japan
389NCT01921374
(ClinicalTrials.gov)
August 20138/8/2013Mother-caregivers of Children With Duchenne Muscular DystrophyProfile of Mother-caregivers of Children With Duchenne Muscular DystrophyOther Diseases or ConditionsBiological: sleep parameters;Biological: Hormonal profile;Biological: Inflammatory profile;Biological: Cardiovascular profile;Biological: Metabolic profileMonica Levy AndersenNULLCompleted25 Years65 YearsFemale60N/ABrazil
390EUCTR2012-004527-20-BE
(EUCTR)
30/07/201324/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Belgium;Brazil;Australia;Germany;Korea, Republic of;Sweden
391EUCTR2012-004527-20-ES
(EUCTR)
17/07/201312/07/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Sweden;Korea, Republic of;Germany;Australia;Belgium;Brazil;Poland;Canada;Czech Republic;Switzerland
392EUCTR2012-004527-20-IT
(EUCTR)
01/07/201327/06/2013Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophyA Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
220Phase 3United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
393EUCTR2011-005042-35-BE
(EUCTR)
26/06/201321/03/2013A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyA Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN053
Product Code: BMN053
INN or Proposed INN: PS524
BioMarin Nederland B.V.NULLNot RecruitingFemale: no
Male: yes
42Phase 1;Phase 2United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
394EUCTR2011-005042-35-IT
(EUCTR)
20/06/201322/03/2013A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyA Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping
MedDRA version: 15.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO053
Product Code: PRO053
INN or Proposed INN: PS524
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
42Phase 1;Phase 2United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
395EUCTR2011-005042-35-NL
(EUCTR)
13/06/201315/08/2013A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophyA Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping
MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO053
Product Code: PRO053
INN or Proposed INN: PS524
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
42Phase 1;Phase 2United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
396NCT02018731
(ClinicalTrials.gov)
June 201317/12/2013L-citrulline and Metformin in Becker's Muscular DystrophyPilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular DystrophyBecker's Muscular Dystrophy (BMD)Drug: Metformin and Metformin & L-Citrulline;Drug: L-Citrulline and Metformin & L-CitrullineUniversity Hospital, Basel, SwitzerlandNULLCompleted18 YearsN/ABoth20Phase 2Switzerland
397NCT02081625
(ClinicalTrials.gov)
June 20135/3/2014Exploratory Study of NS-065/NCNP-01 in DMDExploratory Study of NS-065/NCNP-01 in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: NS-065/NCNP-01National Center of Neurology and Psychiatry, JapanNippon Shinyaku Co., Ltd.Completed5 Years18 YearsMale10Phase 1Japan
398NCT01957059
(ClinicalTrials.gov)
June 20132/7/2013A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy.Duchenne Muscular DystrophyDrug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing ExtensionBioMarin PharmaceuticalNULLTerminated5 Years18 YearsMale9Phase 1/Phase 2Belgium;France;Italy;Netherlands;United Kingdom
399EUCTR2011-005040-10-IT
(EUCTR)
16/05/201327/02/2013A study to test if multiple injections of PRO045 are safe and effective in people who suffer from Duchenne muscular dystrophyA phase I/II, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of PRO045 in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO045
Product Code: PRO045
INN or Proposed INN: PS220
Prosensa Therapeutics BVNULLNot RecruitingFemale: no
Male: yes
45Phase 1;Phase 2Belgium;United Kingdom;Italy
400NCT01803412
(ClinicalTrials.gov)
May 1, 201328/2/2013A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian SubjectsAn Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy.Muscular DystrophiesDrug: DrisapersenBioMarin PharmaceuticalNULLTerminated5 YearsN/AMale53Phase 3United States;Canada
401NCT01856868
(ClinicalTrials.gov)
May 20139/5/2013Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)An Open-label Pilot Study of Purified Tea-derived Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy.Becker Muscular DystrophyDrug: (-)-epicatechinCraig McDonald, MDCardero Therapeutics, Inc.Completed18 Years60 YearsMale7Phase 1/Phase 2United States
402NCT01847573
(ClinicalTrials.gov)
May 20132/5/2013Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular DystrophyA Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: HT-100Processa PharmaceuticalsNULLTerminated6 Years20 YearsMale17Phase 1/Phase 2United States
