Current trials in DMD
The table below lists all DMD trials registered or updated on clinicaltrials.gov in the last 1000 days. Because it links directly to clinicaltrials.gov it is automatically updated whenever the information on clinicaltrials.gov changes.
Please note that TREAT-NMD can take no responsibility for the accuracy of the information below, which is pulled directly from clinicaltrials.gov.
Trials for DMD listed on clinicaltrials.gov
Regular Physical Exercise in Duchenne Muscular Dystrophy
24th May 2019
Condition: Muscular Dystrophy, Duchenne
Intervention: Other: Physical Exercise
Sponsors: Haukeland University Hospital; Extrastiftelsen
Not yet recruiting
Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
12th October 2018
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: Edasalonexent; Drug: Placebo
Sponsor: Catabasis Pharmaceuticals
Recruiting
An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
18th September 2018
Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: SRP-5051
Sponsor: Sarepta Therapeutics, Inc.
Recruiting
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
7th December 2018
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: SRP-9001; Drug: Placebo
Sponsor: Sarepta Therapeutics, Inc.
Recruiting
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients
20th June 2019
Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: Eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Not yet recruiting
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
18th December 2017
Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: SRP-5051
Sponsor: Sarepta Therapeutics, Inc.
Completed
Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
8th May 2015
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: Edasalonexent; Drug: Placebo
Sponsor: Catabasis Pharmaceuticals
Active, not recruiting
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
17th January 2018
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: Cosyntropin; Other: Placebo
Sponsor: Mallinckrodt
Recruiting
Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy
10th January 2012
Condition: Muscular Dystrophy, Duchenne
Intervention:
Sponsors: University of North Carolina, Chapel Hill; National Institute of Neurological Disorders and Stroke (NINDS)
Active, not recruiting
Rimeporide in Patients With Duchenne Muscular Dystrophy
17th March 2016
Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: Rimeporide
Sponsor: EspeRare Foundation
Completed
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
21st July 2014
Condition: Muscular Dystrophy, Duchenne
Interventions: Other: Magnetic Resonance Imaging (MRI); Other: Pulmonary Function Testing (PFT); Other: Metabolic Exercise Testing using stationary bicycle; Other: Echocardiogram
Sponsors: University of Florida; CureDuchenne
Completed
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
10th November 2014
Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: Eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Completed
A Study of Tadalafil for Duchenne Muscular Dystrophy
30th May 2013
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: Tadalafil; Drug: Placebo
Sponsor: Eli Lilly and Company
Terminated
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
20th September 2016
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: PF-06252616
Sponsor: Pfizer
Terminated
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
7th November 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: rAAVrh74.MCK.GALGT2
Sponsor: Kevin Flanigan
Active, not recruiting
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
20th February 2018
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Vamorolone; Drug: Prednisone; Other: Placebo
Sponsors: ReveraGen BioPharma, Inc.; European Union; Cooperative International Neuromuscular Research Group; Newcastle University; University of Pittsburgh
Recruiting
Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
31st January 2017
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: 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/day
Sponsors: ReveraGen BioPharma, Inc.; University of Pittsburgh; Cooperative International Neuromuscular Research Group
Active, not recruiting
Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
10th March 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Biological: rAAVrh74.MCK.GALGT2; Other: PLACEBO (Saline)
Sponsor: Kevin Flanigan
Withdrawn
A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
8th December 2014
Condition: Duchenne Muscular Dystrophy
Interventions: Biological: PF-06252616; Drug: Placebo
Sponsor: Pfizer
Terminated
An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
3rd May 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: 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/day
Sponsors: ReveraGen 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)
Completed
A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
3rd May 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Vamorolone 0.25 mg/kg/day; Drug: Vamorolone 0.75 mg/kg/day; Drug: Vamorolone 2.0 mg/kg/day; Drug: Vamorolone 6.0 mg/kg/day
Sponsors: ReveraGen 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 Group
Completed
Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
21st August 2015
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: SRP-4045; Drug: Placebo
Sponsor: Sarepta Therapeutics, Inc.
Completed
Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy
7th September 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Device: Acquisition on TouchScreen; Device: Acquisition on Kinect; Device: Acquisition on LeapMotion
Sponsor: University of Sao Paulo
Completed
Stem Cell Therapy in Duchenne Muscular Dystrophy
16th September 2014
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: Stem Cell
Sponsor: Neurogen Brain and Spine Institute
Withdrawn
Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy
5th March 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Vamorolone
Sponsor: ReveraGen BioPharma, Inc.
