113. 筋ジストロフィー [臨床試験数:567,薬物数:442(DrugBank:93),標的遺伝子数:55,標的パスウェイ数:151

Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α-dystroglycanopathy", "FCMD", "Walker-Warburg syndrome", "Muscle-eye-brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04336826
(ClinicalTrials.gov)
June 1, 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 TherapeuticsNULLNot yet recruiting6 Months2 YearsMale6Phase 2NULL
2EUCTR2020-000698-26-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
3EUCTR2017-004625-32-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;Australia;Bulgaria;Germany;Sweden
4NCT04632940
(ClinicalTrials.gov)
December 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: PlaceboFibroGenNULLNot yet recruiting6 Years11 YearsMale70Phase 3NULL
5NCT04624750
(ClinicalTrials.gov)
November 16, 202020/10/2020Open 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.NULLNot yet recruiting6 Years18 YearsAll14Phase 3NULL
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT04184882
(ClinicalTrials.gov)
November 12, 20202/12/2019A 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: ASP0367;Drug: PlaceboAstellas Pharma IncNULLRecruiting8 Years16 YearsMale18Phase 1United States
7EUCTR2019-002076-13-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;Taiwan;Greece;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of
8NCT04634682
(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
9NCT04587908
(ClinicalTrials.gov)
November 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.NULLNot yet recruiting5 YearsN/AMale80Phase 3Japan
10NCT03692312
(ClinicalTrials.gov)
November 202016/3/2018Efficacy 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 DystrophyCongenital Myotonic DystrophyDrug: Tideglusib;Drug: PlaceboAMO Pharma LimitedNULLNot yet recruiting6 Years16 YearsAll56Phase 2/Phase 3United States;Canada;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT04616807
(ClinicalTrials.gov)
October 31, 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.NULLNot yet recruiting18 YearsN/AAll50NULL
12NCT03340675
(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
13NCT04585464
(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, PK and Food Effect 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 TrialsRecruiting19 Years55 YearsAll152Phase 1United States
14NCT04322357
(ClinicalTrials.gov)
October 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 TrainingUniversity of FloridaU.S. Army Medical Research and Development CommandNot yet recruiting5 Years8 YearsMale65Phase 2NULL
15EUCTR2019-002921-31-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT04371666
(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: PlaceboFibroGenNULLRecruiting12 YearsN/AMale90Phase 3United States
17NCT04428476
(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.NULLEnrolling by invitation10 YearsN/AAll14Phase 2United States
18EUCTR2018-001762-42-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 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Switzerland;France;Mexico;Canada;Poland;Belgium;Denmark;Australia;Norway;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
19NCT04386304
(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.NULLRecruiting16 Years59 YearsMale20Phase 1United States
20NCT03992430
(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.NULLRecruiting7 Years13 YearsMale152Phase 3United States;Canada;Korea, Republic of;Taiwan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT04433234
(ClinicalTrials.gov)
July 8, 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.NULLEnrolling by invitation5 YearsN/AMale8Phase 2Japan
22EUCTR2019-003563-22-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
23EUCTR2017-004625-32-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;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
24ChiCTR2000032525
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
25EUCTR2018-001762-42-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26EUCTR2019-003563-22-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
27EUCTR2019-003563-22-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
140Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
28EUCTR2019-003563-22-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
29NCT04060199
(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;Canada;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Russian Federation;Spain;Taiwan;Turkey;Sweden
30EUCTR2019-000601-77-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
24Phase 2United States;Canada;Belgium;Spain;Ireland;Germany;Netherlands;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2015-002069-52-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
32EUCTR2018-001762-42-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.NULLNot RecruitingFemale: no
Male: yes
122Phase 3United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;United Kingdom;Switzerland;France;Mexico;Canada;Poland;Belgium;Denmark;Australia;Norway;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of
33EUCTR2017-004554-42-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
34EUCTR2016-000602-10-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
35EUCTR2019-002076-13-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;Taiwan;Greece;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2019-002076-13-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 3United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of
37NCT04254172
(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
38NCT04179409
(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.Enrolling by invitation6 MonthsN/AMale6Phase 2United States
39NCT04264442
(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
40EUCTR2017-004279-30-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2017-000397-10-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
42EUCTR2019-000601-77-ES
(EUCTR)
15/01/202005/07/2019A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients 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]
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;Italy;United Kingdom
43NCT04240314
(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
44NCT03895528
(ClinicalTrials.gov)
January 3, 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 BioPharmaceuticalsNULLAvailable12 MonthsN/AAllNULL
45EUCTR2018-001762-42-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
122Phase 3United States;Taiwan;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Australia;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46EUCTR2019-002076-13-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]
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
47EUCTR2019-000601-77-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
48NCT04129294
(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
49EUCTR2015-002069-52-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 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
50EUCTR2015-002069-52-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]
Sarepta Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
222 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSerbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Denmark;Australia;Bulgaria;Norway;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51NCT02878694
(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
52EUCTR2019-000601-77-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
53EUCTR2018-001762-42-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]
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
54EUCTR2018-004740-36-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 3France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden
55EUCTR2019-001181-15-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56EUCTR2018-004740-36-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
57EUCTR2017-002704-27-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]
ReveraGen BioPharma, Inc.NULLAuthorised-recruitment may be ongoing or finished 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;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden
58EUCTR2017-000397-10-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
185Phase 3United States;France;Canada;Spain;Belgium;Germany;Netherlands;United Kingdom;Italy
59EUCTR2018-004009-22-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
60EUCTR2019-001825-28-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]
The 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
61EUCTR2019-001181-15-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]
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
62EUCTR2018-001762-42-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
63NCT03907072
(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
64NCT04004000
(ClinicalTrials.gov)
August 23, 201925/6/2019Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1An 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)Facioscapulohumeral Muscular Dystrophy 1Drug: LosmapimodFulcrum TherapeuticsNULLActive, not recruiting18 Years65 YearsAll14Phase 2Netherlands
65EUCTR2017-002704-27-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
66NCT04003974
(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 TherapeuticsNULLActive, not recruiting18 Years65 YearsAll80Phase 2United States;Canada;France;Spain;Germany
67EUCTR2017-002704-27-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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
120Phase 2Czech Republic;Canada;Greece;Belgium;Spain;Australia;Israel;Netherlands;Germany;Italy;United Kingdom;Sweden
68EUCTR2018-004009-22-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]
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
69EUCTR2018-000692-32-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]
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
70EUCTR2018-004009-22-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
71EUCTR2017-002704-27-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 IIb 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
72NCT04054375
(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
73EUCTR2019-000337-39-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
74EUCTR2018-004009-22-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
75NCT04004065
(ClinicalTrials.gov)
June 26, 201927/6/2019Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients 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.NULLRecruiting4 Years21 YearsMale45Phase 2United States;Belgium;Canada;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76NCT03985878
(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
77EUCTR2017-002704-27-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
78NCT03959189
(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
79EUCTR2017-000397-10-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
80EUCTR2018-004009-22-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81NCT03789734
(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 CorporationNULLRecruiting19 Years55 YearsAll37Phase 1Korea, Republic of
82EUCTR2019-000337-39-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
83EUCTR2017-000397-10-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]
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
84NCT03783923
(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 TherapeuticsNULLActive, not recruiting18 YearsN/AAll30Phase 3United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden
85EUCTR2019-000305-79-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
86NCT03943290
(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
87NCT03936894
(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 Years5 YearsMale6Phase 1/Phase 2United States
88NCT03796637
(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
89NCT03917719
(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
90EUCTR2015-001192-48-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
91NCT02592941
(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
92EUCTR2017-004279-30-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]
Santhera Pharmaceuticals (Switzerland) LimitedNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
266 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
93EUCTR2018-000975-34-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]
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
94EUCTR2017-004625-32-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]
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
95EUCTR2018-000464-29-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
126Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
96EUCTR2017-004554-42-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
97EUCTR2017-004554-42-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]
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
98EUCTR2018-000464-29-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
99EUCTR2017-004554-42-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
100NCT03779646
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
101EUCTR2017-002213-60-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]
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
102NCT03648827
(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 TherapeuticsNULLActive, not recruiting2 Years7 YearsMale20Phase 2United States
103EUCTR2017-004279-30-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]
Santhera Pharmaceuticals (Switzerland) LimitedNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
266 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
104EUCTR2017-004625-32-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]
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): noUnited States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden
105NCT03675126
(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.NULLEnrolling by invitation4 YearsN/AMale60Phase 1/Phase 2United States;Canada
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
106JPRN-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
107EUCTR2016-000602-10-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
108JPRN-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
109EUCTR2018-000464-29-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
110EUCTR2015-002069-52-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
111NCT03777319
(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 AssociationRecruiting4 Years7 YearsMale24Phase 1United States
112EUCTR2016-004623-23-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]
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
113EUCTR2017-001629-41-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
48Phase 2Netherlands
114EUCTR2018-000975-34-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
115NCT03642145
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
116EUCTR2017-004279-30-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
117EUCTR2018-000975-34-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
118NCT03703882
(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
119EUCTR2017-002704-27-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
120ChiCTR1800018340
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
121EUCTR2017-004625-32-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