403EUCTR2011-005040-10-GB
(EUCTR)
22/04/201310/10/2012A study to test if multiple injections of BMN 045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophyA phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by BMN 045-induced DMD exon 45 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 045
Product Code: BMN 045
INN or Proposed INN: PS220
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: no
Male: yes
45Phase 1;Phase 2France;Belgium;Italy;United Kingdom
404NCT01761292
(ClinicalTrials.gov)
April 201320/12/2012A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMDA Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: GivinostatItalfarmacoNULLCompleted7 Years11 YearsMale20Phase 1/Phase 2Italy
405NCT01826487
(ClinicalTrials.gov)
March 26, 201326/3/2013Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation DystrophinopathyMuscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornDrug: Ataluren;Drug: PlaceboPTC TherapeuticsNULLCompleted7 Years16 YearsMale230Phase 3United States;Australia;Belgium;Brazil;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Bulgaria;Czech Republic
406NCT04740554
(ClinicalTrials.gov)
March 1, 20131/2/2021Heart Rate Variability in Individuals With Duchenne Muscular DystrophyCharacterization of Heart Rate Variability in Individuals With Duchenne Muscular Dystrophy - Influence of Corticosteroids and BetablockersDuchenne Muscular DystrophyBehavioral: Duchenne Muscular Dystrophy group with Deflazacort;Behavioral: Duchenne Muscular Dystrophy group with Prednisone/Predisolone;Behavioral: Duchenne Muscular Dystrophy group without Corticosteroids therapy;Behavioral: Control Group Typically DevelopingUniversity of Sao PauloNULLCompleted11 Years18 YearsMale40NULL
407NCT02814110
(ClinicalTrials.gov)
March 1, 201323/6/2016Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular DystrophyEfficacy and the Safety of Granulocyte Colony-stimulating Factor Treatment in Children and Adolescents With Muscular Dystrophy: An Open StudyIncrease Muscle Strength in Patients With Muscular DystrophyDrug: Granulocyte colony-stimulating factor (Filgrastim)Medical University of BialystokNULLRecruiting5 Years15 YearsAll27Phase 1Poland
408NCT01826422
(ClinicalTrials.gov)
March 20134/4/2013Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB PatientsEffect of Eicosapentaenoic Fatty Acid (EPA) and Docosahexaenoic Fatty Acids (DHA) Supplementation on the Inflammation State and Metabolic Disorders in Patients With Duchenne Muscular Dystrophy or Becker Muscular DystrophyMuscular Dystrophy, DuchenneDietary Supplement: EPA and DHA;Dietary Supplement: Placebo ComparatorCoordinación de Investigación en Salud, MexicoInstituto Nacional de RehabilitacionCompleted6 Years18 YearsMale40N/AMexico
409EUCTR2010-023744-33-IT
(EUCTR)
26/02/201328/12/2012Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen.Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen. - FOR-DMD Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids.
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: DELTACORTENE*20CPR 5MG
INN or Proposed INN: PREDNISONE
Trade Name: DELTACORTENE*20CPR 5MG
INN or Proposed INN: PREDNISONE
Trade Name: DEFLAN*10CPR 6MG
INN or Proposed INN: DEFLAZACORT
AZIENDA OSPEDALIERA DI PADOVANULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
300United States;Canada;United Kingdom;Italy
410EUCTR2012-002566-12-IT
(EUCTR)
04/02/201317/10/2012A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular DystrophyA Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy - DSC Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: SOC;Classification code 10028395;Term: Musculoskeletal and connective tissue disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat
Product Code: ITF2357
INN or Proposed INN: Givinostat
Other descriptive name: givinostat
Product Name: Givinostat
Product Code: ITF2357
INN or Proposed INN: Givinostat
Other descriptive name: Givinostat
ITALFARMACONULLNot RecruitingFemale: no
Male: yes
20Phase 2Italy
411NCT03433807
(ClinicalTrials.gov)
January 29, 20138/2/2018Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: IdebenoneSanthera PharmaceuticalsNULLTemporarily not available8 YearsN/AAllUnited States
412NCT01826474
(ClinicalTrials.gov)
January 201320/3/2013Phase IIb Study of PRO045 in Subjects With Duchenne Muscular DystrophyA Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PRO045, 0.15 mg/kg/week;Drug: PRO045, 1.0 mg/kg/week;Drug: PRO045, 3.0 mg/kg/week;Drug: PRO045, 6.0 mg/kg/week;Drug: PRO045, 9.0 mg/kg/week;Drug: PRO045, selected doseBioMarin PharmaceuticalNULLTerminated5 Years18 YearsMale15Phase 1/Phase 2Belgium;France;Italy;Netherlands;United Kingdom