Available
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
1st February 2017
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: RO7239361; Drug: Placebo for RO7239361
Sponsor: Hoffmann-La Roche
Recruiting
Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
16th November 2016
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: (+)- Epicatechin
Sponsors: Craig McDonald, MD; Cardero Therapeutics, Inc.
Completed
Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
29th January 2016
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: DS-5141b
Sponsors: Daiichi Sankyo Co., Ltd.; Orphan Disease Treatment Institute Co., Ltd.
Active, not recruiting
Investigation Of Factors Affecting Hand Functions in Nonambulatory Patients With Duchenne Muscular Dystrophy
10th May 2018
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Hacettepe University
Completed
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
1st January 2015
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: PRO044 SC 6 mg/kg; Drug: PRO044 IV 6 mg/kg; Drug: PRO044 IV 9 mg/kg
Sponsor: BioMarin Pharmaceutical
Terminated
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
26th November 2013
Condition: Duchenne's Muscular Dystrophy (DMD)
Interventions: Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.; Drug: Placebo
Sponsor: University Hospital, Basel, Switzerland
Completed
Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
24th February 2015
Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: Translarna
Sponsors: PTC Therapeutics; TREAT-NMD; Cooperative International Neuromuscular Research Group
Recruiting
Prognostic Factors Affecting Duchenne Muscular Dystrophy
14th December 2017
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Assiut University
Not yet recruiting
(-)- Epicatechin Becker Muscular Dystrophy
2nd August 2017
Condition: Becker Muscular Dystrophy
Intervention: Drug: (-)-Epicatechin
Sponsors: Craig McDonald, MD; Cardero Therapeutics, Inc.
Completed
Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
18th August 2010
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Epigallocatechin-Gallate; Drug: Placebo
Sponsor: Charite University, Berlin, Germany
Completed
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
22nd May 2018
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Casimersen; Drug: Golodirsen
Sponsor: Sarepta Therapeutics, Inc.
Enrolling by invitation
Tamoxifen in Duchenne Muscular Dystrophy
27th November 2017
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Tamoxifen; Drug: Matching placebo
Sponsor: University Hospital, Basel, Switzerland
Recruiting
A Registered Cohort Study on Duchenne Muscular Dystrophy
9th July 2019
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Ning Wang, MD., PhD.
Recruiting
A Phase 2 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
1st July 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: SRP-5051
Sponsor: Sarepta Therapeutics, Inc.
Recruiting
Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
8th November 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: BMN 044 IV 6 mg/kg; Drug: BMN 044 IV 9 mg/kg; Drug: BMN 044 SC 6 mg/kg
Sponsor: BioMarin Pharmaceutical
Terminated
Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
8th April 2013
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: 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 dose
Sponsor: BioMarin Pharmaceutical
Terminated
Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD
15th July 2016
Condition: Muscular Dystrophy, Duchenne
Interventions: Other: Cardiac MRI with contrast; Other: Cardiac MRI without contrast; Other: Blood Test; Other: Heart Rate; Other: Pulmonary Function Test; Other: Genetic Testing; Other: Repeat MRI scan
Sponsors: University of California, Los Angeles; National Institutes of Health (NIH); Children’s Hospital of Orange County; National Heart, Lung, and Blood Institute (NHLBI)
Recruiting
Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
19th June 2014
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Prednisolone
Sponsors: Washington University School of Medicine; Nationwide Children's Hospital; Feinberg School of Medicine, Northwestern University; University of Texas Southwestern Medical Center; University of California, Davis; Nemours Hospital, Orlando, FL
Completed
Physical Activity Level of Norwegian Boys With Duchenne Muscular Dystrophy
13th May 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Behavioral: Physical activity registration
Sponsors: Haukeland University Hospital; Norwegian School of Sport Sciences; Oslo University Hospital
Not yet recruiting
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
30th November 2018
Condition: Duchenne Muscular Dystrophy
Intervention: Other: Visit frequency
Sponsor: Pfizer
Recruiting
An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
17th April 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Edasalonexent
Sponsor: Catabasis Pharmaceuticals
Enrolling by invitation
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
3rd May 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Canakinumab Injection [Ilaris]
Sponsors: Children's Research Institute; Foundation to Eradicate Duchenne
Recruiting
Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
17th May 2013
Condition: Becker Muscular Dystrophy
Intervention: Drug: (-)-epicatechin
Sponsors: Craig McDonald, MD; Cardero Therapeutics, Inc.
