↑ 疾患リストへ ← 戻る

 113. 筋ジストロフィー [臨床試験数:289,薬物数:306(DrugBank:83),標的遺伝子数:57,標的パスウェイ数:156] 

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 of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03340675January 20204 November 2019Oral 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 CenterNot recruiting7 YearsN/AMale48Phase 2
2NCT04129294November 6, 201911 November 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.Recruiting4 Years17 YearsMale6Phase 1/Phase 2Japan
3NCT04060199October 23, 201911 November 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;Japan
4NCT03907072September 4, 201911 November 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.Recruiting5 Years12 YearsMale150Phase 2/Phase 3United States;Belgium;Canada;Czechia;France;Italy;Sweden
5NCT04004000August 23, 20199 September 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 TherapeuticsRecruiting18 Years65 YearsAll16Phase 2Netherlands
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT04003974August 9, 201911 November 2019Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)A 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 (FSHD)Drug: Losmapimod;Drug: Placebo oral tabletFulcrum TherapeuticsRecruiting18 Years65 YearsAll66Phase 2United States;Canada;France;Germany;Spain
7NCT03895528August 201929 July 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 BioPharmaceuticalsNot recruiting12 MonthsN/AAllPhase 3
8NCT03992430August 201915 July 2019A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) PatientsA 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 TherapeuticsNot recruiting7 Years13 YearsMale152Phase 3
9NCT04004065June 26, 20194 November 2019A Phase 2 Study 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.Recruiting7 Years21 YearsMale24Phase 2United States;Canada
10NCT03985878June 19, 201924 June 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 TherapeuticsNot recruiting2 Years5 YearsMale15Phase 2
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03959189June 17, 20194 November 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.Recruiting18 Years60 YearsAll12Phase 1United States
12NCT03783923May 15, 201930 September 2019A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)A 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 DystrophyDrug: Deflazacort;Drug: PlaceboPTC TherapeuticsRecruiting18 YearsN/AAll100Phase 3United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden
13NCT03943290May 10, 201914 October 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.Recruiting18 YearsN/AAll150Phase 2United States;Canada;Spain
14NCT03433807May 9, 201926 August 2019Expanded 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 PharmaceuticalsNot recruiting8 YearsN/AAllPhase 3United States
15NCT03936894May 1, 201928 October 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 Research InstituteFoundation to Eradicate DuchenneRecruiting2 Years5 YearsMale6Phase 1/Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT03917719March 14, 201917 June 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 PharmaceuticalsRecruiting4 Years10 YearsMale100Phase 3United States
17EUCTR2015-001192-48-FR04/03/201930 April 2019Traitement 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-DOPPatients 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 autologues
Pharmaceutical Form: Suspension for injection
CHU CAENAuthorised Female: yes
Male: yes
10Phase 2France
18NCT03789734March 201914 January 2019Safety 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 CorporationNot recruiting19 Years55 YearsAll37Phase 1Korea, Republic of
19EUCTR2018-000464-29-DE31/01/201930 April 2019A 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 DMDDuchenne 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
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: EDASALONEXENT
Current Sponsor code: CAT-1004
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use
Product Name: Edasalonexent
Product Code: CAT-1004
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: EDASALONEXENT
Current Sponsor code: CAT-1004
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use
Catabasis Pharmaceuticals, Inc.Authorised Female: no
Male: yes
126Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
20EUCTR2017-004554-42-ES22/01/201928 February 2019The 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 trialDuchenne muscular dystrophy
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: TAMOX
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Tamoxifen
CAS Number: 54965-24-1
Other descriptive name: TAMOXIFEN CITRATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
University of Basel Children's Hospital, Division of NeuropediatricsAuthorised Female: no
Male: yes
100Phase 3France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT03779646January 16, 201928 January 2019Bisoprolol 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
22NCT03769116December 22, 201811 November 2019A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)A 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week ExtensionMuscular Dystrophy, DuchenneDrug: SRP-9001;Drug: PlaceboSarepta Therapeutics, Inc.Recruiting4 Years7 YearsMale40Phase 2United States
23NCT03648827December 21, 201814 October 2019A 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 TherapeuticsNot recruiting2 Years7 YearsMale20Phase 2United States
24NCT03675126December 19, 201826 August 2019An 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.Recruiting7 YearsN/AMale54Phase 1/Phase 2United States;Canada
25JPRN-jRCTs03118003818/12/201822 July 2019Tranilast-MDA multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MDmuscular 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.
Tsuyoshi MatsumuraRecruiting>=13 age oldBoth20N/ANone
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26JPRN-UMIN0000319652018/12/142 April 2019A 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-MDHeart 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 HospitalRecruiting13years-oldNot applicableMale and Female20Not selectedJapan
27NCT03777319December 5, 201811 February 2019Spironolactone 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
28EUCTR2016-004623-23-GB19/11/201830 April 2019A 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 DystrophyTreatment 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 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
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: Tideglusib
CAS Number: 865854-05-3
Current Sponsor code: AMO-02
Other descriptive name: 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione
Concentration unit: mg milligram(s)
Concentration type: up to
Concentration number: 1000-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use
AMO Pharma LtdAuthorised Female: yes
Male: yes
56Phase 2;Phase 3United States;Canada;United Kingdom
29EUCTR2017-001629-41-NL15/11/201828 February 2019Study 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 of givinostat in patients with Becker Muscular Dystrophy - NADistrofia 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Givinostat (hydrochloride monohydrate)
CAS Number: 732302-99-7
Current Sponsor code: ITF2357
Other descriptive name: GIVINOSTAT
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
ITALFARMACO S.P.A.AuthorisedFemale: no
Male: yes
48Phase 2Netherlands
30NCT03642145October 31, 201815 July 2019A 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 TherapeuticsNot recruiting2 Years4 YearsMale0Phase 3United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT03652259October 26, 20184 November 2019Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency)A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (ß-Sarcoglycan Deficiency).Limb-Girdle Muscular Dystrophy, Type 2EDrug: SRP-9003Sarepta Therapeutics, Inc.Recruiting4 Years15 YearsAll6Phase 1/Phase 2United States
32NCT03703882October 2, 201822 October 2019Phase 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 PharmaceuticalsNot recruiting4 Years7 YearsMale131Phase 3United States;Australia;Canada;Germany;Ireland;Israel;Sweden;United Kingdom
33EUCTR2017-002704-27-SE15/08/201827 August 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 DMDDuchenne 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: VAMOROLONE
CAS Number: 13209-41-1
Current Sponsor code: VBP15
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1.33-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: VAMOROLONE
CAS Number: 13209-41-1
Current Sponsor code: VBP15
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 4-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Product Name: Prednisone
Pharmaceutical Form: Tablet
INN or Proposed INN: PREDNISONE
CAS Number: 53-03-2
Other descriptive name: PREDNISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
ReveraGen BioPharma, Inc.AuthorisedFemale: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
34NCT03558958August 8, 20189 September 2019Safety 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 FundNot recruiting12 Years25 YearsMale10Phase 2United States
35NCT03532542August 2, 201811 November 2019An 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.Recruiting7 Years23 YearsMale260Phase 3United States;Belgium;Italy;Spain;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT03603288July 4, 201828 October 2019Phase 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 PharmaceuticalsRecruiting11 YearsN/AMale266Phase 3United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom
37NCT03603171July 1, 201826 August 2019Clinical 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 SchoserRecruiting18 Years90 YearsAll60Phase 2Germany
38NCT03439670June 19, 201822 October 2019A 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 PittsburghRecruiting4 Years7 YearsMale120Phase 2United States;Australia;Belgium;Canada;Czechia;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy
39EUCTR2017-004139-35-BE18/06/201820 August 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 DystrophyDuchenne 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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: TETRACOSACTIDE HEXAACETATE
CAS Number: 22633-88-1
Other descriptive name: Cosyntropin acetate
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1-
Pharmaceutical form of the placebo: Suspension for injection
Route of administration of the placebo: Subcutaneous use
Mallinckrodt ARD Inc.AuthorisedFemale: no
Male: yes
132Phase 2United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Germany;Netherlands
40EUCTR2017-004139-35-ES12/06/201818 June 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 DystrophyDuchenne 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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: TETRACOSACTIDE HEXAACETATE
CAS Number: 22633-88-1
Other descriptive name: Cosyntropin acetate
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 1-
Pharmaceutical form of the placebo: Suspension for injection
Route of administration of the placebo: Subcutaneous use
Mallinckrodt ARD Inc.AuthorisedFemale: no
Male: yes
132Phase 2United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Germany;Netherlands
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41NCT03354039June 12, 201817 June 2019Tamoxifen 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, SwitzerlandRecruiting78 Months16 YearsMale99Phase 3Germany;Netherlands;Spain;Switzerland;United Kingdom
42NCT03424460June 11, 201822 October 2019Venous Thromboembolism in Myotonic Dystrophy Type 1Venous Thromboembolism in Myotonic Dystrophy Type 1Myotonic Dystrophy 1Genetic: RNA extraction;Biological: Monocytes and megacaryocytes culture and RNA extraction;Biological: Haemostasis testsAssistance Publique - Hôpitaux de ParisAFM-Téléthon (Funding);Recherche Clinique Paris Descartes Necker Cochin Sainte AnneRecruiting18 YearsN/AAll69N/AFrance
43EUCTR2017-002686-21-BE28/05/201830 April 2019A 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 DystrophyDuchenne 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
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: WVE-210201
Current Sponsor code: WVE-210201
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 60-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intravenous use
Wave Life Sciences Ltd.Not Recruiting Female: no
Male: yes
32Phase 1France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom
44NCT03406780April 4, 201823 April 2019A 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.Not recruiting10 YearsN/AMale18Phase 2United States
45NCT03375255February 5, 20189 September 2019A 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.Not recruiting12 YearsN/AMale15Phase 1United States;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
46NCT03508947January 24, 201823 April 2019Safety 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.Not recruiting5 Years18 YearsMale36Phase 1United States;Belgium;Canada;France;Italy;Netherlands;United Kingdom
47EUCTR2017-001223-49-BG16/01/201820 August 2018A 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 ExtensionNonsense 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
Pharmaceutical Form: Granules in sachet
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Pharmaceutical form of the placebo: Granules in sachet
Route of administration of the placebo: Oral use
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Granules in sachet
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Pharmaceutical form of the placebo: Granules in sachet
Route of administration of the placebo: Oral use
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Granules in sachet
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Pharmaceutical form of the placebo: Granules in sachet