413NCT01603407
(ClinicalTrials.gov)
January 20133/4/2012Finding the Optimum Regimen for Duchenne Muscular DystrophyDuchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid RegimenDuchenne Muscular DystrophyDrug: Prednisone;Drug: DeflazacortUniversity of RochesterNewcastle University;University Medical Center Freiburg;National Institute of Neurological Disorders and Stroke (NINDS)Completed4 Years7 YearsMale196Phase 3United States;Canada;Germany;Italy;United Kingdom
414EUCTR2011-001266-17-CZ
(EUCTR)
20/12/201230/10/2012An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
INN or Proposed INN: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
415EUCTR2011-005040-10-BE
(EUCTR)
03/12/201204/09/2012A study to test if multiple injections of PRO045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophyA phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of PRO045 in subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping
MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: PRO045
Product Code: PRO045
INN or Proposed INN: PS220
BioMarin Nederland B.V.NULLNot RecruitingFemale: no
Male: yes
45Phase 1;Phase 2Belgium;Italy;United Kingdom
416EUCTR2010-023744-33-GB
(EUCTR)
12/11/201220/09/2012Trial to find best steroid treatment for Duchenne muscular dystrophyDuchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen - FOR-DMD Duchenne muscular dystrophy (DMD)
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Deflazacort
Product Name: Deflazacort 6 mg Tablets
INN or Proposed INN: Deflazacort
Trade Name: Prednisone
Product Name: Prednisone 5 mg tablets
INN or Proposed INN: Prednisone
University of RochesterNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
300United States;Canada;Germany;Italy;United Kingdom
417JPRN-UMIN000009307
2012/11/0810/11/2012Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophy Duchenne muscular dystrophyoral adm,inistration of aspirinKobe University Graduate School of MedicineNULLComplete: follow-up complete5years-oldNot applicableMale6Phase 1,2Japan
418EUCTR2011-001266-17-DK
(EUCTR)
11/10/201211/10/2012An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 15.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Phase 3Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
419EUCTR2011-004853-18-IT
(EUCTR)
18/09/201227/09/2012An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation DystrophinopathyAn Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 15.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 15.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: na
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: na
PTC THERAPEUTICS, INC.NULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;United Kingdom;Italy;Sweden
420EUCTR2011-004853-18-GB
(EUCTR)
21/08/201229/03/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot Recruiting Female: no
Male: yes
96 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
421EUCTR2011-001266-17-BG
(EUCTR)
09/08/201229/05/2012An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
422EUCTR2011-001266-17-HU
(EUCTR)
02/08/201218/06/2012An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
INN or Proposed INN: NA
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
423EUCTR2009-012037-30-ES
(EUCTR)
27/06/201229/03/2012Hereditary neuromuscular diseaseA Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 ? 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLNot RecruitingFemale: no
Male: yes
280Phase 3France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden
424EUCTR2011-004853-18-ES
(EUCTR)
13/06/201217/04/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden
425EUCTR2010-020069-26-HU
(EUCTR)
25/05/201228/02/2012A phase III, randomized, double blind, placebo-controlledclinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - N/A A phase III, randomized, double blind, placebo-controlledclinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - N/A Duchenne muscular dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3Japan;Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Norway;Netherlands;Germany
426NCT01557400
(ClinicalTrials.gov)
May 20, 201215/3/2012Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and CanadaAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation DystrophinopathyDuchenne Muscular Dystrophy;Becker Muscular Dystrophy;DystrophinopathyDrug: AtalurenPTC TherapeuticsNULLCompletedN/AN/AMale94Phase 3Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
427EUCTR2011-004853-18-FR
(EUCTR)
02/05/201213/08/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 14.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot Recruiting Female: no
Male: yes