Completed
Aerobic Exercise in Boys With Duchenne Muscular Dystrophy (DMD)
24th October 2017
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Ann & Robert H Lurie Children's Hospital of Chicago
Completed
A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
8th October 2013
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: 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 Extension
Sponsor: BioMarin Pharmaceutical
Terminated
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy
14th June 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Not yet recruiting
Strength Training in Duchenne Muscular Dystrophy
20th April 2015
Condition: Duchenne Muscular Dystrophy
Interventions: Procedure: Aim 2 Exercise group; Procedure: Aim 1 Exercise Dosing; Procedure: Aim 2 Control group
Sponsors: University of Florida; National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Completed
Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD)
15th February 2018
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Idebenone
Sponsor: Santhera Pharmaceuticals
Available
Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy
15th December 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: rAAVrh74.MHCK7.micro-dystrophin
Sponsor: Sarepta Therapeutics, Inc.
Active, not recruiting
The Burden of Access in Duchenne Muscular Dystrophy in the US
15th May 2019
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsors: University of Florida; Engage Health, Inc.
Recruiting
Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
26th April 2018
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: WVE-210201; Drug: Placebo
Sponsor: Wave Life Sciences Ltd.
Completed
Virtual Reality in Individuals With Duchenne Muscular Dystrophy
23rd December 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Behavioral: DMD gesture task; Behavioral: DMD button-press task; Behavioral: Control group gesture task; Behavioral: Control group button-press task
Sponsor: University of Sao Paulo
Completed
A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 (Suvodirsen) in Ambulatory Patients With Duchenne Muscular Dystrophy
8th April 2019
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: WVE-210201 (suvodirsen); Drug: Placebo
Sponsor: Wave Life Sciences Ltd.
Not yet recruiting
Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
27th July 2018
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: idebenone 150 mg film-coated tablets
Sponsor: Santhera Pharmaceuticals
Recruiting
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
8th January 2019
Condition: Duchenne Muscular Dystrophy
Intervention: Other: Dystrophin levels
Sponsor: PTC Therapeutics
Recruiting
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
26th April 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: TAS-205; Drug: Placebo
Sponsor: Taiho Pharmaceutical Co., Ltd.
Completed
Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
25th May 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: NS-065/NCNP-01
Sponsors: NS Pharma, Inc.; Nippon Shinyaku Co., Ltd.; Cooperative International Neuromuscular Research Group; Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Active, not recruiting
A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
22nd August 2018
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Deflazacort
Sponsor: PTC Therapeutics
Withdrawn
The PTC124 (Ataluren) Clinical Trial for Duchenne Muscular Dystrophy: Exploration of the Experiences of Parents, Clinician Researchers, and the Industry Sponsor
16th August 2010
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Completed
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
5th December 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Genetic: PF-06939926
Sponsor: Pfizer
Enrolling by invitation
Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy
1st March 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: Stem Cells
Sponsor: Stem Cells Arabia
Recruiting
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
3rd August 2017
Condition: Becker Muscular Dystrophy
Interventions: Drug: givinostat; Drug: placebo
Sponsor: Italfarmaco
Recruiting
Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients
22nd July 2014
Condition: Duchenne Muscular Dystrophy
Interventions: Biological: Myoblast transplantation; Procedure: Saline injection
Sponsor: CHU de Quebec-Universite Laval
Recruiting
Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
3rd February 2015
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Eplerenone; Drug: Spironolactone
Sponsors: Ohio State University; University of California, Los Angeles; University of Utah; University of Colorado, Denver; University of Kansas Medical Center; Vanderbilt University Medical Center
Completed
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
2nd August 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: givinostat; Drug: placebo
Sponsors: Italfarmaco; Syneos Health
Recruiting
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
17th November 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: pamrevlumab (FG-3019)
Sponsor: FibroGen
Active, not recruiting
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
17th April 2015
Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Completed
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
23rd May 2012
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Prednisone; Drug: Deflazacort
Sponsors: University of Rochester; Newcastle University; University Medical Center Freiburg; National Institute of Neurological Disorders and Stroke (NINDS)
Active, not recruiting
Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
30th October 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Deflazacort
Sponsors: PTC Therapeutics; Parexel; Dohmen Life Science Services
Approved for marketing
Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
3rd March 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: rAAVrh74.MCK.micro-Dystrophin
Sponsors: Jerry R. Mendell; Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Completed
2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children
16th April 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Other: speckle tracking (2D strain) echocardiography
Sponsor: University Hospital, Montpellier
Completed
Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life.
