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

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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

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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

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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

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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

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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

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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

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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

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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

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Rimeporide in Patients With Duchenne Muscular Dystrophy

17th March 2016

Condition: Muscular Dystrophy, Duchenne
Intervention: Drug: Rimeporide
Sponsor: EspeRare Foundation
Completed

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy

5th March 2019

Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Vamorolone
Sponsor: ReveraGen BioPharma, Inc.
Available

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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

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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

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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

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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

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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

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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

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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

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Prognostic Factors Affecting Duchenne Muscular Dystrophy

14th December 2017

Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Assiut University
Not yet recruiting

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(-)- Epicatechin Becker Muscular Dystrophy

2nd August 2017

Condition: Becker Muscular Dystrophy
Intervention: Drug: (-)-Epicatechin
Sponsors: Craig McDonald, MD; Cardero Therapeutics, Inc.
Completed

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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

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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

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Tamoxifen in Duchenne Muscular Dystrophy

27th November 2017

Condition: Duchenne Muscular Dystrophy
Interventions: Drug: Tamoxifen; Drug: Matching placebo
Sponsor: University Hospital, Basel, Switzerland
Recruiting

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A Registered Cohort Study on Duchenne Muscular Dystrophy

9th July 2019

Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Ning Wang, MD., PhD.
Recruiting

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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Spironolactone Versus Prednisolone in DMD

17th December 2018

Condition: Muscular Dystrophy, Duchenne
Interventions: Drug: Spironolactone; Drug: Prednisolone
Sponsors: Kevin Flanigan; Muscular Dystrophy Association
Recruiting

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Natural History of Duchenne Muscular Dystrophy

20th March 2019

Condition: Duchenne Muscular Dystrophy
Intervention:
Sponsor: Genethon
Not yet recruiting

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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

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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

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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

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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

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Extension Study of Drisapersen in DMD Subjects

22nd December 2015

Condition: Duchenne Muscular Dystrophy
Intervention: Drug: Drisapersen
Sponsor: BioMarin Pharmaceutical
No longer available

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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The Duchenne Registry

24th February 2014

Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy
Intervention:
Sponsors: The Duchenne Registry; Parent Project Muscular Dystrophy
Recruiting

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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

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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

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Study of Eteplirsen in DMD Patients

2nd October 2014

Condition: Duchenne Muscular Dystrophy (DMD)
Intervention: Drug: eteplirsen
Sponsor: Sarepta Therapeutics, Inc.
Completed

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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Sodium Nitrate to Improve Blood Flow

28th July 2016

Condition: Becker Muscular Dystrophy
Intervention: Dietary Supplement: Sodium nitrate
Sponsor: Cedars-Sinai Medical Center
Completed

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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

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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

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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

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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

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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

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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

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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

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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

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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

28th November 2011

Condition: Muscular Dystrophies
Intervention: Drug: GSK2402968
Sponsor: GlaxoSmithKline
Terminated

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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

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Muscle Oxygenation in Effort in Neuromuscular Diseases

2nd June 2016

Condition: Neuromuscular Diseases
Intervention: Other: muscle oxygenation
Sponsor: University Hospital, Lille
Unknown status

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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

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12 Apr 2017