96 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
428EUCTR2011-004853-18-DE
(EUCTR)
02/05/201212/03/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, Inc.NULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
429EUCTR2011-004853-18-BE
(EUCTR)
27/04/201203/01/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy Nonsense mutation dystrophinopathy
MedDRA version: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
430EUCTR2011-004853-18-SE
(EUCTR)
26/04/201227/02/2012Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A Nonsense mutation dystrophinopathy
MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
Product Name: ataluren
Product Code: PTC124
INN or Proposed INN: ataluren
PTC Therapeutics, IncNULLNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
431EUCTR2009-012037-30-IT
(EUCTR)
27/03/201226/04/2012(DELOS) DuchEnne MD Long-term IdebenOne StudyA Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Idebenone
Product Code: NA
INN or Proposed INN: IDEBENONE
SANTHERA PHARMACEUTICALS (SWITZERLAND) LTDNULLNot RecruitingFemale: no
Male: yes
280Phase 3France;United States;Canada;Spain;Belgium;Austria;Netherlands;Germany;Italy;Sweden
432EUCTR2011-001266-17-IT
(EUCTR)
20/03/201208/03/2012An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
INN or Proposed INN: NA
Other descriptive name: NA
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.NULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Russian Federation;Chile;Israel;United Kingdom;Italy;Czech Republic;Hungary;Canada;Argentina;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of
433NCT01618331
(ClinicalTrials.gov)
March 20127/6/2012Protein Supplementation and Exercise in Patients With FSH Muscular Dystrophy- a Randomized Placebo Controlled StudyThe Effect of Protein Supplementation Doing Regular Exercise in Patients With Facioscapulohumeral Muscular Dystrophy - a Blinded RCT StudyFacioscapulohumeral Muscle DystrophyBehavioral: Regular exercise;Dietary Supplement: Protein-carbohydrate supplementationGrete Andersen, MDThe Augustinus Foundation, Denmark.;Aase and Ejnar Danielsens Foundation;The Danish Rheumatism Association;AP Moeller FoundationCompleted18 Years65 YearsBoth42N/ADenmark
434NCT02207283
(ClinicalTrials.gov)
March 201228/7/2014PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular DystrophyPDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular DystrophyBecker Muscular DystrophyDrug: Tadalafil;Drug: PlaceboCedars-Sinai Medical CenterNULLCompleted15 Years55 YearsMale12Phase 4NULL
435NCT01580501
(ClinicalTrials.gov)
March 201217/4/2012PDE Inhibitors in DMD Study (Acute Dosing Study)Functional Muscle Ischemia and PDE5 Inhibition in Duchenne Muscular Dystrophy: Acute Dosing StudyDuchenne Muscular DystrophyDrug: Tadalafil and SildenafilCedars-Sinai Medical CenterNULLCompleted7 Years15 YearsMale12Phase 1United States
436NCT03076814
(ClinicalTrials.gov)
March 201223/9/2014Functional Muscle Ischemia With Tadalafil Treatment in Becker Muscular DystrophyBecker Muscular DystrophyDrug: Tadalafil;Other: PlaceboCedars-Sinai Medical CenterNULLWithdrawn15 Years55 YearsMale0N/ANULL
437NCT01540409
(ClinicalTrials.gov)
February 27, 201223/2/2012Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyOpen-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201Duchenne Muscular Dystrophy (DMD)Drug: AVI-4658 (Eteplirsen)Sarepta Therapeutics, Inc.NULLCompleted7 Years13 YearsMale12Phase 2United States
438NCT01648634
(ClinicalTrials.gov)
February 13, 201220/7/2012Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Examine the Effect of Nebivolol, a Beta-Blockade Drug, for the Prevention of Ventricular Systolic Dysfunction in Patients With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy;Cardiomyopathy;Heart FailureDrug: Nebivolol;Drug: PlaceboAssistance Publique - Hôpitaux de ParisAssociation Française contre les Myopathies (AFM), ParisCompleted10 Years15 YearsMale51Phase 3France
439NCT01540604
(ClinicalTrials.gov)
February 201223/2/2012CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic CarriersAn Open-label, Un-controlled, Single-centre Trial Investigating the Efficacy and Safety of CRD007 in Children With Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or Children Being Symptomatic Carriers for DMD or BMDDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: CRD007Cardoz ABNULLCompleted2 Years11 YearsBothPhase 2Sweden
440NCT01521546
(ClinicalTrials.gov)
February 201226/1/2012Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular DystrophyEarly Treatment With Aldosterone Antagonism Attenuates Cardiomyopathy in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: eplerenone;Drug: placeboSubha RamanBallou SkiesCompleted7 YearsN/AMale42N/AUnited States