122EUCTR2017-004139-35-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]
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
123EUCTR2017-002704-27-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;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
124NCT03558958
(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
125EUCTR2015-002069-52-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: 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 3Norway;Germany;Sweden;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;United States;Serbia;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
126NCT03532542
(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;Czechia;Germany;Israel;Italy;Spain;Sweden;United Kingdom
127EUCTR2017-004625-32-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
128NCT03400852
(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
129EUCTR2016-001615-21-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]
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
130NCT03603288
(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 PharmaceuticalsNULLRecruiting11 YearsN/AMale266Phase 3United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
131NCT03603171
(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
132EUCTR2017-002686-21-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
133NCT03439670
(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 PittsburghActive, not recruiting4 Years7 YearsMale121Phase 2United States;Australia;Belgium;Canada;Czechia;Greece;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy
134EUCTR2017-004139-35-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
135EUCTR2017-004279-30-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
136EUCTR2017-004139-35-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]
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
137NCT03354039
(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, SwitzerlandNULLRecruiting78 Months16 YearsMale99Phase 3France;Germany;Netherlands;Spain;Switzerland;United Kingdom
138NCT03424460
(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/AAll69N/AFrance
139EUCTR2017-002686-21-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]
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
140EUCTR2017-002686-21-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
141EUCTR2016-003257-15-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]
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
142EUCTR2017-002686-21-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]
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
143NCT03406780
(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
144NCT03375255
(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
145NCT03508947
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
146EUCTR2017-001223-49-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 3United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of
147EUCTR2017-003568-10-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
148NCT03123913
(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 RochesterNULLRecruiting18 Years65 YearsMale20Phase 1United States
149NCT03238235
(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
150EUCTR2017-003568-10-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
151EUCTR2016-001654-18-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
152EUCTR2016-001654-18-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
153EUCTR2016-001654-18-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
154NCT03333590
(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
155NCT02653833
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
156EUCTR2016-001654-18-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
157EUCTR2016-001654-18-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
158NCT03373968
(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/AMale100Phase 2/Phase 3United States;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;United Kingdom
159EUCTR2016-001654-18-IT
(EUCTR)
19/09/201707/02/2018Clinical 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 CORPORATIONNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
160Phase 3United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
160EUCTR2015-002069-52-CZ
(EUCTR)
11/09/201714/03/2017A 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: 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;Portugal;Greece;Spain;Ireland;Russian Federation;Chile;Israel;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
161EUCTR2016-001654-18-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]
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
162NCT03218995
(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.NULLActive, not recruiting6 Months48 MonthsMale12Phase 2Belgium;France;Italy;United Kingdom;Germany
163EUCTR2016-001654-18-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
164EUCTR2017-000397-10-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]
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
165NCT03863119
(ClinicalTrials.gov)
July 15, 201721/2/2019Expanded 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
166EUCTR2016-000951-29-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]
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
167NCT03179631
(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 Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Disease;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, InbornDrug: Ataluren;Drug: PLACEBOPTC TherapeuticsNULLActive, not recruiting5 YearsN/AMale250Phase 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
168NCT03039686
(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
169NCT03167255
(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)Active, not recruiting4 Years10 YearsMale16Phase 2United States;Canada
170EUCTR2016-000401-36-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]
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
242 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSerbia;France;United States;Canada;Belgium;Spain;Israel;Germany;Netherlands;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
171EUCTR2015-002069-52-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
222Phase 3Serbia;Portugal;United States;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden
172EUCTR2016-000951-29-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
173NCT04226924
(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
174EUCTR2016-000951-29-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
175EUCTR2016-000951-29-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
176NCT02851797
(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 YearsMale169Phase 3United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;United Kingdom
177EUCTR2016-000951-29-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, IncNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
15Phase 2France;Belgium;Germany;Italy;United Kingdom
178EUCTR2016-000401-36-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
179EUCTR2016-000401-36-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]
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
242 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom
180EUCTR2015-002069-52-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 States;Canada;Spain;Israel;Germany;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
181EUCTR2015-002069-52-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: 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;Spain;Ukraine;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Denmark;Australia;Bulgaria;Germany;Sweden