Route of administration of the placebo: Oral use
PTC Therapeutics, Inc.AuthorisedFemale: no
Male: yes
250Phase 3United States;Taiwan;Hong Kong;Turkey;Russian Federation;India;Mexico;Canada;Argentina;Poland;Malaysia;Brazil;Australia;Bulgaria;China;Japan;Korea, Republic of
48EUCTR2017-003568-10-GB05/01/201821 May 2018An Extension Study to Assess the Long-term Safety and Efficacy of 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) - VBP15-LTEDuchenne 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Vamorolone
CAS Number: 13209-41-1
Other descriptive name: EV substance code: SUB188638
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
Product Name: Vamorolone
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Vamorolone
CAS Number: 13209-41-1
Other descriptive name: EV substance code: SUB188638
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
Product Name: Vamorolone
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Vamorolone
CAS Number: 13209-41-1
Other descriptive name: EV substance code: SUB188638
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
Product Name: Vamorolone
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Vamorolone
CAS Number: 13209-41-1
Other descriptive name: EV substance code: SUB188638
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 40-
ReveraGen BioPharma Inc.AuthorisedFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
49EUCTR2018-000464-29-IE10 December 2018A study to compare edasalonexent with placebo in pediatric patients with Duchenne Muscular dystrophy.A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - Phase 3 Study of Edasalonexent in Duchenne Muscular DystrophyDuchenne 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
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Edasalonexent
Current Sponsor code: CAT-1004
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 100-250
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use
Catabasis Pharmaceuticals, Inc.Not AvailableFemale: no
Male: yes
126Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
50EUCTR2018-000464-29-SE7 January 2019A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy.A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMDDuchenne 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
Pharmaceutical Form: Capsule, soft
INN or Proposed INN: Edasalonexent
Current Sponsor code: CAT-1004
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number: 100-250
Pharmaceutical form of the placebo: Capsule, soft
Route of administration of the placebo: Oral use
Catabasis Pharmaceuticals, Inc.Not AvailableFemale: no
Male: yes
126Phase 3United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
51EUCTR2018-000975-34-FR20 August 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-DMDX51-001Duchenne 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
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: WVE-210201
Current Sponsor code: WVE-210201
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 60-
Wave Life Sciences Ltd.Not AvailableFemale: no
Male: yes
40Phase 1United States;France;Canada;Belgium;Netherlands;Italy;United Kingdom
52NCT03123913December 18, 201718 March 2019Study 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 RochesterRecruiting18 Years65 YearsMale20Phase 1United States
53NCT03238235December 12, 201716 September 2019Clinical 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: placeboItalfarmacoRecruiting18 Years65 YearsMale48Phase 2Italy;Netherlands
54NCT03375164December 11, 201724 June 2019Systemic Gene Delivery Clinical Trial for Duchenne Muscular DystrophySystemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophinDuchenne Muscular DystrophyDrug: rAAVrh74.MHCK7.micro-dystrophinSarepta TherapeuticsNot recruiting3 Months7 YearsMale4Phase 1/Phase 2United States
55EUCTR2016-001654-18-DE13/11/201728 February 2019Clinical 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 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
Pharmaceutical Form: Injection
INN or Proposed INN: Anti-myostatin Adnectin
Current Sponsor code: RO7239361
Other descriptive name: BMS-986089-01, anti-myostatin
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10.7-
Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe
Route of administration of the placebo: Subcutaneous use
Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL)
Product Code: RO7239361
Pharmaceutical Form: Injection
INN or Proposed INN: Anti-myostatin Adnectin
Current Sponsor code: RO7239361
Other descriptive name: BMS-986089-01, anti-myostatin
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 21.4-
Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe
Route of administration of the placebo: Subcutaneous use
Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL)
Product Code: RO7239361
Pharmaceutical Form: Injection
INN or Proposed INN: Anti-myostatin Adnectin
Current Sponsor code: RO7239361
Other descriptive name: BMS-986089-01, anti-myostatin
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe
F. Hoffmann-La Roche LtdAuthorised Female: no
Male: yes
159Phase 3United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
56NCT03333590November 6, 201720 May 2019Gene 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 FlaniganNot recruiting4 YearsN/AMale6Phase 1/Phase 2United States
57NCT03218995August 16, 201726 August 2019Study 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.Recruiting6 Months48 MonthsMale12Phase 2Belgium;France;Italy;United Kingdom;Germany
58NCT03863119July 15, 201718 March 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.Not recruitingN/AN/AMaleN/AUnited States
59NCT03039686July 6, 20174 November 2019Clinical 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 RocheNot recruiting6 Years11 YearsMale166Phase 2/Phase 3United States;Argentina;Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;Sweden;United Kingdom
60NCT03167255July 6, 20171 April 2019Extension 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)Not recruiting4 Years10 YearsMale16Phase 2United States;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
61NCT03179631July 6, 201711 November 2019Long-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 TherapeuticsRecruiting5 YearsN/AMale250Phase 3United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Jordan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Sri Lanka;Taiwan;Thailand;Turkey;Argentina;Chile
62NCT02851797June 1, 20177 October 2019Clinical 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 HealthRecruiting6 Years17 YearsMale213Phase 3United States;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;United Kingdom
63EUCTR2016-000951-29-BE29/05/201728 May 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 SkippingDuchenne 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: eteplirsen
CAS Number: 1173755-55-9
Current Sponsor code: AVI-4658
Other descriptive name: ETEPLIRSEN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Sarepta Therapeutics, IncAuthorisedFemale: no
Male: yes
12Phase 2France;Belgium;Germany;Italy
64EUCTR2016-004462-26-GB16/02/201728 February 2019A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)Duchenne muscular dystrophy (DMD)
MedDRA version: 19.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.25-
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.75-
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 2.0-
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 6.0-
ReveraGen BioPharma, Inc.Not Recruiting Female: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom
65EUCTR2016-004263-38-SE13/02/201710 October 2018An 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Current Sponsor code: 17a,21-dihydroxy-16a-methyl-pregna-1,4,9(11)-triene-3,20-dione
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.25-
Product Name: Vamorolone
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.75-
Product Name: Vamorolone
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 2.0-
Product Name: Vamorolone
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 6.0-
ReveraGen BioPharma Inc.Not RecruitingFemale: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
66EUCTR2016-004263-38-GB10/02/201728 February 2019An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)A Phase II Open-label, Multicenter Extension Study to Assess the Longterm Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD)Duchenne muscular dystrophy (DMD)
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Vamarolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.25-
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.75-
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 2.0-
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: vamorolone
CAS Number: 13209-41-1
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 6.0-
ReveraGen BioPharma, Inc.Not Recruiting Female: no
Male: yes
48Phase 2United States;Canada;Australia;Israel;United Kingdom;Sweden
67NCT03038399February 2, 201714 October 2019Long-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 GroupNot recruiting4 Years7 YearsMale46Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
68EUCTR2017-000397-10-IT5 February 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 termineDistrofia Muscolare di Duchenne (DMD)
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Givinostat (idrocloruro monoidrato)
Product Code: ITF2357
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Givinostat (idrocloruro monoidrato)
CAS Number: 732302-99-7
Current Sponsor code: ITF2357
Other descriptive name: GIVINOSTAT
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
ITALFARMACO S.p.A.Not AvailableFemale: no
Male: yes
100Phase 3France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy
69EUCTR2017-001223-49-PL15 October 2018A 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 ExtensionNonsense 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
Pharmaceutical Form: Granules in sachet
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Pharmaceutical form of the placebo: Granules in sachet
Route of administration of the placebo: Oral use
Trade Name: Translarna 250 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Granules in sachet
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Pharmaceutical form of the placebo: Granules in sachet
Route of administration of the placebo: Oral use
Trade Name: Translarna 1000 mg granules for oral suspension
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Granules in sachet
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
Pharmaceutical form of the placebo: Granules in sachet
Route of administration of the placebo: Oral use
PTC Therapeutics, Inc.Not AvailableFemale: no
Male: yes
250Phase 3United States;Taiwan;Hong Kong;Turkey;Russian Federation;India;Mexico;Canada;Argentina;Malaysia;Poland;Brazil;Australia;Bulgaria;China;Japan;Korea, Republic of
70EUCTR2017-002686-21-FR25 June 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 DystrophyDuchenne 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
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: WVE-210201
Current Sponsor code: WVE-210201
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intravenous use
Wave Life Sciences Ltd.Not AvailableFemale: no
Male: yes
32Phase 1United States;France;Czech Republic;Belgium;Spain;Ireland;Denmark;Netherlands;Italy;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
71EUCTR2017-002704-27-BE30 April 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 IIbDuchenne 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: VAMOROLONE
CAS Number: 13209-41-1
Current Sponsor code: VBP15
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1.33-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: VAMOROLONE
CAS Number: 13209-41-1
Current Sponsor code: VBP15
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 4-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Product Name: Prednisone
Pharmaceutical Form: Tablet
INN or Proposed INN: PREDNISONE
CAS Number: 53-03-2
Other descriptive name: PREDNISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
ReveraGen BioPharma, Inc.Not Available Female: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden
72EUCTR2017-002704-27-NL28 February 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 DMDDuchenne 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: VAMOROLONE
CAS Number: 13209-41-1
Current Sponsor code: VBP15
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1.33-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Product Name: Vamorolone
Product Code: VBP15
Pharmaceutical Form: Oral suspension
INN or Proposed INN: VAMOROLONE
CAS Number: 13209-41-1
Current Sponsor code: VBP15
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 4-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Product Name: Prednisone
Pharmaceutical Form: Tablet
INN or Proposed INN: PREDNISONE
CAS Number: 53-03-2
Other descriptive name: PREDNISONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
ReveraGen BioPharma, Inc.Not Available Female: no
Male: yes
120Phase 2United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden
73EUCTR2017-004279-30-SE30 April 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-EDuchenne 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IDEBENONE
CAS Number: 58186-27-9
Other descriptive name: IDEBENONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Santhera Pharmaceuticals (Switzerland) LimitedNot Available Female: no
Male: yes
266Phase 3France;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden
74EUCTR2017-004554-42-DE19 November 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 trialDuchenne 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Tamoxifen
CAS Number: 54965-24-1
Other descriptive name: TAMOXIFEN CITRATE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