441NCT01388764
(ClinicalTrials.gov)
January 20125/7/2011Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on CorticosteroidsPilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on CorticosteroidsDystrophinopathy;Duchenne Muscular Dystrophy;Becker's Muscular DystrophyDrug: L-arginineMassachusetts General HospitalNULLCompleted7 Years11 YearsMale7Phase 1United States
442NCT02516085
(ClinicalTrials.gov)
January 20123/8/2015Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and MetforminDuchenne Muscular DystrophyDrug: Metformin;Drug: L-ArginineUniversity Hospital, Basel, SwitzerlandNULLCompleted7 Years10 YearsBoth5Phase 1NULL
443NCT01519349
(ClinicalTrials.gov)
January 201223/1/2012Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body MyositisPhase I Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis.Becker Muscular Dystrophy;Sporadic Inclusion Body MyositisBiological: rAAV1.CMV.huFollistatin344Nationwide Children's HospitalParent Project Muscular Dystrophy;The Myositis Association (Grant Sponsor)Completed18 YearsN/AAll15Phase 1United States
444EUCTR2011-004667-76-SE
(EUCTR)
19/12/201121/10/2011An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD.An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: pemirolast
Product Code: CRD007
INN or Proposed INN: PEMIROLAST
Cardoz ABNULLNot RecruitingFemale: yes
Male: yes
Sweden
445EUCTR2011-001266-17-ES
(EUCTR)
18/11/201103/10/2011An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
INN or Proposed INN: GSK2402968
GlaxoSmithKline, S.A.NULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
446EUCTR2011-001266-17-DE
(EUCTR)
16/11/201131/08/2011An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
447EUCTR2011-001266-17-NL
(EUCTR)
03/11/201128/09/2011An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Germany;Netherlands;Norway;Japan;Korea, Republic of
448NCT01350154
(ClinicalTrials.gov)
November 20114/5/2011Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy PatientsDoes Modulation of the nNOS System in Patients With Muscular Dystrophy and Defect nNOS Signalling Affect Cardiac, Muscular or Cognitive Function?Becker Muscular DystrophyDrug: Sildenafil;Drug: PlaceboRigshospitalet, DenmarkGlostrup University Hospital, CopenhagenCompleted18 Years80 YearsMale17Phase 2Denmark
449NCT01462292
(ClinicalTrials.gov)
October 26, 20113/10/2011A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)An Exploratory Study to Assess Two Doses of GSK2402968 in the Treatment of Ambulant Boys With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968 3mg/kg/week;Drug: GSK2402968 6 mg/kg/week;Drug: Placebo to match GSK2402968 3 mg/kg/week;Drug: Placebo to match GSK2402968 6 mg/kg/weekGlaxoSmithKlineNULLCompleted5 YearsN/AMale51Phase 2United States
450EUCTR2011-001266-17-GB
(EUCTR)
03/10/201131/08/2011 An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot Recruiting Female: no
Male: yes
200 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noTaiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
451NCT01610440
(ClinicalTrials.gov)
October 201121/5/2012Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular DystrophyPhase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: human umbilical cord mesenchymal stem cellsShenzhen Beike Bio-Technology Co., Ltd.The Second Affiliated Hospital of Kunming Medical UniversityRecruiting5 Years12 YearsBoth15Phase 1/Phase 2China
452NCT01478022
(ClinicalTrials.gov)
October 201114/11/2011To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20Open, Randomized, 3 Period Cross-over Design, in Healthy Volunteers to Compare the Pharmacokinetics Profiles of 3 Treatments: ISO 20, IBU 200 and IBU Plus ISO Combinations (200 + 20) Administered Per os as Single DosesDuchenne Muscular Dystrophy (DMD)Drug: Isosorbide Dinitrate;Drug: Ibuprofen;Other: Ibuprofen and Isosorbide Dinitrate combinationParent Project, ItalyNULLCompleted18 Years27 YearsAll12Phase 1Italy
453NCT01480245
(ClinicalTrials.gov)
September 201123/11/2011Open Label Study of GSK2402968 in Subjects With Duchenne Muscular DystrophyAn Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968GlaxoSmithKlineNULLTerminated5 YearsN/AMale233Phase 3Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czech Republic;Denmark;France;Germany;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom
454EUCTR2011-001266-17-BE
(EUCTR)
08/08/201130/06/2011An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A Duchenne Muscular Dystrophy
MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
200Phase 3Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Poland;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
455EUCTR2010-024659-10-DK
(EUCTR)
03/08/201121/06/2011Can certain muscle wasting diseases be improved with sildenafil?Can stimulation of the nNOS system in muscle disease with nNOS insufficiency improve heart and skeletal muscle function and cognition? Muscular dystrophy with nNOS insufficiency
MedDRA version: 14.0;Level: LLT;Classification code 10028301;Term: Muscle disorder NOS;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Revatio
Product Name: Sildenafil
Product Code: EMEA/H/C/000638
RigshospitaletNULLNot RecruitingFemale: no
Male: yes
Denmark
456NCT01645098
(ClinicalTrials.gov)
August 20118/6/2012Sedation During Muscle Biopsy in Patients With Duchenne Muscular DystrophySedation During Muscle Biopsy in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Ketamine;Drug: DexmedetomidineNationwide Children's HospitalNULLCompletedN/AN/AMale53N/AUnited States
457NCT01422200
(ClinicalTrials.gov)
August 201122/8/2011Flu Vaccine Study in Neuromuscular Patients 2011Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular DiseasesDuchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular DystrophyBiological: 2011-2012 seasonal flu vaccine Subcutaneous;Biological: 2011-2012 seasonal flu vaccine IntramuscularChildren's Hospital Medical Center, CincinnatiNULLCompleted3 Years35 YearsAll22Phase 4United States
458NCT01396239
(ClinicalTrials.gov)
July 20118/7/2011Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsA Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658(Eteplirsen),in the Treatment of Ambulant Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: AVI-4658 (Eteplirsen);Other: PlaceboSarepta Therapeutics, Inc.NULLCompleted7 Years13 YearsMale12Phase 2United States
459EUCTR2010-020069-26-DK
(EUCTR)
15/06/201118/05/2011A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A Duchenne muscular dystrophy
MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan
460NCT01406873
(ClinicalTrials.gov)
June 201120/7/2011Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)Myotonic DystrophyDrug: Mexiletine;Drug: PlaceboUniversity of RochesterNULLCompleted18 Years80 YearsAll42Phase 2United States
461NCT01359670
(ClinicalTrials.gov)
May 201123/5/2011Tadalafil and Sildenafil for Duchenne Muscular DystrophyFunctional Muscle Ischemia and PDE5A Inhibition in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Tadalafil;Drug: SildenafilCedars-Sinai Medical CenterParent Project Muscular DystrophyCompleted7 Years15 YearsMale30Early Phase 1United States
462EUCTR2010-020069-26-ES
(EUCTR)
11/04/201118/02/2011Ensayo clínico de fase III, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad de GSK2402968 en sujetos con distrofia muscular de Duchenne.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/AEnsayo clínico de fase III, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad de GSK2402968 en sujetos con distrofia muscular de Duchenne.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A Duchenne muscular dystrophy
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
MedDRA version: 13;Term: Distrofia muscular de Duchenne
Product Code: GSK2402968GlaxoSmithKline.S.A.NULLNot RecruitingFemale: no
Male: yes
190Phase 3France;Hungary;Czech Republic;Poland;Belgium;Spain;Denmark;Norway;Netherlands;Germany;Italy
463EUCTR2010-020069-26-PL
(EUCTR)
30/03/201103/01/2011A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A Duchenne muscular dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan
464EUCTR2010-020069-26-BE
(EUCTR)
15/03/201125/11/2010A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/AA phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A Duchenne muscular dystrophy
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Name: GSK2402968
Product Code: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Netherlands;Germany;Italy
465EUCTR2010-020069-26-NL
(EUCTR)
02/03/201104/11/2010A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/AA phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A Duchenne muscular dystrophy
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Germany;Netherlands;Italy
466NCT01335295
(ClinicalTrials.gov)
March 201112/4/2011Safety Study of Flavocoxid in Duchenne Muscular DystrophyOpen Pilot Trial to Test the Safety and Tolerability of Flavocoxid in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: FlavocoxidUniversity of MessinaNULLCompleted4 Years16 YearsMale20Phase 1Italy
467EUCTR2010-020069-26-CZ
(EUCTR)
24/02/201113/01/2011A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A Duchenne muscular dystrophy
MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan
468EUCTR2011-000176-33-IT
(EUCTR)
14/02/201123/02/2011Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - NDCell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND Duchenne Muscolar Dystrophy
MedDRA version: 9.1;Level: PT;Classification code 10013801
INN or Proposed INN: Tacrolimus
Product Name: hMABs
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Product Name: hMABs
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Product Name: hMABs
Other descriptive name: HLA-identical allogeneic mesoangioblasts
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABORNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
Italy
469NCT01183767
(ClinicalTrials.gov)
December 30, 201017/8/2010Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular DystrophySunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Epigallocatechin-Gallate;Drug: PlaceboCharite University, Berlin, GermanyNULLCompleted5 Years10 YearsAll33Phase 2/Phase 3Germany
470NCT01254019
(ClinicalTrials.gov)
December 2, 201021/10/2010A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular DystrophyA Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968 6mg/kg/weekGlaxoSmithKlineNULLCompleted5 YearsN/AMale186Phase 3Argentina;Belgium;Brazil;Canada;Chile;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;Czech Republic
471NCT02245711
(ClinicalTrials.gov)
December 201012/9/2014Cell Therapy in Limb Girdle Muscular DystrophyIntrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular DystrophyLimb Girdle Muscular DystrophyBiological: Stem CellNeurogen Brain and Spine InstituteNULLWithdrawn15 Years60 YearsAll0Phase 1India
472NCT01247207
(ClinicalTrials.gov)
November 30, 201022/11/2010Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation DystrophinopathyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsNULLEnrolling by invitationN/AN/AMale270Phase 3United States;Canada
473EUCTR2010-018412-32-ES
(EUCTR)
18/11/201001/12/2010Estudio clínico de fase II, doble ciego, explorador, de grupos paralelos y controlado con placebo para evaluar dos pautas posológicas de GSK2402968 para la eficacia, seguridad, tolerabilidad y farmacocinética en sujetos ambulatorios con distrofia muscular de DuchenneEstudio clínico de fase II, doble ciego, explorador, de grupos paralelos y controlado con placebo para evaluar dos pautas posológicas de GSK2402968 para la eficacia, seguridad, tolerabilidad y farmacocinética en sujetos ambulatorios con distrofia muscular de Duchenne Distrofia muscular de Duchenne
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
54France;Spain;Netherlands;Germany;United Kingdom
474EUCTR2010-020069-26-IT
(EUCTR)
16/11/201021/12/2010A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - NDA phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - ND Duchenne muscular dystrophy
MedDRA version: 9.1;Level: LLT;Classification code 10013801
Product Name: GSK2402968
Product Code: GSK2402968
INN or Proposed INN: GSK2402968
GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
180Phase 3France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Netherlands;Germany;Italy
475NCT01207908
(ClinicalTrials.gov)
November 201022/9/2010Safety and Efficacy Study of IGF-1 in Duchenne Muscular DystrophyIGF-1 Therapy and Muscle Function in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: IGF-1;Other: Standard steroid treatmentChildren's Hospital Medical Center, CincinnatiTercica- Subsidiary of Ipsen;Charley's FundActive, not recruiting5 YearsN/AMale40Phase 1/Phase 2United States
476NCT01239758
(ClinicalTrials.gov)
October 20101/11/2010Extension Study of ACE-031 in Subjects With Duchenne Muscular DystrophyAn Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: ACE-031 (Extension of cohort 1 from core study, A031-03);Biological: ACE-031 (Extension of cohort 2 from core study, A031-03);Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)Acceleron Pharma, Inc.NULLTerminated4 YearsN/AMale11Phase 2Canada
477EUCTR2010-020069-26-FR
(EUCTR)
16/09/201015/07/2010A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophyA randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy Duchenne muscular dystrophy
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot Recruiting Female: no
Male: yes
180 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Norway;Netherlands;Germany;Italy
478EUCTR2010-018412-32-NL
(EUCTR)
13/09/201024/05/2010A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy - Duchenne Muscular Dystrophy
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
54Phase 2United Kingdom;Germany;Netherlands;France;Spain
479NCT01168908
(ClinicalTrials.gov)
September 201022/7/2010Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular DystrophyPhase 2 Clinical Trial of Sildenafil for Cardiac Dysfunction in Duchenne Muscular Dystrophy and Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: SildenafilHugo W. Moser Research Institute at Kennedy Krieger, Inc.Johns Hopkins UniversityTerminated18 Years50 YearsMale20Phase 2United States
480NCT01153932
(ClinicalTrials.gov)
September 201029/6/2010Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular DystrophyA Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968;Drug: matched placeboGlaxoSmithKlineNULLCompleted5 YearsN/AMale53Phase 2Australia;Belgium;France;Germany;Israel;Netherlands;Spain;Turkey;United Kingdom
481EUCTR2010-018412-32-DE
(EUCTR)
05/08/201031/03/2010A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophyA phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
54Phase 2France;Spain;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom
482EUCTR2010-018412-32-FR
(EUCTR)
03/08/201003/08/2010A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophyA phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot Recruiting Female: no
Male: yes
54 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;Spain;Belgium;Netherlands;Germany;United Kingdom
483NCT01128855
(ClinicalTrials.gov)
July 12, 201020/5/2010A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD SubjectsA Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: 3 mg/kg GSK2402968;Drug: 6 mg/kg GSK2402968;Drug: 9 mg/kg GSK2402968;Drug: 12 mg/kg GSK2402968;Other: PlaceboGlaxoSmithKlineNULLCompleted9 YearsN/AMale20Phase 1United States;France
484EUCTR2009-016482-28-DE
(EUCTR)
25/06/201015/03/2010SUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular DystrophySUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular Dystrophy - SUNIMUD Duchenne Muscular Dystrophy;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Sunphenon EGCG
INN or Proposed INN: Sunphenon
Charite Universitätsmedizin BerlinNULLNot RecruitingFemale: no
Male: yes
40Phase 2Germany
485EUCTR2009-017649-67-IE
(EUCTR)
22/06/201013/01/2010Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMDBone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD Bone Health in Duchenne Muscular Dystrophy- a randomised controlled study of Risedronate useTrade Name: Actonel Once a week 5mg film coated tablets
Product Name: Risedronate sodium
Product Code: Risedronate
Trade Name: Calcichew D3 Forte
Product Name: Calcichew D3 Forte
Product Code: Calcichew D3 Forte
The Central Remedial Clinic and The Children's University HospitalNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
70Ireland
486EUCTR2010-018412-32-GB
(EUCTR)
17/06/201016/04/2010A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophyA phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
54Phase 2France;Spain;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom
487EUCTR2010-018412-32-BE
(EUCTR)
15/06/201007/04/2010A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy Duchenne Muscular Dystrophy
MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Code: GSK2402968GlaxoSmithKline Research and Development LTDNULLNot RecruitingFemale: no
Male: yes
54Phase 2France;Spain;Belgium;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom
488EUCTR2009-012037-30-NL
(EUCTR)
03/06/201017/12/2009Hereditary neuromuscular diseaseA Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS Duchenne Muscular Dystrophy
MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Idebenone
Product Code: SNT-MC17
INN or Proposed INN: IDEBENONE
Santhera Pharmaceuticals (Switzerland) LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
266Phase 3France;United States;Belgium;Spain;Austria;Germany;Netherlands;Italy;Switzerland;Sweden
489NCT01596803
(ClinicalTrials.gov)
May 1, 20105/4/2012Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD)Effects of Antioxidants Supplementation on Muscular Function of Patients Affected by Facioscapulohumeral Dystrophy (FSHD)Facioscapulohumeral Muscular DystrophyProcedure: Taking of blood;Dietary Supplement: needle biopsy of the vastus lateralis muscle;Dietary Supplement: Vit C Vit E Zn Se;Dietary Supplement: Placebo Vit E Placebo Vit C Zn SeUniversity Hospital, MontpellierHospital Clinical Research Project 2010;Association Amis FSH France;FSH Dutch Fondation The NetherlandCompleted18 Years60 YearsAll54N/AFrance
490EUCTR2009-013762-63-NL
(EUCTR)
16/04/201025/08/2009A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyA phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy Duchenne Muscular DystrophyProduct Name: PRO044
Product Code: PRO044
INN or Proposed INN: h44AON188
Prosensa Therapeutics B.VNULLNot RecruitingFemale: no
Male: yes
18Phase 1/2Belgium;Netherlands;Italy;Sweden
491