182EUCTR2016-000602-10-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
183EUCTR2016-000401-36-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
184EUCTR2016-004462-26-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]
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
185EUCTR2016-004262-26-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
186EUCTR2016-004262-26-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
187EUCTR2016-004263-38-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
188EUCTR2016-004263-38-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]
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
189EUCTR2016-000401-36-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
190EUCTR2015-002069-52-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: 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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
191EUCTR2016-001615-21-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
192NCT03038399
(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
193EUCTR2016-000401-36-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]
ITALFARMACO S.p.A.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
242 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noSerbia;France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom
194EUCTR2015-002069-52-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
195EUCTR2015-002530-50-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
196EUCTR2015-002069-52-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
197EUCTR2014-002072-92-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]
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
198EUCTR2016-000602-10-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
199EUCTR2016-000602-10-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) LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden
200EUCTR2016-000602-10-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) LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
266Phase 3France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
201EUCTR2016-001615-21-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
202NCT02740972
(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
203EUCTR2016-000602-10-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
204EUCTR2016-000602-10-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
205NCT02964377
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
206NCT03236662
(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 YearsMale10Phase 2United States
207NCT02927080
(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
208NCT02835079
(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
209EUCTR2016-000602-10-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
210EUCTR2013-005489-20-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
211NCT02907619
(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
212NCT02500381
(ClinicalTrials.gov)
September 28, 201614/7/2015Study of SRP-4045 and SRP-4053 in DMD PatientsA 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.NULLRecruiting7 Years13 YearsMale222Phase 3United States;Australia;Belgium;Bulgaria;Canada;Czechia;France;Germany;Greece;Hungary;Israel;Italy;Poland;Spain;Sweden;United Kingdom
213EUCTR2016-000602-10-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
214NCT02814019
(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 PharmaceuticalsNULLActive, not recruiting10 YearsN/AMale255Phase 3United States;Austria;Belgium;Bulgaria;France;Germany;Hungary;Ireland;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom
215EUCTR2014-002072-92-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
216NCT02760277
(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
217NCT02858908
(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
218NCT02836418
(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
219EUCTR2014-002008-25-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]
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
220EUCTR2016-000624-25-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
221NCT02819557
(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
222NCT02760264
(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
223NCT02858362
(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
224NCT02808585
(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
225EUCTR2014-005296-81-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
226NCT02752048
(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
227EUCTR2016-000067-16-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
228NCT02958202
(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
229NCT02704325
(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
230NCT02439216
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
231NCT02603562
(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
232NCT02710591
(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
233EUCTR2015-004333-27-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]
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
234JPRN-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
235EUCTR2015-001955-54-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
236EUCTR2015-003681-87-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
237EUCTR2015-001955-54-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
238NCT02485938
(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
239EUCTR2015-002530-50-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
240EUCTR2015-001910-88-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
241EUCTR2015-001955-54-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
242NCT02515669
(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
243NCT02606136
(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
244NCT02636686
(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
245EUCTR2015-001955-54-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
220Portugal;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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
246EUCTR2015-003195-68-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
247NCT02579239
(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
248EUCTR2015-002530-50-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
249NCT02571205
(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 TrustNULLUnknown status12 Years17 YearsMale15United Kingdom
250EUCTR2015-002530-50-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
251NCT02530905
(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
252NCT02667483
(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
253NCT02531217
(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
254NCT03067831
(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
255EUCTR2013-005489-20-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
256EUCTR2015-001912-36-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
257EUCTR2015-001967-38-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]
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
258NCT02420379
(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
259NCT02434627
(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
260NCT02484560
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
261NCT02525302
(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
262EUCTR2013-003605-26-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
263NCT02354352
(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
264EUCTR2014-002072-92-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
265NCT02376816