University of Basel Children's Hospital, Division of NeuropediatricsNot AvailableFemale: no
Male: yes
100Phase 3Turkey;Netherlands;Germany;Switzerland
75NCT02740972December 201626 August 2019Safety 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 SolutionsNot recruiting4 Years9 YearsMale16Phase 2United States;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
76EUCTR2016-000602-10-DE17/11/201630 April 2019A 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 - SIDEROSDuchenne 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IDEBENONE
CAS Number: 58186-27-9
Other descriptive name: IDEBENONE
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Santhera Pharmaceuticals (Switzerland) LimitedAuthorised Female: no
Male: yes
266Phase 3United States;Spain;Ireland;Austria;Israel;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden
77NCT02835079November 201620 August 2018Treatment Effect of Tamoxifen on Patients With DMDTreatment Effect of Tamoxifen on Patients With DMDDuchenne Muscular DystrophyDrug: TamoxifenHadassah Medical OrganizationNot recruiting5 Years16 YearsMale19Phase 1
78NCT02927080November 201611 November 2019Study 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.Not recruiting18 YearsN/AAll95Phase 2United States;Canada;Spain
79NCT02964377November 201628 January 2019Plus 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.Not recruiting8 Years17 YearsMale15Phase 1/Phase 2United States
80NCT03236662November 201618 June 2018(-)- 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.Not recruiting18 Years70 YearsMale10Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
81NCT02907619October 13, 201620 May 2019An 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-06252616PfizerNot recruiting6 Years18 YearsMale59Phase 2United States;Canada;Italy;Japan;United Kingdom
82NCT02858908October 201611 June 2018Study 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 LimitedNot recruiting12 Years45 YearsAllPhase 2United Kingdom
83NCT02877784October 20164 November 2019Screening in Oculopharyngeal Muscular DystrophyDetermination of Accurate Screening Tools for Dysphagia in Oculopharyngeal Muscular DystrophyOculopharyngeal Muscular DystrophyOther: The Center for Neurologic Studies Bulbar Function Scale;Device: Iowa Oral Performance Instrument;Drug: Capsaicin;Procedure: Videofluoroscopic swallowing study;Other: Swallowing Related Quality of Life Questionnaire;Other: Functional Oral Intake Scale;Other: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised;Other: Eating Assessment Tool-10;Other: Communicative Effectiveness SurveyUniversity of FloridaRecruiting18 Years80 YearsAll30Early Phase 1United States
84NCT02500381September 28, 201622 July 2019Study 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.Recruiting7 Years13 YearsMale222Phase 3United States;Australia;Belgium;Bulgaria;Canada;Czechia;France;Germany;Hungary;Israel;Italy;Poland;Spain;Sweden;United Kingdom
85NCT02760277July 28, 201626 August 2019An 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)Not recruiting4 Years7 YearsMale48Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
86NCT02836418July 12, 201616 December 2017Study 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.Not recruiting16 Years25 YearsAll8Phase 1/Phase 2United States;Denmark;Italy
87EUCTR2014-002008-25-FR06/07/201630 April 2019A 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/APatients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
MedDRA version: 19.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4053
Product Code: SRP-4053
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not Available
CAS Number: 1422959-91-8
Current Sponsor code: SRP-4053
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use
Sarepta Therapeutics, Inc.Not Recruiting Female: no
Male: yes
48Phase 1;Phase 2France;Italy;United Kingdom
88NCT02878694July 20165 September 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-SalpetriereNot recruiting18 Years75 YearsBoth10Phase 2/Phase 3France
89EUCTR2016-000624-25-DK22/06/201622 May 2017An 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 DystrophyLimb 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not available yet
Current Sponsor code: ATYR1940
Other descriptive name: ATYR1940
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
aTyr Pharma, Inc.Not RecruitingFemale: yes
Male: yes
22Phase 1;Phase 2United States;Denmark;Italy
90NCT02819557June 16, 20164 February 2019Study of Ataluren in =2 to <5 Year-Old Males 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 TherapeuticsNot recruiting2 Years5 YearsMale14Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
91NCT02760264June 201618 January 2018A 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 6.0 mg/kg/day;Drug: Vamorolone 2.0 mg/kg/day;Drug: Vamorolone 0.75 mg/kg/day;Drug: Vamorolone 0.25 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 GroupNot recruiting4 Years6 YearsMale48Phase 2United States;Australia;Canada;Israel;Sweden;United Kingdom
92NCT02808585June 20168 April 2019Study 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.Not recruiting18 YearsN/AAll29Phase 2United States
93NCT02858362June 201620 May 2019PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMDA Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With SMT C1100 in Ambulatory Paediatric Male Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: ezutromidSummit TherapeuticsNot recruiting5 Years10 YearsMale40Phase 2United States;United Kingdom
94EUCTR2014-005296-81-BE02/05/20168 August 2016A clinical study to provide drisapersen (study medication) to patients with Duchenne 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 studyDuchenne 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Drisapersen
CAS Number: 1251830-50-8
Current Sponsor code: BMN-051
Other descriptive name: DRISAPERSEN SODIUM
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
BioMarin Pharmaceutical Inc.Not 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
95NCT02752048May 201611 February 2019A 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.Not recruiting5 YearsN/AMale36Phase 2Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
96EUCTR2016-000067-16-GB20/04/201610 September 2018A 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 DystrophyTreatment 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
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: Tideglusib
Other descriptive name: 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione
Concentration unit: mg milligram(s)
Concentration type: up to
Concentration number: 1000-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use
AMO Pharma Ltd.Not RecruitingFemale: yes
Male: yes
16Phase 2United Kingdom
97NCT02704325April 201612 February 2018Gene 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 FlaniganNot recruiting9 YearsN/AMale0Phase 1/Phase 2United States
98NCT02958202April 20165 February 2018Extension 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 PharmaceuticalNot recruiting5 YearsN/AMale7Phase 2Belgium;Italy;Netherlands;Sweden
99NCT02603562March 30, 201616 December 2017Evaluate 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.Not recruiting12 Years25 YearsAll8Phase 1/Phase 2United States;France;Italy
100EUCTR2015-004333-27-GB19/01/201628 February 2019A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular DystrophyA 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 DMDDuchenne Muscular Dystrophy
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: SMT C1100
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Pending
CAS Number: 945531-77-1
Current Sponsor code: SMT C1100
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 200-
Summit (Oxford) LimitedNot Recruiting Female: no
Male: yes
40Phase 2United States;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
101JPRN-UMIN0000205802016/01/152 April 2019Pilot study of tranilast for cardiomyopathy of muscular dystrophymuscular dystrophyTranilast 300mg/day for three months (oral intake)National Hospital Organization Toneyama National HospitalNational Cerebral and Cardiovascular CenterNot Recruiting20years-oldNot applicableMale and Female2Not applicableJapan
102EUCTR2015-003681-87-BE12/01/20168 August 2016A 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 dystrophyDuchenne 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
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: PS188 (company code)
CAS Number: 1802402-63-6
Current Sponsor code: PS188
Other descriptive name: PS188
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Product Name: BMN 044
Product Code: BMN 044
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: PS188 (company code)
CAS Number: 1802402-63-6
Current Sponsor code: PS188
Other descriptive name: PS188
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
BioMarin Pharmaceutical Inc.Not RecruitingFemale: no
Male: yes
50Phase 2United States;Belgium;Netherlands;Italy;Sweden
103NCT02485938January 201620 August 2018HOPE-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.Not recruiting12 YearsN/AMale25Phase 1/Phase 2United States
104EUCTR2016-000951-29-FR24 July 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 SkippingDuchenne 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: eteplirsen
CAS Number: 1173755-55-9
Current Sponsor code: AVI-4658
Other descriptive name: ETEPLIRSEN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Sarepta Therapeutics, IncNot AvailableFemale: no
Male: yes
12Phase 1France
105EUCTR2015-001910-88-DK17/12/20152 October 2017A 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 DystrophiesLimb 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not available yet
Current Sponsor code: ATYR1940
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use
aTyr Pharma, Inc.Not RecruitingFemale: yes
Male: yes
16Phase 1;Phase 2France;United States;Denmark;Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
106EUCTR2015-001955-54-NL08/12/20158 August 2016A 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 studyDuchenne 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
Pharmaceutical Form: Solution for injection
BioMarin Nederland BVNot 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
107NCT02515669December 2, 201526 August 2019Study 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 RocheNot recruiting5 Years10 YearsMale43Phase 1/Phase 2United States;Canada
108NCT02653833December 201516 December 2017The 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 CenterRecruiting18 Years45 YearsMale19Early Phase 1United States
109NCT02636686November 27, 201529 January 2018Extension 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 PharmaceuticalNot recruiting5 Years80 YearsMaleN/AUnited 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
110EUCTR2015-002530-50-ES04/11/201519 February 2018Rimeporide 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. - Rim4DMDDuchenne 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
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: N(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate
Current Sponsor code: EMD 87580
Other descriptive name: Rimeporide
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-
EspeRareNot RecruitingFemale: no
Male: yes
20Phase 1France;Spain;Italy;United Kingdom;Switzerland
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
111NCT02571205November 20151 October 2018Testosterone 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 TrustNot recruiting12 Years17 YearsMale15Phase 1/Phase 2United Kingdom
112NCT02606136November 20154 February 2019Trial 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)FibroGenNot recruiting12 YearsN/AMale22Phase 2United States
113NCT02530905October 14, 20153 December 2018Dose-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 TherapeuticsNot recruiting7 Years21 YearsMale12Phase 1United States
114NCT02592941October 201512 March 2018Deflazacort 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 ServicesNot recruiting5 YearsN/AAllN/AUnited States
115NCT02667483October 201515 April 2019Study 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.Not recruiting5 Years10 YearsMale7Phase 1/Phase 2Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
116NCT02531217September 20152 July 2018Safety, 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.Not recruiting18 Years65 YearsAll9Phase 1/Phase 2United States;Italy;Netherlands
117NCT03067831September 201518 June 2018Bone 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 ArabiaRecruiting4 Years25 YearsAll20Phase 1/Phase 2Jordan
118EUCTR2015-001912-36-NL04/08/201522 May 2017A 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not available yet
Current Sponsor code: ATYR1940
Other descriptive name: ATYR1940
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
aTyr Pharma, Inc.Not RecruitingFemale: yes
Male: yes
32Phase 1;Phase 2United States;Netherlands;Italy
119EUCTR2015-001967-38-GB03/08/201511 April 2016A 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 patientsDuchenne muscular dystrophy (DMD)
MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SMT C1100 (F5; nanosuspension)
Product Code: SMT C1100 (F5)
Pharmaceutical Form: Oral suspension
Current Sponsor code: SMT-C1100
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 100-
Product Name: SMT C1100 (F6; Powder for Oral Suspension)
Product Code: SMT C1100 (F6)
Pharmaceutical Form: Powder for oral suspension
Current Sponsor code: SMT-C1100
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 250-
Summit (Oxford) LimitedAuthorisedFemale: no
Male: yes
Phase 1United Kingdom
120NCT02420379July 1, 201511 March 2019Safety 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 TherapeuticsNot recruiting4 Years6 YearsMale33Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
121NCT02434627June 201524 October 2016Sodium Nitrate for Muscular DystrophySodium Nitrate for Muscular DystrophyBecker Muscular DystrophyDrug: Sodium NitrateCedars-Sinai Medical CenterRecruiting15 Years45 YearsMale12Phase 1United States
122NCT02484560June 20153 August 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 HospitalNot recruiting8 Years14 YearsMale10Phase 1Turkey
123NCT02525302May 201525 March 2019HT-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 TherapeuticsNot recruiting6 Years20 YearsMale10Phase 2United States
124NCT02369731April 201516 December 2017Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular DystrophyLong-Term Observational Study of Translarna Safety and Effectiveness in Usual CareMuscular Dystrophy, DuchenneDrug: TranslarnaPTC TherapeuticsTREAT-NMD;Cooperative International Neuromuscular Research Group (CINRG)RecruitingN/AN/AAll200N/AAustria;France;Germany;Israel;Sweden
125NCT02354352March 20, 201525 February 2019Therapeutic 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 CenterNot recruiting7 YearsN/AMale52Phase 3United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
126NCT02376816March 201516 December 2017Clinical 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)Not recruiting7 YearsN/AMale2Phase 1United States
127NCT01976091February 201526 August 2019Gene 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. MendellNot recruiting7 YearsN/AAll6Phase 1/Phase 2United States
128NCT02383511February 20157 September 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 TherapeuticsNot recruiting5 Years13 YearsMale12Phase 1United Kingdom
129NCT02310906January 13, 201524 June 2019Phase 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 TherapeuticsInstitut 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-TyneNot recruiting6 Years15 YearsMale39Phase 1/Phase 2United States;France;Italy;United Kingdom
130NCT02328482January 201529 July 2019Continuation 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.Not recruiting18 Years80 YearsAll9Phase 3Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
131NCT02354781January 201516 December 2017Clinical 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 TherapeuticsNot recruiting7 YearsN/AMale3Phase 1/Phase 2United States
132JPRN-UMIN0000160922014/12/312 April 2019Analysis of the effects of animal fat free diet and aspirin for prostaglandins in patients with myotonic dystrophymyotonic 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 UniversityNot RecruitingNot applicableNot applicableMale and Female10Not applicableJapan
133EUCTR2014-002072-92-GB30/12/201419 September 2016A 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'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
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: PF-06252616
Current Sponsor code: PF-06252616
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Solution for injection/infusion
Route of administration of the placebo: Intravenous use
Product Code: PF-06252616
Pharmaceutical Form: Powder for solution for infusion
INN or Proposed INN: PF-06252616
Current Sponsor code: PF-06252616
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 260-
Pharmaceutical form of the placebo: Solution for injection/infusion
Route of administration of the placebo: Intravenous use
Pfizer Inc. 235 East 42nd Street, New York, NY 10017AuthorisedFemale: no
Male: yes
105Phase 2Canada;Poland;Australia;Bulgaria;Japan;United Kingdom;Italy;United States
134EUCTR2014-003100-78-GB01/12/201430 March 2015A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet.SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet.Duchenne Muscular Dystrophy
MedDRA version: 17.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Code: SMT C1100
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Not available
CAS Number: 945531-77-1
Current Sponsor code: SMT C1100
Other descriptive name: SMT C1100
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use
Summit (Oxford) LimitedAuthorisedFemale: no
Male: yes
Phase 1bUnited Kingdom
135NCT01805024December 201411 June 2018Congenital 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 PharmaceuticalsNot recruiting5 Years16 YearsAll20Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
136NCT02251600December 201416 December 2017A 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 TherapeuticsNot recruiting4 Years16 YearsMale24Phase 1United States
137NCT02295748December 201416 December 2017An 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 TherapeuticsNot recruiting4 YearsN/AMale24Phase 1United States
138NCT02312011December 20142 July 2018A 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.Not recruiting20 Years55 YearsAll48Phase 1/Phase 2United States
139NCT02329769December 201416 December 2017Open 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 PharmaceuticalNot recruiting9 Years20 YearsMale15Phase 2Belgium;Italy;Netherlands;Sweden
140EUCTR2013-003605-26-SE26/11/20145 September 2016A 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 dystrophyDuchenne 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
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: PS188 (company code)
CAS Number: 1802402-63-6
Current Sponsor code: PS188
Other descriptive name: PS188
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
BioMarin Nederland B.V.Not RecruitingFemale: no
Male: yes
18Phase 2Belgium;Netherlands;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
141NCT02310763November 24, 201414 January 2019A 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: PlaceboPfizerNot recruiting6 Years15 YearsMale121Phase 2United States;Australia;Bulgaria;Canada;Italy;Japan;Poland;United Kingdom
142NCT02255552November 17, 201415 July 2019Study 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 TherapeuticsNot recruiting7 Years16 YearsMale109Phase 3United States
143NCT02036463November 20142 March 2015A 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 InstituteNot recruiting3 Years6 YearsMale0Phase 2United States
144NCT02286947November 201420 May 2019Safety 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 TherapeuticsNot recruiting7 Years21 YearsMale24Phase 2United States
145EUCTR2014-002210-23-DK14/10/201430 April 2019Can local botulinum toxin improve swallowing difficulties in persons with msucle disease?Can local intramuscular botulinum toxin improve dysphagia in patients with myopathic dysphagia and constriction of the cricoid muscle?Oculopharyngesl muscle dystrophy, inclusion body myositis
MedDRA version: 18.1 Level: LLT Classification code 10019897 Term: Hereditary progressive muscular dystrophy System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Trade Name: Xeomin
Product Name: Xeomin
Pharmaceutical Form: Concentrate and solvent for solution for injection
CAS Number: 93384-43-1
Other descriptive name: BOTULINUM TOXIN TYPE A
Concentration unit: U/ml unit(s)/millilitre
Concentration type: equal
Concentration number: 100-
RigshospitaletNot Recruiting Female: yes
Male: yes
15Phase 2Denmark
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
146EUCTR2013-004427-37-IT01/10/20149 March 2015Efficacy 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) - ESPP001Restrictive 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
Pharmaceutical Form: Tablet
INN or Proposed INN: ISOSORBIDE DINITRATE
CAS Number: 87-33-2
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Trade Name: IBUPROFEN 200MG
Pharmaceutical Form: Tablet
INN or Proposed INN: IBUPROFEN
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Trade Name: Isosorbide Dinitrate 40 mg
Product Name: Isosorbide Dinitrate
Pharmaceutical Form: Tablet
INN or Proposed INN: ISOSORBIDE DINITRATE
CAS Number: 87-33-2
Trade Name: Isosorbide Dinitrate 60 mg
Product Name: Isosorbide Dinitrate
Pharmaceutical Form: Tablet
INN or Proposed INN: ISOSORBIDE DINITRATE
CAS Number: 87-33-2
Trade Name: Isosorbide Dinitrate 80 mg
Product Name: Isosorbide Dinitrate
Pharmaceutical Form: Tablet
INN or Proposed INN: ISOSORBIDE DINITRATE
CAS Number: 87-33-2
PARENT PROJECT ONLUSAuthorisedFemale: no
Male: yes
188Italy
147EUCTR2014-001753-17-NL19/09/201416 February 2015A 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 DystrophiesFacioscapulohumeral 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
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: N/A
Current Sponsor code: ATYR1940
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Concentrate for solution for infusion
Route of administration of the placebo: Intravenous use
aTyr Pharma, Inc.AuthorisedFemale: yes
Male: yes
44France;United States;Netherlands;Italy
148EUCTR2014-002008-25-GB10/09/20148 January 2018A 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/APatients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: SRP-4053
Product Code: SRP-4053
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not Available
CAS Number: 1422959-91-8
Current Sponsor code: SRP-4053
Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use
Sarepta Therapeutics, Inc.AuthorisedFemale: no
Male: yes
48Phase 1;Phase 2France;United States;Italy;United Kingdom
149NCT01834066September 201419 February 2015Study 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, PuneRecruiting6 Years25 YearsBoth25Phase 1/Phase 2India
150NCT02235844September 201430 September 2019Allogeneic 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 PhDNot recruiting28 Years31 YearsMale1Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
151NCT02246478September 20146 October 2015A 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.Not recruiting5 Years15 YearsMale21Phase 1Japan
152NCT02251457August 201418 March 2019Study 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 SciencesNot recruiting18 Years100 YearsAll35Phase 1United States
153JPRN-UMIN0000148362014/05/012 April 2019A 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 UniversityNot Recruiting12years-oldNot applicableMale and Female10Not selectedJapan
154NCT02196467May 201426 August 2019Transplantation 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 LavalRecruiting16 YearsN/AMale10Phase 1/Phase 2Canada
155NCT02167217April 17, 201411 June 2018Historically 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, FLNot recruiting1 Month30 MonthsMale24Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
156NCT02090959March 31, 201417 June 2019An 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 TherapeuticsNot recruiting7 Years18 YearsMale219Phase 3Switzerland;Turkey;United Kingdom;United States;Czech Republic;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Australia;Belgium;Brazil
157NCT02015481February 201416 December 2017Safety 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.Not recruiting18 Years80 YearsAll25Phase 2United States;Canada;Israel
158NCT01890798January 201419 February 2015Drisapersen 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: DrisapersenGlaxoSmithKlineNot recruiting5 YearsN/AMale0Phase 3
159NCT02056808November 201319 February 2015A 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.Not recruiting5 Years11 YearsMale12Phase 1United Kingdom
160NCT02285673November 201319 February 2015Efficacy 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 UniversityRecruiting7 Years20 YearsMale10Phase 1/Phase 2Turkey
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
161NCT01970735October 201319 February 2015Clinical, 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 NiceRecruiting18 Years75 YearsBoth100N/AFrance
162NCT01978366October 201326 August 2019Open 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-100Akashi TherapeuticsNot recruiting6 Years20 YearsMale17Phase 2United States
163NCT01995032October 201311 June 2018L-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, SwitzerlandNot recruiting78 Months10 YearsAll47Phase 3Switzerland
164NCT02847975October 201320 August 2018Sodium Nitrate to Improve Blood FlowSodium Nitrate to Improve Blood FlowBecker Muscular DystrophyDietary Supplement: Sodium nitrateCedars-Sinai Medical CenterNot recruiting15 Years55 YearsMale11Phase 1United States
165EUCTR2013-001194-25-GB25/09/20133 April 2017A 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 DystrophyDuchenne 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Trade Name: Cialis 5 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Trade Name: Cialis 10 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Trade Name: Cialis 20 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Eli Lilly and CompanyNot 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_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
166EUCTR2013-001732-21-FR06/09/201319 February 2018A 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 - MyometMyotonic dystrophy type 1 (DM1) also known as Steinert disease;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Trade Name: metformin 500mg
Pharmaceutical Form: Tablet
INN or Proposed INN: METFORMIN
CAS Number: 657-24-9
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 500-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Centre d'Etude des Cellules Souches (CECS)Not RecruitingFemale: yes
Male: yes
30Phase 2France
167NCT01865084September 201316 December 2017A 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 CompanyNot recruiting7 Years14 YearsMale331Phase 3United States;Argentina;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Puerto Rico;Russian Federation;Spain;Taiwan;Turkey;United Kingdom
168NCT02147639September 201319 February 2015Effects 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 CenterRecruiting15 Years45 YearsMale20Phase 2/Phase 3United States
169EUCTR2011-005042-35-GB23/08/201314 March 2016A 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 dystrophyDuchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping
MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: BMN 053
Product Code: BMN 053
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: PS524
Current Sponsor code: PS524
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
BioMarin Pharmaceutical Inc.AuthorisedFemale: no
Male: yes
45Phase 1/2United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan
170EUCTR2013-002115-99-GB23/08/20133 April 2017A 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 DystrophyDuchenne 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
CAS Number: 945531-77-1
Current Sponsor code: SMT C1100
Concentration unit: mg/g milligram(s)/gram
Concentration type: equal
Concentration number: 200-
Summit Corporation plcNot RecruitingFemale: no
Male: yes
Phase 1United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
171EUCTR2013-001194-25-IT09/08/201314 March 2016A 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 DystrophyDuchenne 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
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Trade Name: Cialis 5 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Trade Name: Cialis 10 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Trade Name: Cialis 20 mg film-coated tablets
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: TADALAFIL
CAS Number: 171596-29-5
Current Sponsor code: LY450190
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 20-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use
Eli Lilly and CompanyNot RecruitingFemale: no
Male: yes
306Phase 3United States;Taiwan;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of
172NCT01918384August 201314 September 2015Phase 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;NobelpharmaNot recruiting4 YearsN/AMale21Phase 2Japan
173NCT01921374August 201315 February 2016Mother-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 AndersenNot recruiting25 Years65 YearsFemale60N/ABrazil
174NCT01957059June 201316 December 2017A 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 PharmaceuticalNot recruiting5 Years18 YearsMale9Phase 1/Phase 2Belgium;France;Italy;Netherlands;United Kingdom
175NCT02018731June 201323 May 2016L-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, SwitzerlandNot recruiting18 YearsN/ABoth20Phase 2Switzerland
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
176NCT01803412May 1, 201325 June 2018A 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 PharmaceuticalNot recruiting5 YearsN/AMale53Phase 3United States;Canada
177NCT01761292May 20135 March 2018A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant 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: GivinostatItalfarmacoNot recruiting7 Years11 YearsMale20Phase 1/Phase 2Italy
178NCT01847573May 201326 August 2019Safety, 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-100Akashi TherapeuticsNot recruiting6 Years20 YearsMale17Phase 1/Phase 2United States
179NCT01856868May 201328 January 2019Use 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.Not recruiting18 Years60 YearsMale7Phase 1/Phase 2United States
180NCT02814110March 1, 201316 December 2017Efficacy 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 of Muscle Strength in Patients With Muscular DystrophyDrug: Granulocyte colony-stimulating factor (Filgrastim)Medical University of BialystokRecruiting5 Years15 YearsAll27Phase 1Poland
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
181NCT01826422March 20138 February 2016Effect 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 in 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 Comparator: Sunflower oilCoordinación de Investigación en Salud, MexicoInstituto Nacional de RehabilitacionRecruiting6 Years18 YearsMale70Phase 2Mexico
182NCT01826487March 201315 April 2019Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular DystrophyA Phase 3 Efficacy and Safety 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: Ataluren;Drug: PlaceboPTC TherapeuticsNot recruiting7 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
183EUCTR2012-002566-12-IT04/02/201319 February 2018A 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 - DSCDuchenne 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
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Givinostat
CAS Number: 732302-99-7
Current Sponsor code: ITF2357
Other descriptive name: givinostat
Concentration unit: mg milligram(s)
Concentration type: up to
Concentration number: 100-
Product Name: Givinostat
Product Code: ITF2357
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Givinostat
CAS Number: 732302-99-7
Current Sponsor code: ITF2357
Other descriptive name: Givinostat
Concentration unit: mg milligram(s)
Concentration type: up to
Concentration number: 100-
ITALFARMACONot RecruitingFemale: no
Male: yes
20Phase 2Italy
184NCT01603407January 201329 April 2019Finding 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)Not recruiting4 Years7 YearsMale196Phase 3United States;Canada;Germany;Italy;United Kingdom
185NCT01826474January 201316 December 2017Phase 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 PharmaceuticalNot recruiting5 Years18 YearsMale15Phase 1/Phase 2Belgium;France;Italy;Netherlands;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
186EUCTR2011-005040-10-BE03/12/20128 August 2016A 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 dystrophyDuchenne 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PS220
Current Sponsor code: PS220
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
BioMarin Nederland B.V.Not RecruitingFemale: no
Male: yes
45Phase 1;Phase 2Belgium;Italy;United Kingdom
187JPRN-UMIN0000093072012/11/082 April 2019Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophyDuchenne muscular dystrophyoral adm,inistration of aspirinKobe University Graduate School of MedicineNot Recruiting5years-oldNot applicableMale6Phase 1,2Japan
188EUCTR2011-004853-18-GB21/08/201228 February 2019Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation DystrophinopathyNonsense 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 Level: 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: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
PTC Therapeutics, Inc.Not Recruiting Female: no
Male: yes
96Phase 3France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
189EUCTR2011-004853-18-BE27/04/201212 March 2018Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophyAn Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation DystrophinopathyNonsense mutation dystrophinopathy
MedDRA version: 16.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 16.1 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: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Product Name: ataluren
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ataluren
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
PTC Therapeutics, IncNot RecruitingFemale: no
Male: yes
96Phase 3Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden
190NCT01580501March 201219 February 2015PDE Inhibitors in DMD Study (Acute Dosing Study)Functional Muscle Ischemia and PDE5 Inhibition in Duchenne Muscular Dystrophy: Acute Dosing StudyDuchenne Muscular DystrophyDrug: Tadalafil and SildenafilCedars-Sinai Medical CenterNot recruiting7 Years15 YearsMale12Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
191NCT01618331March 201219 February 2015Protein Supplementation and Exercise in Patients With FSH Muscular Dystrophy- a Randomized Placebo Controlled StudyThe Effect of Protein Supplementation Doing Regular Exercise in Patients With Facioscapulohumeral Muscular Dystrophy - a Blinded RCT StudyFacioscapulohumeral Muscle DystrophyBehavioral: Regular exercise;Dietary Supplement: Protein-carbohydrate supplementationGrete Andersen, MDThe Augustinus Foundation, Denmark.;Aase and Ejnar Danielsens Foundation;The Danish Rheumatism Association;AP Moeller FoundationNot recruiting18 Years65 YearsBoth42N/ADenmark
192NCT02207283March 20128 August 2016PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular DystrophyPDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular DystrophyBecker Muscular DystrophyDrug: Tadalafil;Drug: PlaceboCedars-Sinai Medical CenterNot recruiting15 Years55 YearsMale12Phase 4
193NCT03076814March 201230 October 2017Functional Muscle Ischemia With Tadalafil Treatment in Becker Muscular DystrophyBecker Muscular DystrophyDrug: Tadalafil;Other: PlaceboCedars-Sinai Medical CenterNot recruiting15 Years55 YearsMale0N/A
194NCT01540409February 27, 201215 July 2019Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular DystrophyOpen-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201Duchenne Muscular Dystrophy (DMD)Drug: AVI-4658 (Eteplirsen)Sarepta TherapeuticsNot recruiting7 Years13 YearsMale12Phase 2United States
195NCT01648634February 13, 201211 June 2018Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Examine the Effect of Nebivolol, a Beta-Blockade Drug, for the Prevention of Ventricular Systolic Dysfunction in Patients With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy;Cardiomyopathy;Heart FailureDrug: Nebivolol;Drug: PlaceboAssistance Publique - Hôpitaux de ParisAssociation Française contre les Myopathies (AFM), ParisNot recruiting10 Years15 YearsMale51Phase 3France
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
196NCT01521546February 201219 October 2017Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular DystrophyEarly Treatment With Aldosterone Antagonism Attenuates Cardiomyopathy in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: eplerenone;Drug: placeboSubha RamanBallou SkiesNot recruiting7 YearsN/AMale42N/AUnited States
197NCT01540604February 201219 February 2015CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic CarriersAn Open-label, Un-controlled, Single-centre Trial Investigating the Efficacy and Safety of CRD007 in Children With Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or Children Being Symptomatic Carriers for DMD or BMDDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: CRD007Cardoz ABNot recruiting2 Years11 YearsBothPhase 2Sweden
198NCT01388764January 201219 February 2015Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on CorticosteroidsPilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on CorticosteroidsDystrophinopathy;Duchenne Muscular Dystrophy;Becker's Muscular DystrophyDrug: L-arginineMassachusetts General HospitalNot recruiting7 Years11 YearsMale7Phase 1United States
199NCT01519349January 201216 December 2017Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body MyositisPhase I Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis.Becker Muscular Dystrophy;Sporadic Inclusion Body MyositisBiological: rAAV1.CMV.huFollistatin344Nationwide Children's HospitalParent Project Muscular Dystrophy;The Myositis Association (Grant Sponsor)Not recruiting18 YearsN/AAll15Phase 1United States
200NCT02516085January 201217 August 2015Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and MetforminDuchenne Muscular DystrophyDrug: Metformin;Drug: L-ArginineUniversity Hospital, Basel, SwitzerlandNot recruiting7 Years10 YearsBoth5Phase 1
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
201EUCTR2011-004667-76-SE19/12/201113 May 2013An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD.An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriersDuchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD
MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1 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: pemirolast
Product Code: CRD007
Pharmaceutical Form: Tablet
INN or Proposed INN: PEMIROLAST
CAS Number: 69372-19-6
Current Sponsor code: CRD007
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Cardoz ABNot RecruitingFemale: yes
Male: yes
Sweden
202NCT01350154November 201119 February 2015Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy PatientsDoes Modulation of the nNOS System in Patients With Muscular Dystrophy and Defect nNOS Signalling Affect Cardiac, Muscular or Cognitive Function?Becker Muscular DystrophyDrug: Sildenafil;Drug: PlaceboRigshospitalet, DenmarkGlostrup University Hospital, CopenhagenNot recruiting18 Years80 YearsMale17Phase 2Denmark
203NCT01462292October 26, 201116 December 2017A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)An Exploratory Study to Assess Two Doses of GSK2402968 in the Treatment of Ambulant Boys With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968 3mg/kg/week;Drug: GSK2402968 6 mg/kg/week;Drug: Placebo to match GSK2402968 3 mg/kg/week;Drug: Placebo to match GSK2402968 6 mg/kg/weekGlaxoSmithKlineNot recruiting5 YearsN/AMale51Phase 2United States
204EUCTR2011-001266-17-GB03/10/201128 February 2019An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - N/ADuchenne 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
Pharmaceutical Form: Injection
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
GlaxoSmithKline Research and Development LTDNot Recruiting Female: no
Male: yes
200Phase 3Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
205NCT01478022October 201110 September 2018To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20Open, Randomized, 3 Period Cross-over Design, in Healthy Volunteers to Compare the Pharmacokinetics Profiles of 3 Treatments: ISO 20, IBU 200 and IBU Plus ISO Combinations (200 + 20) Administered Per os as Single DosesDuchenne Muscular Dystrophy (DMD)Drug: Isosorbide Dinitrate;Drug: Ibuprofen;Other: Ibuprofen and Isosorbide Dinitrate combinationParent Project, ItalyNot recruiting18 Years27 YearsAll12Phase 1Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
206NCT01610440October 201119 February 2015Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular DystrophyPhase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: human umbilical cord mesenchymal stem cellsShenzhen Beike Bio-Technology Co., Ltd.The Second Affiliated Hospital of Kunming Medical UniversityRecruiting5 Years12 YearsBoth15Phase 1/Phase 2China
207NCT01480245September 201116 December 2017Open Label Study of GSK2402968 in Subjects With Duchenne Muscular DystrophyAn Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968GlaxoSmithKlineNot recruiting5 YearsN/AMale233Phase 3Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czech Republic;Denmark;France;Germany;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom
208EUCTR2011-001266-17-BE08/08/201121 August 2017An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular DystrophyAn open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - N/ADuchenne Muscular Dystrophy
MedDRA version: 16.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: GSK2402968
Product Code: GSK2402968
Pharmaceutical Form: Injection
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
GlaxoSmithKline Research and Development LTDNot RecruitingFemale: no
Male: yes
200Phase 3Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Poland;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of
209NCT01422200August 201119 February 2015Flu Vaccine Study in Neuromuscular Patients 2011Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular DiseasesDuchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular DystrophyBiological: 2011-2012 seasonal flu vaccineChildren's Hospital Medical Center, CincinnatiRecruiting3 Years35 YearsBoth30N/AUnited States
210NCT01645098August 201119 October 2017Sedation During Muscle Biopsy in Patients With Duchenne Muscular DystrophySedation During Muscle Biopsy in Patients With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Ketamine;Drug: DexmedetomidineNationwide Children's HospitalNot recruitingN/AN/AMale53N/AUnited States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
211NCT01396239July 201120 May 2019Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy PatientsA Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658(Eteplirsen),in the Treatment of Ambulant Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: AVI-4658 (Eteplirsen);Other: PlaceboSarepta TherapeuticsNot recruiting7 Years13 YearsMale12Phase 2United States
212NCT01406873June 201111 June 2018Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1)Myotonic DystrophyDrug: Mexiletine;Drug: PlaceboUniversity of RochesterNot recruiting18 Years80 YearsAll42Phase 2United States
213NCT01359670May 201111 June 2018Tadalafil and Sildenafil for Duchenne Muscular DystrophyFunctional Muscle Ischemia and PDE5A Inhibition in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Tadalafil;Drug: SildenafilCedars-Sinai Medical CenterParent Project Muscular DystrophyNot recruiting7 Years15 YearsMale30N/AUnited States
214NCT01335295March 201119 February 2015Safety Study of Flavocoxid in Duchenne Muscular DystrophyOpen Pilot Trial to Test the Safety and Tolerability of Flavocoxid in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: FlavocoxidUniversity of MessinaNot recruiting4 Years16 YearsMale20Phase 1Italy
215EUCTR2011-000176-33-IT14/02/201119 March 2012Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - NDCell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - NDDuchenne Muscolar Dystrophy
MedDRA version: 9.1 Level: PT Classification code 10013801
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Tacrolimus
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 1-
Product Name: hMABs
Pharmaceutical Form: Suspension for injection
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Concentration unit: % percent
Concentration type: equal
Concentration number: 1-
Product Name: hMABs
Pharmaceutical Form: Suspension for injection
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Concentration unit: % percent
Concentration type: equal
Concentration number: 1-
Product Name: hMABs
Pharmaceutical Form: Suspension for injection
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Concentration unit: % percent
Concentration type: equal
Concentration number: 1-
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABORAuthorisedFemale: no
Male: yes
Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
216NCT01183767December 30, 201010 December 2018Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular DystrophySunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Epigallocatechin-Gallate;Drug: PlaceboCharite University, Berlin, GermanyNot recruiting5 Years10 YearsAll33Phase 2/Phase 3Germany
217NCT02245711December 20105 November 2018Cell Therapy in Limb Girdle Muscular DystrophyIntrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular DystrophyLimb Girdle Muscular DystrophyBiological: Stem CellNeurogen Brain and Spine InstituteNot recruiting15 Years60 YearsAll0Phase 1India
218NCT01247207November 30, 201014 October 2019Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation DystrophinopathyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsRecruitingN/AN/AMale160Phase 3United States;Canada
219NCT01207908November 201019 February 2015Safety and Efficacy Study of IGF-1 in Duchenne Muscular DystrophyIGF-1 Therapy and Muscle Function in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: IGF-1;Other: Standard steroid treatmentChildren's Hospital Medical Center, CincinnatiTercica- Subsidiary of Ipsen;Charley's FundNot recruiting5 YearsN/AMale40Phase 1/Phase 2United States
220NCT01239758October 201019 February 2015Extension Study of ACE-031 in Subjects With Duchenne Muscular DystrophyAn Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: ACE-031 (Extension of cohort 1 from core study, A031-03);Biological: ACE-031 (Extension of cohort 2 from core study, A031-03);Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)Acceleron Pharma, Inc.Not recruiting4 YearsN/AMale11Phase 2Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
221NCT01153932September 201019 February 2015Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular DystrophyA Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: GSK2402968;Drug: matched placeboGlaxoSmithKlineNot recruiting5 YearsN/AMale53Phase 2Australia;Belgium;France;Germany;Israel;Netherlands;Spain;Turkey;United Kingdom
222NCT01168908September 201011 June 2018Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular DystrophyPhase 2 Clinical Trial of Sildenafil for Cardiac Dysfunction in Duchenne Muscular Dystrophy and Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: SildenafilHugo W. Moser Research Institute at Kennedy Krieger, Inc.Johns Hopkins UniversityNot recruiting18 Years50 YearsMale20Phase 2United States
223EUCTR2010-018412-32-DE05/08/20103 February 2014A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophyA phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophyDuchenne Muscular Dystrophy
MedDRA version: 14.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Code: GSK2402968
Pharmaceutical Form: Injection
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
GlaxoSmithKline Research and Development LTDNot RecruitingFemale: no
Male: yes
54Phase 2France;Spain;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom
224NCT01128855July 12, 201016 December 2017A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD SubjectsA Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular DystrophyMuscular DystrophiesDrug: 3 mg/kg GSK2402968;Drug: 6 mg/kg GSK2402968;Drug: 9 mg/kg GSK2402968;Drug: 12 mg/kg GSK2402968;Other: PlaceboGlaxoSmithKlineNot recruiting9 YearsN/AMale20Phase 1United States;France
225EUCTR2009-016482-28-DE25/06/201010 October 2018SUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular DystrophySUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular Dystrophy - SUNIMUDDuchenne Muscular Dystrophy;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Product Name: Sunphenon EGCG
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Sunphenon
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use
Charite Universitätsmedizin BerlinNot RecruitingFemale: no
Male: yes
40Phase 2Germany
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
226EUCTR2009-017649-67-IE22/06/201019 March 2012Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMDBone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMDBone Health in Duchenne Muscular Dystrophy- a randomised controlled study of Risedronate useTrade Name: Actonel Once a week 5mg film coated tablets
Product Name: Risedronate sodium
Product Code: Risedronate
Pharmaceutical Form: Film-coated tablet
Trade Name: Calcichew D3 Forte
Product Name: Calcichew D3 Forte
Product Code: Calcichew D3 Forte
Pharmaceutical Form: Chewable tablet
The Central Remedial Clinic and The Children's University HospitalAuthorisedFemale: no
Male: yes
70Ireland
227NCT01596803June 201019 February 2015Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD)Effects of Antioxidants Supplementation on Muscular Function of Patients Affected by Facioscapulohumeral Dystrophy (FSHD)Facioscapulohumeral Muscular DystrophyProcedure: Taking of blood;Dietary Supplement: needle biopsy of the vastus lateralis muscle;Dietary Supplement: Vit C Vit E Zn Se;Dietary Supplement: Placebo Vit E Placebo Vit C Zn SeUniversity Hospital, MontpellierHospital Clinical Research Project 2010;Association Amis FSH France;FSH Dutch Fondation The NetherlandRecruiting18 Years60 YearsBoth54N/AFrance
228EUCTR2009-013762-63-NL16/04/20105 August 2013A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyA phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophyDuchenne Muscular DystrophyProduct Name: PRO044
Product Code: PRO044
Pharmaceutical Form: Solution for injection
INN or Proposed INN: h44AON188
Current Sponsor code: PRO044
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 92-
Prosensa Therapeutics B.VNot RecruitingFemale: no
Male: yes
18Phase 1/2aBelgium;Netherlands;Italy;Sweden
229NCT01099761April 201016 December 2017Study of ACE-031 in Subjects With Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: ACE-031 0.5 mg/kg q4wk;Biological: ACE-031 1.0 mg/kg q2wk;Other: PlaceboAcceleron Pharma, Inc.Not recruiting4 YearsN/AMale24Phase 2Canada
230NCT01126697February 201025 June 2018Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular DystrophiesPITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular DystrophiesDuchenne Muscular Dystrophy;Becker Muscular Dystrophy;Limb Girdle Muscular DystrophyDrug: Coenzyme Q10 and LisinoprilCooperative International Neuromuscular Research GroupUnited States Department of DefenseNot recruiting8 YearsN/AAll63Phase 2/Phase 3United States;Japan;Canada
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
231EUCTR2009-013169-24-GB19/01/201019 March 2012A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMDA Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMDNonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
MedDRA version: 12.0 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
MedDRA version: 12.0 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy
Product Name: Ataluren 125 mg
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Product Name: Ataluren 250 mg
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Product Name: Ataluren 1000 mg
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
PTC Therapeutics IncNot RecruitingFemale: no
Male: yes
30Phase 2aUnited Kingdom
232NCT01070511January 201019 February 2015Tadalafil in Becker Muscular DystrophyFunctional Muscle Ischemia and PDE5A Inhibition in Becker Muscular DystrophyBecker Muscular DystrophyDrug: Tadalafil;Drug: PlaceboCedars-Sinai Medical CenterMuscular Dystrophy AssociationNot recruiting18 Years55 YearsMale48Phase 4United States
233EUCTR2010-020047-12-FR2 October 2017Effet d’un traitement préventif par Nebivolol chez les enfants atteints de dystrophie de DuchenneEtude de l'effet d'un traitement préventif par Nebivolol sur l'apparition et la progression de la dysfonction cardiaque chez les enfants atteints de dystrophie de Duchenne - NEBIDYSEnfants atteints de dystrophie de Duchenne
MedDRA version: 13.1 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: Temerit
Product Name: Temerit
Pharmaceutical Form: Tablet
INN or Proposed INN: Nébivolol
CAS Number: 118457-15-1
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)Not AvailableFemale: no
Male: yes
Phase 3France
234EUCTR2010-020069-26-DE21 October 2013A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy.A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy. - N/ADuchenne muscular dystrophy
MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Product Code: GSK2402968
Pharmaceutical Form: Injection
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Injection
Route of administration of the placebo: Subcutaneous use
GlaxoSmithKline Research and Development LTDNot RecruitingFemale: no
Male: yes
180Phase 3Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan
235EUCTR2010-024566-22-FR2 October 2017A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/AA double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/ADuchenne Muscular Dystrophy
MedDRA version: 12.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Product Name: GSK2402968
Product Code: GSK2402968
Pharmaceutical Form: Injection*
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
GlaxoSmithKline Research and Development LTDNot AvailableFemale: no
Male: yes
32Phase 1France
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
236EUCTR2010-024566-22-Outside-EU/EEA20 March 2012A clinical study to investigate the pharmacokinetics safety, and tolerability of a single dose of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy (GSK Study Number DMD114118)A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/ADuchenne Muscular Dystrophy
MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Product Name: GSK2402968
Product Code: GSK2402968
Pharmaceutical Form: Injection
Current Sponsor code: GSK2402968
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
GlaxoSmithKline Research and Development LTDNot AvailableFemale: no
Male: yes
32United States
237NCT01009294November 30, 200915 April 2019Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: Ataluren (PTC124)PTC TherapeuticsGenzyme, a Sanofi CompanyNot recruiting7 YearsN/AMale6Phase 2United States;United Kingdom
238EUCTR2009-012037-30-BE14/07/200912 May 2014Hereditary neuromuscular diseaseA Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOSDuchenne Muscular Dystrophy
MedDRA version: 17.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Idebenone
Product Code: SNT-MC17
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IDEBENONE
CAS Number: 58186279
Current Sponsor code: SNT-MC17
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Santhera Pharmaceuticals (Switzerland) LimitedAuthorisedFemale: no
Male: yes
266Phase 3France;United States;Spain;Belgium;Austria;Netherlands;Germany;Italy;Switzerland;Sweden
239NCT02432885June 200911 May 2015Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy TrialMyocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor TherapyMyocardial Fibrosis;Muscular DystrophiesDrug: EnalaprilInCor Heart InstituteFederal University of Minas Gerais;University of Sao PauloNot recruiting6 YearsN/ABoth76Phase 3
240EUCTR2008-007648-32-BE14/04/200919 March 2012A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular DystrophyDuchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy
Product Name: ATALUREN
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Product Name: ATALUREN
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Product Name: ATALUREN
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: ATALUREN
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1000-
PTC Therapeutics, Inc.Not RecruitingFemale: no
Male: yes
174Phase 2bUnited Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
241NCT01982695March 200916 December 2017Cardiomyopathy in DMD: Lisinopril vs. LosartanCompare Efficacy of the Angiotensin Converting Enzyme Inhibitor (ACEi) Lisinopril With Angiotensin II Receptor Antagonist Losartan (ARB) for the Cardiomyopathy of Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD);CardiomyopathyDrug: Losartan;Drug: LisinoprilNationwide Children's HospitalBoston Children’s Hospital;University of California, Davis;Unverisity of Kansas Medical Center;University of Minnesota - Clinical and Translational Science Institute;St. Louis Children's HospitalNot recruitingN/AN/AMale23N/AUnited States
242NCT00844597January 200919 October 2017Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsClinical Study to Assess the Safety fo AVI-4658 in Subjects With Duchenne Muscular Dystrophy Due to a Frame-shift Mutation Amenable to Correction by Skipping Exon 51.Duchenne Muscular DystrophyDrug: AVI-4658 for InjectionSarepta TherapeuticsBritish Medical Research CouncilNot recruiting5 Years15 YearsMale19Phase 1/Phase 2United Kingdom
243NCT00847379January 200916 December 2017Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)A Phase 2b Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: Ataluren (PTC124)PTC TherapeuticsGenzyme, a Sanofi CompanyNot recruitingN/AN/AMale173Phase 2/Phase 3United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
244NCT02241434January 20095 November 2018Stem Cell Therapy in Duchenne Muscular DystrophyThe Role of Autologous Bone Marrow Mononuclear Cell Therapy in Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: Stem CellNeurogen Brain and Spine InstituteNot recruiting3 Years25 YearsAll0Phase 1India
245NCT02241928January 20095 November 2018Stem Cell Therapy in Muscular DystrophyRole of Autologous Mononuclear Cell Therapy in Muscular DystrophyMuscular DystrophyBiological: Stem CellNeurogen Brain and Spine InstituteNot recruiting6 Months60 YearsAll0Phase 1India
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
246EUCTR2009-009871-36-DE25 January 2016Study within children with Duchenne Muscular DystrophyEffect and Safety of preventive Treatment with ACE-Inhibitor and Beta-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy - DMD-KardioTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]Trade Name: EnalHexal®, 5 mg
Pharmaceutical Form: Tablet
INN or Proposed INN: ENALAPRIL
CAS Number: 75847733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Trade Name: EnaHexal®, 10mg
Pharmaceutical Form: Tablet
INN or Proposed INN: ENALAPRIL
CAS Number: 75847733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Trade Name: MetoHEXAL® Succ ® 23,75 mg Retardtabletten
Pharmaceutical Form: Tablet
INN or Proposed INN: METOPROLOL SUCCINATE
CAS Number: 98418474
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 23.75-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Trade Name: MetoHEXAL® Succ ® 47,5 mg Retardtabletten
Pharmaceutical Form: Tablet
INN or Proposed INN: METOPROLOL SUCCINATE
CAS Number: 98418474
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 47.5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Trade Name: MetoHEXAL® Succ ® 95 mg Retardtabletten
Pharmaceutical Form: Tablet
INN or Proposed INN: METOPROLOL SUCCINATE
CAS Number: 98418474
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 95-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Friedrich- Alexander- Universität Erlangen NürnbergNot RecruitingFemale: no
Male: yes
Germany
247EUCTR2007-004695-39-GB05/12/200826 June 2012Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsDose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) PatientsDuchenne Muscular Dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Product Name: AVI-4658
Product Code: AVI-4658
Pharmaceutical Form: Concentrate for solution for injection
CAS Number: 1173755-55-9
Current Sponsor code: AVI-4658
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 100-
AVI BioPharma, Inc.Not RecruitingFemale: no
Male: yes
United Kingdom
248EUCTR2008-007236-18-IT01/12/20085 August 2013Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of Cardiac Magnetic Resonance study. - NDEffects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of Cardiac Magnetic Resonance study. - NDDuchenne and Becher muscular dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Trade Name: DILATREND
Pharmaceutical Form: Tablet
INN or Proposed INN: Carvedilol
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 6.25-
Trade Name: TRIATEC
Pharmaceutical Form: Tablet
INN or Proposed INN: Ramipril
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 2.5-
POLICLINICO UNIVERSITARIO AGOSTINO GEMELLINot RecruitingFemale: no
Male: yes
Italy
249NCT00819845December 20088 February 2016Ramipril Versus Carvedilol in Duchenne and Becker PatientsEffects of Cardioprotective Therapy, Carvedilol vs Ramipril, in Patients Affected by Duchenne and Becker Muscular Dystrophy. Clinical Significance and Prognostic Value of Cardiac Magnetic Resonance Study.Duchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: carvedilol;Drug: ramiprilCatholic University, ItalyRecruiting2 Years45 YearsMale194Phase 4Italy
250NCT02050776December 20085 November 2018Stem Cell Therapy in Limb Girdle Muscular DystrophyThe Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year StudyLimb Girdle Muscular DystrophyBiological: Autologous bone marrow mononuclear cell transplantationNeurogen Brain and Spine InstituteNot recruiting15 Years60 YearsAll0Phase 1India
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
251NCT00758225September 200819 February 2015Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001Duchenne Muscular DystrophyDrug: IdebenoneSanthera PharmaceuticalsNot recruitingN/AN/AMale21Phase 2Belgium
252EUCTR2007-007752-34-BE14/08/200819 March 2012A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular DystrophyA Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular DystrophyDuchennes Muscular Dystrophy
MedDRA version: 12.0 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Product Name: Idebenone
Product Code: SNT-MC17
Pharmaceutical Form: Tablet
INN or Proposed INN: Idebenone
Current Sponsor code: SNT-MC17
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Santhera Pharmaceuticals (Switzerland) LtdNot RecruitingFemale: no
Male: yes
Phase 2Belgium
253NCT00759876July 31, 200815 April 2019Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)A Phase 2a Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: AtalurenPTC TherapeuticsGenzyme, a Sanofi CompanyNot recruitingN/AN/AMale36Phase 2United States
254NCT01421992June 200819 February 2015Methylphenidate in Myotonic Dystrophy Type 1Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1Dystrophia Myotonica 1Drug: Methylphenidate;Drug: PlaceboLaval UniversityNot recruiting18 Years65 YearsBoth28Phase 2/Phase 3Canada
255EUCTR2007-005478-29-FR17/03/200819 March 2012A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyA Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular DystrophyDuchenne Muscular Dystrophy, Becker Muscular Dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy
Product Name: PTC124
Product Code: PTC124
Pharmaceutical Form: Powder for oral suspension
CAS Number: 775304-57-9
Current Sponsor code: PTC124
Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125, 250, 1000-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use
PTC Therapeutics, Inc.AuthorisedFemale: no
Male: yes
165Phase 2bUnited Kingdom;Germany;Belgium;France;Spain;Italy;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
256EUCTR2007-005808-41-ES25/02/200819 March 2012Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con Calcifediol Multicentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathyEstudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con Calcifediol Multicentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathyPortadores sintomaticos de una mutación en el gen disferlina. Symptomatic carriers of a mutation in the dysferlin gene
MedDRA version: 9.1 Level: LLT Classification code 10028356 Term: Muscular dystrophy
Trade Name: Hidroferol®
Product Name: Calcifediol
Pharmaceutical Form: Oral liquid
INN or Proposed INN: CALCIFEDIOL
CAS Number: 19356-17-3
Other descriptive name: HIDROFEROL
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0,266-
Institut de Recerca de l'Hospital de la Santa Creu i Sant PauAuthorisedFemale: yes
Male: yes
Spain
257NCT00592553February 200816 December 2017Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD)A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular DystrophyDuchenne Muscular Dystrophy;Becker Muscular DystrophyDrug: PTC124 High Dose;Drug: PTC124 Low Dose;Drug: PlaceboPTC TherapeuticsNot recruiting5 YearsN/AMale174Phase 2United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom
258EUCTR2007-004819-54-BE22/01/20088 August 2016A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne DiseaseA phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administrationDuchenne 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: Drisapersen
Product Code: PRO051
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PRO051
Current Sponsor code: PRO051
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Product Name: Drisapersen
Product Code: PRO051
Pharmaceutical Form: Solution for injection
INN or Proposed INN: PRO051
Current Sponsor code: PRO051
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 200-
Prosensa Therapeutics BVNot RecruitingFemale: no
Male: yes
12Phase 1;Phase 2Belgium;Netherlands;Sweden
259NCT00577577December 200719 February 2015Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1A Placebo Controlled, Randomized, Double-Blind Phase II Clinical Trial to Evaluate Tolerability, Safety and Efficacy Endpoints After Administration of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) for 24 Weeks in Adults With Myotonic Dystrophy Type 1Myotonic Dystrophy Type 1Drug: rhIGF-I/rhIGFBP-3;Drug: placeboInsmed IncorporatedMuscular Dystrophy AssociationNot recruiting21 Years65 YearsBoth60Phase 2United States
260NCT00606775December 200719 February 2015The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular DystrophyCarvedilol for the Prevention of Minor Cardiac Damage and Cardiac Function in Duchenne Muscular DystrophyDuchenne Muscular Dystrophy;CardiomyopathiesDrug: CarvedilolSuzuka HospitalNagoya UniversityRecruiting8 Years45 YearsMale60Phase 4Japan
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
261NCT00159250October 26, 200728 October 2019Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular DystrophyRestoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658Duchenne Muscular DystrophyDrug: AVI-4658 (PMO)Sarepta Therapeutics, Inc.Department of Health, United KingdomNot recruiting10 Years17 YearsMale7Phase 1/Phase 2United Kingdom
262EUCTR2006-003833-33-GB13/06/200731 October 2016Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658Duchenne Muscular Dystrophy
MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy
Product Name: AVI-4658
Product Code: AVI-4658
Pharmaceutical Form: Solution for injection
Current Sponsor code: AVI-4658
Other descriptive name: Phosphorodiamidate Morpholino Oligomer
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Intramuscular use
Imperial College, LondonNot RecruitingFemale: no
Male: yes
9Phase 1;Phase 2United Kingdom
263EUCTR2006-006776-37-NL14/05/200719 March 2012Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHDTowards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHDHealthy volunteers. Later the protocol will be applied to Facioscapular humeral dystrophy and/or other muscular dystrophies in general.
MedDRA version: 8.1 Level: LLT Classification code 10011328 Term: Creatine
Product Name: creatine-13C
Product Code: Cr13C
Pharmaceutical Form: Powder for oral solution
Pharmaceutical form of the placebo: Powder for oral solution
Route of administration of the placebo: Oral use
Radboud University Nijmegen Medical CenterAuthorisedFemale: yes
Male: yes
Netherlands
264NCT00308113April 200719 October 2017CoQ10 and Prednisone in Non-Ambulatory DMDPITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: Prednisone;Dietary Supplement: Coenzyme Q10Cooperative International Neuromuscular Research GroupUnited States Department of DefenseNot recruiting10 Years18 YearsMale3Phase 3United States;Australia;Puerto Rico
265NCT00451074March 200719 February 2015Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop CodonsA Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon MutationsDuchenne Muscular DystrophyDrug: Gentamicin infusions twice a week for six monthsNationwide Children's HospitalNational Institutes of Health (NIH);National Institute of Neurological Disorders and Stroke (NINDS)Not recruiting5 Years20 YearsMale12Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
266NCT01344798November 200619 February 2015Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2CPhase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2CLimb Girdle Muscular Dystrophy Type 2C;Gamma-sarcoglycanopathyBiological: AAV1-gamma-sarcoglycan vector injectionGenethonNot recruiting15 YearsN/ABoth9Phase 1France
267NCT00428935March 200619 February 2015Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular DystrophyPhase 1 Clinical Trial of rAAV2.5-CMV-mini-Dystrophin Gene Vector in Duchenne Muscular DystrophyDuchenne Muscular DystrophyBiological: rAAV2.5-CMV-minidystrophin (d3990)Nationwide Children's HospitalAsklepios Biopharmaceutical, Inc.Not recruiting5 Years15 YearsMale6Phase 1United States
268NCT00296621February 200619 February 2015Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular DystrophyEfficacy Study of Oral Glutamine Supplementation in Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: L-Glutamine;Drug: placeboAssistance Publique - Hôpitaux de ParisNot recruitingN/AN/AMale30Phase 2France
269EUCTR2006-003075-12-IE2 October 2017Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction.Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction.Duchenne muscular dystrophy is an X-linked inherited disorder which primarily affects skeletal muscle through a mutation in dystrophin which maps to the long arm of the X chromosome (Xp21). Patients with skeletal myopathy such as DMD eventually progress to develop dilated cardiomyopathy as dystrophin is expressed in all forms of muscle including cardiac and smooth muscle. Often the cause of death in patients with DMD is cardiac failure and arrhythmia.Trade Name: Eucardic
Product Name: Eucardic
Product Code: No code
Pharmaceutical Form: Tablet
INN or Proposed INN: CARVEDILOL
CAS Number: 72956093
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 3.125mg-6.25mg
Department Of Paediatric CardiologyNot AvailableFemale: yes
Male: yes
30Phase 4Ireland
270NCT00264888December 200519 February 2015Safety and Efficacy Study of PTC124 in Duchenne Muscular DystrophyA Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PTC124PTC TherapeuticsMuscular Dystrophy AssociationNot recruiting5 YearsN/AMale38Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
271NCT00233519November 200519 October 2017Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)Effects of SomatoKine (Iplex) (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)Myotonic DystrophyDrug: SomatoKine/IPLEXUniversity of RochesterNational Institute of Neurological Disorders and Stroke (NINDS);Imsmed IncorporatedNot recruiting21 Years60 YearsAll17Phase 1/Phase 2United States
272NCT00654784October 200519 October 2017Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular DystrophyA Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD)Drug: idebenone;Drug: placeboSanthera PharmaceuticalsNot recruiting8 Years16 YearsMale21Phase 2Belgium
273EUCTR2005-002520-33-BE06/09/200519 March 2012A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular DystrophyA Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular DystrophyDuchenne Muscular DystrophyProduct Name: idebenone
Pharmaceutical Form: Tablet
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Santhera Pharmaceuticals LLCNot RecruitingFemale: no
Male: yes
21Phase 2aBelgium
274NCT00243789September 200519 February 2015Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular DystrophyA Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMDMuscular Dystrophy, DuchenneDrug: PentoxifyllineCooperative International Neuromuscular Research GroupNot recruiting7 YearsN/AMale64Phase 1/Phase 2United States;Argentina;Australia;Canada;Israel;Italy
275EUCTR2004-000622-67-GB03/06/20051 May 2012A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy.A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy.Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy.Product Name: MYO-029
Pharmaceutical Form: Powder for injection*
INN or Proposed INN: Not Available
Current Sponsor code: MYO-029
Other descriptive name: Recombinant human anti-GDF-8 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 70-
Pharmaceutical form of the placebo: Powder for injection*
Route of administration of the placebo: Intravenous use
Wyeth Research Division of Wyeth Pharmaceuticals Inc.AuthorisedFemale: yes
Male: yes
136United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
276EUCTR2005-000663-26-AT25/04/20053 April 2017Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsAImmunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsADuchenne Muscular DystrophyTrade Name: Sandimmun Optoral
Product Name: Sandimmun Optoral
Product Code: 29180.00.00, 34681.00.00, 34681.01.00, 34681.02.00
Pharmaceutical Form: Capsule*
Other descriptive name: Cyclosporin A
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: range
Concentration number: 3,5-4-
Pharmaceutical form of the placebo: Capsule*
Route of administration of the placebo: Oral use
Trade Name: Decortin
Product Name: Decortin
Product Code: not available
Pharmaceutical Form: Tablet
Other descriptive name: Prednison
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0,75 -10 d on/ 10 d off
Universitätsklinik FreiburgNot RecruitingFemale: no
Male: yes
150Phase 2;Phase 3Austria
277NCT00104078February 200519 February 2015Study Evaluating MYO-029 in Adult Muscular DystrophyBecker Muscular Dystrophy;Facioscapulohumeral Muscular Dystrophy;Limb-Girdle Muscular DystrophyDrug: MYO-029Wyeth is now a wholly owned subsidiary of PfizerNot recruiting18 YearsN/ABoth108Phase 1/Phase 2United States
278NCT00167609November 200419 February 2015Efficacy and Safety of DHEA for Myotonic DystrophyPhase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic DystrophyMyotonic DystrophyDrug: dehydroepiandrosterone 100 and 400 mgUniversity of VersaillesAssociation Française contre les Myopathies (AFM), Paris;AP-HPNot recruiting18 Years70 YearsBoth75Phase 2/Phase 3France
279NCT00110669January 200419 February 2015High-dose Prednisone in Duchenne Muscular DystrophyA Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: PrednisoneCooperative International Neuromuscular Research GroupNot recruiting4 Years10 YearsMale64Phase 3United States;India
280NCT00102453March 200219 February 2015Pentoxifylline in Duchenne Muscular DystrophyAn Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: PentoxifyllineCooperative International Neuromuscular Research GroupNot recruiting4 Years7 YearsMale17Phase 1/Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
281NCT00033813January 200219 February 2015KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Oxatomide (tinset)Cooperative International Neuromuscular Research GroupNot recruiting5 Years10 YearsMale15Phase 2United States
282NCT00033189September 200119 February 2015An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Coenzyme Q10Cooperative International Neuromuscular Research GroupNot recruiting5 Years11 YearsMale15Phase 2United States
283NCT00018109June 200119 February 2015A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)Muscular Dystrophy, DuchenneDrug: glutamine;Drug: creatine monohydrateNational Center for Research Resources (NCRR)Children's Research InstituteNot recruiting5 Years10 YearsMalePhase 3United States
284NCT01882400May 200118 January 2018Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular DystrophyÉvaluation Multidimensionnelle de la réponse au Traitement de l'ostéoporose spontanée et Induite Par Les corticostéroïdes à l'Aide d'un Bisphosphonate à Administration Orale Chez Des Malades Porteurs d'Une Dystrophie Musculaire sévère.Osteoporosis;Muscular Dystrophy;Cystic FibrosisDrug: Bisphosphonate treatmentGilles BoireProcter and GambleNot recruiting5 Years18 YearsAll11Phase 4Canada
285NCT00016653June 200019 February 2015Creatine and Glutamine in Steroid-Naive Duchenne Muscular DystrophyA Multicenter Randomized Placebo-controlled Double-blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular DystrophyMuscular Dystrophy, DuchenneDrug: Creatine Monohydrate;Drug: GlutamineCooperative International Neuromuscular Research GroupNot recruiting5 Years9 YearsMale48Phase 2/Phase 3United States;Belgium;Israel;Puerto Rico
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
286NCT00005574February 200019 February 2015Gentamicin Treatment of Muscular DystrophyGentamicin Treatment of Patients With Muscular Dystrophy Due to Nonsense Mutations in DystrophinBecker Muscular Dystrophy;Duchenne Muscular DystrophyDrug: GentamicinNational Institute of Neurological Disorders and Stroke (NINDS)Not recruitingN/AN/ABoth4Phase 1United States
287NCT00004685January 19987 April 2015Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular DystrophyMuscular Dystrophy, FacioscapulohumeralDrug: albuterolOhio State UniversityNot recruiting18 Years80 YearsBoth90N/A
288NCT00004646April 199519 February 2015Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular DystrophyDuchenne Muscular DystrophyDrug: prednisoneNational Center for Research Resources (NCRR)National Institute of Neurological Disorders and Stroke (NINDS);University of RochesterNot recruiting5 Years15 YearsMale20Phase 3
289JPRN-JapicCTI-14253823 April 2019A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular DystrophyA Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular DystrophyDuchenne Muscular DystrophyIntervention name : Tadalafil
Dosage And administration of the intervention : During the double-blind period, tadalafil or matching placebo will be administered orally once daily at one of 2 target doses (0.3 mg/kg or 0.6 mg/kg).During the open-label extension period all patients will initially receive tadalafil.
Eli Lilly Japan K.K.714MalePhase 3

先頭へ