16th October 2013
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsors: Oslo University Hospital; The Research Council of Norway; University Hospital of North Norway
Completed
Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
3rd February 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Biological: rAAV1.CMV.huFollistin344
Sponsors: Jerry R. Mendell; Duchenne Alliance; Milo Therapeutics
Completed
Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy
7th October 2013
Condition: Duchenne Muscular Dystrophy
Intervention: Device: Whole Body Vibration Therapy
Sponsor: Children's Hospital of Eastern Ontario
Completed
Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
6th November 2009
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: Drug: Ataluren (PTC124)
Sponsors: PTC Therapeutics; Genzyme, a Sanofi Company
Terminated
Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
14th January 2008
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Interventions: Drug: PTC124 High Dose; Drug: PTC124 Low Dose; Drug: Placebo
Sponsor: PTC Therapeutics
Completed
Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
19th February 2009
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: Drug: Ataluren (PTC124)
Sponsors: PTC Therapeutics; Genzyme, a Sanofi Company
Terminated
Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
12th September 2005
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: AVI-4658 (PMO)
Sponsors: Imperial College London; Department of Health, United Kingdom
Completed
Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
10th September 2014
Condition: Duchenne's Muscular Dystrophy
Intervention: Biological: Umbilical Cord Mesenchymal Stem Cells
Sponsors: Allergy and Asthma Consultants, Wichita, Kansas; Aidan Foundation; Neil H. Riordan PhD
Completed
Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
25th May 2011
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Tadalafil; Drug: Sildenafil
Sponsors: Cedars-Sinai Medical Center; Parent Project Muscular Dystrophy
Completed
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
29th September 2014
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Deflazacort
Sponsor: PTC Therapeutics
Completed
Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
25th September 2008
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
Sponsors: PTC Therapeutics; Genzyme, a Sanofi Company
Terminated
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
28th February 2012
Condition: Becker Muscular Dystrophy
Intervention:
Sponsor: Cooperative International Neuromuscular Research Group
Active, not recruiting
A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.
20th December 2012
Condition: Duchenne Muscular Dystrophy
Intervention: Other: Observational study
Sponsor: BioMarin Pharmaceutical
Terminated
Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
7th May 2013
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: HT-100
Sponsor: Akashi Therapeutics
Terminated
The Effect of Kinesiology Taping on Balance in Duchenne Muscular Dystrophy
30th May 2018
Conditions: Balance; Duchenne Muscular Dystrophy
Intervention: Device: Kinesiology tape
Sponsor: Hacettepe University
Completed
Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
23rd July 2010
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: Drug: Sildenafil
Sponsors: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.; Johns Hopkins University
Terminated
Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography
6th April 2018
Conditions: Duchenne Muscular Dystrophy; Muscular Dystrophies; Muscular Dystrophy, Duchenne
Intervention: Device: Multispectral Optoacoustic Tomography
Sponsor: University of Erlangen-Nürnberg Medical School
Completed
Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
18th July 2011
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: AVI-4658 (Eteplirsen); Other: Placebo
Sponsor: Sarepta Therapeutics, Inc.
Completed
This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
5th April 2010
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsors: Washington University School of Medicine; Nationwide Children's Hospital; Boston Children’s Hospital; University of Minnesota - Clinical and Translational Science Institute; University of California, Davis
Completed
Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy
8th April 2010
Condition: Duchenne Muscular Dystrophy
Interventions: Biological: ACE-031 0.5 mg/kg q4wk; Biological: ACE-031 1.0 mg/kg q2wk; Other: Placebo
Sponsor: Acceleron Pharma, Inc.
Terminated
Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
12th June 2015
Condition: Duchenne / Becker Muscular Dystrophy
Intervention: Other: Observation
Sponsor: University Children's Hospital, Zurich
Active, not recruiting
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
23rd December 2009
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: PRO044 SC; Drug: PRO044 IV
Sponsor: BioMarin Pharmaceutical
Completed
A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
27th June 2016
Condition: Duchenne Muscular Dystrophy (DMD)
Interventions: Drug: Idebenone 150 mg film-coated tablets; Drug: placebo
Sponsor: Santhera Pharmaceuticals
Recruiting
Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
8th October 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Sustanon (testosterone)
Sponsor: Newcastle-upon-Tyne Hospitals NHS Trust
Active, not recruiting
Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
28th February 2012
Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: AVI-4658 (Eteplirsen)
Sponsor: Sarepta Therapeutics, Inc.
Completed
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
8th April 2013
Condition: Muscular Dystrophy, Duchenne
Interventions: Dietary Supplement: EPA and DHA; Dietary Supplement: Placebo Comparator
Sponsors: Coordinación de Investigación en Salud, Mexico; Instituto Nacional de Rehabilitacion
Completed
Duchenne Muscular Dystrophy Heart Study
22nd February 2018
Conditions: Duchenne Muscular Dystrophy; Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
Intervention:
Sponsors: Hôpital Cochin; Association Monégasque contre les Myopathies
Recruiting
Lower Limb Flexibility in Duchenne Muscular Dystrophy: Effects on Functional Performance
23rd May 2018
Conditions: Duchenne Muscular Dystrophy; Performance; Flexibility
Intervention:
Sponsor: Hacettepe University
Completed
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
1st May 2018
Condition: Duchenne Muscular Dystrophy
Interventions: Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale; Other: The following data of motor function
Sponsor: University Hospital, Toulouse
Recruiting
Regression of Hamstring Flexibility and Performance in Children With Duchenne Muscular Dystrophy
18th July 2018
Conditions: Duchenne Muscular Dystrophy; Performance; Flexibility
Intervention:
Sponsor: Hacettepe University
Completed
Safety Study of BLS-M22 in Healthy Volunteers
31st December 2018
Condition: Muscular Dystrophy, Duchenne
Interventions: Biological: BLS-M22; Other: Placebo
Sponsor: BioLeaders Corporation
Not yet recruiting
Spironolactone Versus Prednisolone in DMD
17th December 2018
Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: Spironolactone; Drug: Prednisolone
Sponsors: Kevin Flanigan; Muscular Dystrophy Association
Recruiting
Natural History of Duchenne Muscular Dystrophy
20th March 2019
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Genethon
Not yet recruiting
Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
14th December 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Givinostat
Sponsors: Italfarmaco; Cromsource
Recruiting
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
27th August 2018
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
Sponsor: PTC Therapeutics
Active, not recruiting
Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy
30th June 2016
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
Sponsor: PTC Therapeutics
Completed
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
15th April 2016
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: NS-065/NCNP-01; Drug: Placebo
Sponsors: NS Pharma, Inc.; Nippon Shinyaku Co., Ltd.; Cooperative International Neuromuscular Research Group; Therapeutic Research in Neuromuscular Disorders Solutions
Completed
Extension Study of Drisapersen in DMD Subjects
22nd December 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Drisapersen
Sponsor: BioMarin Pharmaceutical
No longer available
Open Label Extension Study of HT-100 in Patients With DMD
7th November 2013
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: HT-100
Sponsor: Akashi Therapeutics
Terminated
Study of SRP-4045 and SRP-4053 in DMD Patients
16th July 2015
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: SRP-4045; Drug: SRP-4053; Drug: Placebo
Sponsor: Sarepta Therapeutics, Inc.
Recruiting
Phase I/II Study of SRP-4053 in DMD Patients
8th December 2014
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Placebo; Drug: SRP-4053
Sponsors: Sarepta Therapeutics, Inc.; Institut de Myologie, France; Consultants for Research in Imaging and Spectroscopy; Great Ormond Street Hospital for Children NHS Foundation Trust; Catholic University of the Sacred Heart; Royal Holloway University; SYSNAV; University College, London; University of Newcastle Upon-Tyne
Completed
Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD
19th August 2019
Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Viltolarsen; Drug: Placebo
Sponsors: NS Pharma, Inc.; Nippon Shinyaku Co., Ltd.
Not yet recruiting
Pulmonary and Upper Limb Functions in Duchenne Muscular Dystrophy
12th June 2018
Conditions: Duchenne Muscular Dystrophy; Upper Limb Function; Respiratory Function Test
Intervention: Other: The Performance of Upper Limb Questionnaire (PUL)
Sponsor: Hacettepe University
Completed
Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD
22nd May 2018
Condition: Duchenne Muscular Dystrophy
Interventions: Device: Armon Ayura (Kinova); Device: JAECO Wrex
Sponsor: Roxanna Marie Bendixen
Recruiting
Relationship Between PFTs and Pdi in DMD
7th November 2017
Condition: Duchenne Muscular Dystrophy
Interventions: Diagnostic Test: Esophageal Balloon; Diagnostic Test: Gastric Balloon; Diagnostic Test: Nasal Pressure Transducer
Sponsors: University of Minnesota - Clinical and Translational Science Institute; Fairview Health Services
Withdrawn
Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker.
18th March 2019
Condition: Muscular Dystrophy, Duchenne and Becker Types
Interventions: Other: multimodal physiotherapy program with RV; Other: multimodal physiotherapy program
Sponsor: University of Malaga
Enrolling by invitation
Safety and Efficacy of P-188 NF in DMD Patients
15th June 2018
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: P-188 NF
Sponsors: Phrixus Pharmaceuticals, Inc.; Charley's Fund
Suspended
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
17th July 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Recruiting
Microdystrophin Gene Transfer Study in Adolescents and Children With DMD
11th December 2017
Condition: Duchenne Muscular Dystrophy
Intervention: Genetic: SGT-001
Sponsor: Solid Biosciences, LLC
Recruiting
Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD
23rd November 2016
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: Genetic: Genetic characterization
Sponsors: Nationwide Children's Hospital; Parent Project Muscular Dystrophy
Active, not recruiting
PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD
8th August 2016
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: ezutromid
Sponsor: Summit Therapeutics
Completed
Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
26th January 2012
Conditions: Becker Muscular Dystrophy; Sporadic Inclusion Body Myositis
Intervention: Biological: rAAV1.CMV.huFollistatin344
Sponsors: Nationwide Children's Hospital; Parent Project Muscular Dystrophy; The Myositis Association (Grant Sponsor)
Completed
An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
20th November 2014
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Deflazacort
Sponsor: PTC Therapeutics
Completed
The Duchenne Registry
24th February 2014
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention:
Sponsors: The Duchenne Registry; Parent Project Muscular Dystrophy
Recruiting
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
13th November 2017
Conditions: Duchenne Muscular Dystrophy Cardiomyopathy; Cardiomyopathy, Dilated
Interventions: Drug: Ifetroban; Drug: Placebo
Sponsors: Cumberland Pharmaceuticals; Vanderbilt University Medical Center
Not yet recruiting
Kinesiology Taping in Duchenne Muscular Dystrophy: Effects on Performance, Gait Characteristics, and Energy Consumption
18th May 2018
Conditions: Performance; Energy; Gait; Duchenne Muscular Dystrophy
Intervention: Device: Kinesiology Taping
Sponsor: Hacettepe University
Completed
Study of Eteplirsen in DMD Patients
2nd October 2014
Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Completed
A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant Children With DMD
4th January 2013
Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: Givinostat
Sponsor: Italfarmaco
Completed
Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
24th July 2012
Conditions: Duchenne Muscular Dystrophy; Cardiomyopathy; Heart Failure
Interventions: Drug: Nebivolol; Drug: Placebo
Sponsors: Assistance Publique - Hôpitaux de Paris; Association Française contre les Myopathies (AFM), Paris
Active, not recruiting
HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
30th June 2015
Conditions: Duchenne Muscular Dystrophy; Cardiomyopathy
Intervention: Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Sponsor: Capricor Inc.
Completed
Effect of Muscle Strength on Hamstring Flexibility in Children With Duchenne Muscular Dystrophy
18th July 2018
Conditions: Duchenne Muscular Dystrophy; Muscle Strength; Lower Extremity; Hamstring Contractures
Intervention:
Sponsor: Hacettepe University
Completed
Quantitative Muscle Ultrasound as a Marker of Progression in Children With Muscular Diseases
25th December 2018
Conditions: Inflammatory Myopathy; Duchenne Muscular Dystrophy
Intervention: Diagnostic Test: Quantitative muscle ultrasound measurements
Sponsor: Benha University
Completed
Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dystrophy
25th April 2018
Conditions: Neuromuscular Diseases; Duchenne Muscular Dystrophy; Dystrophy; Dystrophy, Muscular
Intervention: Other: Assessments
Sponsor: Canan İpek
Recruiting
Bisoprolol in DMD Early Cardiomyopathy
18th December 2018
Conditions: Duchenne Muscular Dystrophy; Cardiomyopathy, Dilated
Intervention: Drug: Bisoprolol Fumarate
Sponsors: Peking Union Medical College Hospital; National Natural Science Foundation of China; Chinese Academy of Medical Sciences
Recruiting
Prevention of Scoliosis in Patients With Duchenne Muscular Dystrophy Using Portable Seat Device
2nd August 2018
Conditions: Scoliosis Neuromuscular; Duchenne Muscular Dystrophy; Lordosis Lumbar
Intervention: Device: Portable seat device devised to maintain lumbar lordosis
Sponsor: Seoul National University Hospital
Recruiting
Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle
20th June 2014
Condition: Duchenne Muscular Dystrophy
Interventions: Other: Exercising; Procedure: Optical and Magnetic Resonance Imaging and Spectroscopy
Sponsors: University of Florida; United States Department of Defense
Completed
Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies
10th April 2014
Conditions: Muscular Dystrophies; Becker Muscular Dystrophy; Duchenne Muscular Dystrophy
Intervention: Other: blood sample
Sponsor: University Hospital, Montpellier
Recruiting
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
2nd December 2011
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsors: University of Florida; University of Pennsylvania; Oregon Health and Science University; Children's Hospital of Philadelphia; Shriners Hospitals for Children; National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Recruiting
Cardiomyopathy in DMD: Lisinopril vs. Losartan
13th November 2013
Conditions: Duchenne Muscular Dystrophy (DMD); Cardiomyopathy
Interventions: Drug: Losartan; Drug: Lisinopril
Sponsors: Nationwide Children's Hospital; Boston Children’s Hospital; University of California, Davis; Unverisity of Kansas Medical Center; University of Minnesota - Clinical and Translational Science Institute; St. Louis Children's Hospital
Completed
Stacking Exercises Aid the Decline in FVC and Sick Time
3rd December 2013
Condition: Duchenne Muscular Dystrophy
Interventions: Device: Lung Volume Recruitment (LVR); Other: Conventional Treatment
Sponsors: Children's Hospital of Eastern Ontario; Jesse's Journey-The Foundation for Gene and Cell Therapy
Completed
Weekly Steroids in Muscular Dystrophy
13th August 2019
Conditions: Limb-girdle Muscular Dystrophy; Becker Muscular Dystrophy
Intervention: Drug: Prednisone
Sponsor: Northwestern University
Enrolling by invitation
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
24th November 2010
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Ataluren
Sponsor: PTC Therapeutics
Enrolling by invitation
Sodium Nitrate to Improve Blood Flow
28th July 2016
Condition: Becker Muscular Dystrophy
Intervention: Dietary Supplement: Sodium nitrate
Sponsor: Cedars-Sinai Medical Center
Completed
Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy
13th January 2014
Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsors: University of Pittsburgh; Respirtech, Inc.
Recruiting
Treatment Effect of Tamoxifen on Patients With DMD
15th July 2016
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Tamoxifen
Sponsor: Hadassah Medical Organization
Active, not recruiting
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
17th August 2015
Condition: Duchenne Muscular Dystrophy
Intervention: Drug: HT-100
Sponsor: Akashi Therapeutics
Terminated
A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
6th December 2010
Condition: Muscular Dystrophies
Intervention: Drug: GSK2402968 6mg/kg/week
Sponsor: GlaxoSmithKline
Completed
A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
31st October 2011
Condition: Muscular Dystrophies
Interventions: Drug: 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/week
Sponsor: GlaxoSmithKline
Completed
The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
12th January 2016
Condition: Muscular Dystrophy
Interventions: Drug: Tadalafil 20 MG; Other: beetroot juice extract
Sponsor: Cedars-Sinai Medical Center
Unknown status
To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
23rd November 2011
Condition: Duchenne Muscular Dystrophy (DMD)
Interventions: Drug: Isosorbide Dinitrate; Drug: Ibuprofen; Other: Ibuprofen and Isosorbide Dinitrate combination
Sponsor: Parent Project, Italy
Completed
The Role of Family Functioning in Promoting Adaptation in Siblings of Individuals With Duchenne Muscular Dystrophy (DMD)
1st July 2011
Conditions: Genetic Disease; Communication
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Terminated
Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
28th November 2011
Condition: Muscular Dystrophies
Intervention: Drug: GSK2402968
Sponsor: GlaxoSmithKline
Terminated
Where Does Hope Fit In? The Relationship Between Hope, Uncertainty, and Coping Efficacy in Mothers of Children With Duchenne/Becker Muscular Dystrophy
18th July 2014
Condition: Stress
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Completed
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
7th June 2017
Conditions: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Disease; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
Interventions: Drug: Ataluren; Drug: PLACEBO
Sponsor: PTC Therapeutics
Recruiting
Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada
19th March 2012
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Dystrophinopathy
Intervention: Drug: Ataluren
Sponsor: PTC Therapeutics
Completed
Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy
13th October 2011
Conditions: Muscular Dystrophy; Muscular Disease
Intervention:
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS)
Completed
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
23rd January 2018
Conditions: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Disorders, Atrophic; Muscular Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
Interventions: Biological: CAP-1002; Drug: Placebo
Sponsor: Capricor Inc.
Active, not recruiting
Endomysial Fibrosis, Muscular Inflammatory Response and Calcium Homeostasis Dysfunction in Duchenne Muscular Dystrophy
4th April 2013
Condition: Duchenne Muscular Distrophy (DMD)
Intervention: Other: Muscle biopsy
Sponsor: University Hospital, Montpellier
Recruiting
Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
20th May 2010
Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Limb Girdle Muscular Dystrophy
Intervention: Drug: Coenzyme Q10 and Lisinopril
Sponsors: Cooperative International Neuromuscular Research Group; United States Department of Defense
Completed
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
21st September 2018
Conditions: Duchenne Muscular Dystrophy; Burden, Dependency; Disability Physical; Disease Management; Impairment; Rare Diseases
Intervention:
Sponsors: Jett Foundation, Inc.; Engage Health, Inc.; Hyman, Phelps, & McNamara, P.C.; Ryans Quest Inc.; Michaels Cause Inc.; Nationwide Children's Hospital; Solid Biosciences, LLC; Santhera Pharmaceuticals; Italfarmaco; Catabasis Pharmaceuticals; Wave Life Sciences Ltd.; Sarepta Therapeutics, Inc.; Hoffmann-La Roche; Pfizer; Capricor Inc.; NS Pharma, Inc.
Completed
User-centred Assistive System for Arm Functions in Neuromuscular Subjects
25th April 2017
Conditions: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Muscular Dystrophy, Becker; Muscular Dystrophy, Limb-Girdle Type 2
Interventions: Device: Assistive device (Armon Ayura) test; Device: Assistive device (Jaeco Wrex) test
Sponsors: Politecnico di Milano; IRCCS Eugenio Medea; Villa Beretta Rehabilitation Center
Recruiting
Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy
8th April 2013
Conditions: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
Interventions: Drug: Ataluren; Drug: Placebo
Sponsor: PTC Therapeutics
Completed
Molecular Analysis of Patients With Neuromuscular Disease
19th October 2006
Conditions: Limb-girdle Muscular Dystrophy; Duchenne Muscular Dystrophy; Becker Muscular Dystrophy; Neuromuscular; Disorder, Hereditary
Intervention:
Sponsors: Boston Children’s Hospital; National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
24th May 2010
Condition: Muscular Dystrophies
Interventions: Drug: 3 mg/kg GSK2402968; Drug: 6 mg/kg GSK2402968; Drug: 9 mg/kg GSK2402968; Drug: 12 mg/kg GSK2402968; Other: Placebo
Sponsor: GlaxoSmithKline
Completed
Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback
28th September 2018
Conditions: Neuromuscular Disease; Duchenne Muscular Dystrophy; Spinal Muscular Atrophy; Virtual Reality; Biofeedback
Interventions: Other: Virtual Reality Training; Other: Biofeedback Training; Other: Conventional rehabilitation
Sponsor: Merve Kurt
Not yet recruiting
A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
4th March 2013
Condition: Muscular Dystrophies
Intervention: Drug: Drisapersen
Sponsor: BioMarin Pharmaceutical
Terminated
Biomechanical and Morphological Changes in Dystrophic Muscle
16th June 2015
Condition: Duchenne Dystrophy Muscular
Intervention: Other: No drug and no placebo were used in this study
Sponsor: University Hospital, Brest
Recruiting
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
5th August 2015
Condition: Muscular Dystrophy (DMD)
Interventions: Drug: RO7239361; Drug: Placebo
Sponsor: Hoffmann-La Roche
Active, not recruiting
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
19th March 2014
Conditions: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
Intervention: Drug: Ataluren
Sponsor: PTC Therapeutics
Completed
Study 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)
22nd June 2016
Condition: Heart Failure
Interventions: Drug: PB1046 Injection; Drug: Placebo Injection
Sponsor: PhaseBio Pharmaceuticals Inc.
Completed
Biomarker for Duchenne Disease (BioDuchenne)
15th December 2016
Conditions: Increased Lordosis/Scoliosis; Hyporeflexia; Duchenne Muscular Dystrophy; Red-Green Color Blindness; Lordosis; Scoliosis; Muscular Atrophy; Muscular Weakness
Intervention:
Sponsor: Centogene AG Rostock
Recruiting
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
9th September 2010
Condition: Osteoporosis
Intervention: Drug: Zoledronic acid
Sponsor: Novartis Pharmaceuticals
Completed
An Efficacy and Safety Trial of Intravenous Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
27th November 2008
Condition: Osteoporosis
Interventions: Drug: Zoledronic acid; Drug: Placebo
Sponsor: Novartis Pharmaceuticals
Completed
Muscle Oxygenation in Effort in Neuromuscular Diseases
2nd June 2016
Condition: Neuromuscular Diseases
Intervention: Other: muscle oxygenation
Sponsor: University Hospital, Lille
Unknown status
Acceptance and Commitment Therapy for Muscle Disease
22nd June 2016
Condition: Muscle Diseases
Intervention: Behavioral: Acceptance and Commitment Therapy (ACT)
Sponsors: King's College Hospital NHS Trust; King's College London; Barts & The London NHS Trust; Muscular Dystrophy Association; University Hospital Southampton NHS Foundation Trust
Completed