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
266NCT01976091
(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
267NCT02383511
(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
268EUCTR2013-003605-26-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
269NCT02310906
(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
270EUCTR2013-005489-20-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
271NCT02354781
(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
272NCT02328482
(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
273JPRN-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
274EUCTR2014-002072-92-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
275EUCTR2013-003605-26-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
276EUCTR2014-003100-78-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]
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
277NCT02312011
(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
278NCT01805024
(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
279NCT02329769
(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
280NCT02295748
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
281NCT02251600
(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
282EUCTR2013-003605-26-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
283NCT02310763
(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
284NCT02255552
(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
285EUCTR2014-002008-25-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
286NCT02286947
(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
287NCT02036463
(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
288EUCTR2014-002210-23-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]
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
289EUCTR2013-004427-37-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
290EUCTR2013-005489-20-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
291EUCTR2014-001753-17-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
292EUCTR2014-002008-25-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]
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
293EUCTR2013-005489-20-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
294EUCTR2013-005489-20-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
295NCT01834066
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
296NCT02235844
(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
297NCT02246478
(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
298EUCTR2013-005489-20-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
299EUCTR2013-005489-20-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]
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
300EUCTR2013-005489-20-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]
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
301NCT02239224
(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
302NCT02251457
(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
303EUCTR2014-001753-17-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
44France;United States;Netherlands;Italy
304JPRN-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
305NCT02196467
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
306NCT02167217
(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
307NCT02090959
(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
308EUCTR2012-004527-20-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
309NCT02015481
(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
310NCT01890798
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
311EUCTR2013-001194-25-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
312NCT02195999
(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
313NCT02056808
(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
314NCT02285673
(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
315NCT01970735
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
316NCT02147639
(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
317NCT01978366
(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
318NCT01995032
(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
319NCT02847975
(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
320EUCTR2013-001194-25-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
321EUCTR2013-001194-25-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
322EUCTR2013-001732-21-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
323EUCTR2013-001194-25-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
324NCT01865084
(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
325EUCTR2012-004527-20-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
326EUCTR2012-004527-20-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
327EUCTR2013-001194-25-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
328EUCTR2011-005042-35-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]
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
329EUCTR2013-002115-99-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
330EUCTR2012-004527-20-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
331JPRN-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
332EUCTR2013-001194-25-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
333EUCTR2012-004527-20-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]
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
334NCT01918384
(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
335NCT01921374
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
336EUCTR2012-004527-20-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
337EUCTR2012-004527-20-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;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden
338EUCTR2012-004527-20-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
339EUCTR2011-005042-35-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
340EUCTR2011-005042-35-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
341EUCTR2011-005042-35-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
342NCT02018731
(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
343NCT02081625
(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
344NCT01957059
(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
345EUCTR2011-005040-10-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
346NCT01803412
(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
347NCT01856868
(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
348NCT01847573
(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
349EUCTR2011-005040-10-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
350NCT01761292
(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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
351NCT01826487
(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
352NCT02814110
(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
353NCT01826422
(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
354EUCTR2010-023744-33-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
355EUCTR2012-002566-12-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
356NCT03433807
(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
357NCT01826474
(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
358NCT01603407
(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
359EUCTR2011-001266-17-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
360EUCTR2011-005040-10-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
361EUCTR2010-023744-33-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
362JPRN-UMIN000009307
2012/11/0810/11/2012Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophy