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

Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy

12th July 2017

Condition:   Becker Muscular Dystrophy
Interventions:   Drug: givinostat;   Drug: placebo
Sponsor:   Italfarmaco
Not yet recruiting - verified July 2017

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Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping

9th July 2017

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Eteplirsen
Sponsor:   Sarepta Therapeutics
Recruiting - verified August 2017

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Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

1st 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 - verified July 2017

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Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

22nd 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 (CINRG);   Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Enrolling by invitation - verified August 2017

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User-centred Assistive System for Arm Functions in Neuromuscular Subjects

20th 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 - verified April 2017

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Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy

25th February 2017

Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: Stem Cells
Sponsor:   Stem Cells Arabia
Active, not recruiting - verified October 2016

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Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

30th 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
Enrolling by invitation - verified April 2017

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Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy

27th January 2017

Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: BMS-986089;   Biological: Placebo for BMS-986089
Sponsor:   Hoffmann-La Roche
Recruiting - verified August 2017

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Virtual Reality in Individuals With Duchenne Muscular Dystrophy

19th 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 - verified April 2017

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Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents

7th November 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: (+)- Epicatechin
Sponsors:   Craig McDonald, MD;   Cardero Therapeutics, Inc.
Recruiting - verified November 2016

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(-)- Epicatechin Becker Muscular Dystrophy

7th November 2016

Condition:   Becker Muscular Dystrophy
Intervention:   Drug: (-)-Epicatechin
Sponsors:   University of California, Davis;   Cardero Therapeutics, Inc.
Recruiting - verified July 2017

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Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)

27th October 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 - verified November 2016

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An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

14th September 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: PF-06252616
Sponsor:   Pfizer
Recruiting - verified August 2017

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Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy

17th August 2016

Condition:   Duchenne Muscular Dystrophy
Interventions:   Device: Acquisition on TouchScreen;   Device: Acquisition on Kinect;   Device: Acquisition on LeapMotion
Sponsor:   University of Sao Paulo
Completed - verified April 2017

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Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy

27th July 2016

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: givinostat;   Drug: placebo
Sponsors:   Italfarmaco;   inVentiv Health Clinical
Recruiting - verified August 2017

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PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD

27th July 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: ezutromid
Sponsor:   Summit Therapeutics
Active, not recruiting - verified June 2017

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Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

25th July 2016

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention:   Genetic: Genetic characterization
Sponsors:   Nationwide Children's Hospital;   Parent Project Muscular Dystrophy
Recruiting - verified July 2017

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Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD

13th 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 - verified August 2017

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Treatment Effect of Tamoxifen on Patients With DMD

22nd June 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Tamoxifen
Sponsor:   Hadassah Medical Organization
Not yet recruiting - verified July 2016

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A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids

17th June 2016

Condition:   Duchenne Muscular Dystrophy (DMD)
Interventions:   Drug: Idebenone 150 mg film-coated tablets;   Drug: placebo
Sponsor:   Santhera Pharmaceuticals
Recruiting - verified August 2017

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Study of Ataluren in ≥2 to <5 Year-Old Males With Duchenne Muscular Dystrophy

16th June 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Ataluren
Sponsor:   PTC Therapeutics
Active, not recruiting - verified April 2017

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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)

8th June 2016

Condition:   Heart Failure
Interventions:   Drug: PB1046 Injection;   Drug: Placebo Injection
Sponsor:   PhaseBio Pharmaceuticals Inc.
Recruiting - verified May 2017

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Acceptance and Commitment Therapy for Muscle Disease

20th 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 UK
Recruiting - verified June 2016

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Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study

19th May 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Other: Set of assessment tools
Sponsors:   University College, London;   Association Française contre les Myopathies (AFM), Paris;   University of Newcastle Upon-Tyne;   Groupe Hospitalier Pitie-Salpetriere;   Leiden University Medical Center;   Radboud University
Recruiting - verified April 2016

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Muscle Oxygenation in Effort in Neuromuscular Diseases

24th May 2016

Condition:   Neuromuscular Diseases
Intervention:   Other: muscle oxygenation
Sponsor:   University Hospital, Lille
Recruiting - verified May 2017

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An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

28th April 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)
Recruiting - verified June 2017

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A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)

28th April 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
Recruiting - verified June 2017

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A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy

6th April 2016

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: TAS-205;   Drug: Placebo
Sponsor:   Taiho Pharmaceutical Co., Ltd.
Active, not recruiting - verified February 2017

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Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

23rd March 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 (CINRG);   Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS)
Recruiting - verified July 2017

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Rimeporide in Patients With Duchenne Muscular Dystrophy

26th January 2016

Condition:   Muscular Dystrophy, Duchenne
Intervention:   Drug: Rimeporide
Sponsor:   EspeRare Foundation
Recruiting - verified July 2017

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Study of DS-5141b in Patients With Duchenne Muscular Dystrophy

26th January 2016

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: DS-5141b
Sponsors:   Daiichi Sankyo Co., Ltd.;   Orphan Disease Treatment Institute Co., Ltd.
Active, not recruiting - verified August 2017

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Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2

23rd December 2015

Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: rAAVrh74.MCK.GALGT2;   Other: PLACEBO (Saline)
Sponsor:   Kevin Flanigan
Withdrawn - verified November 2016

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The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy

14th December 2015

Condition:   Muscular Dystrophy
Interventions:   Drug: Tadalafil 20 MG;   Other: beetroot juice extract
Sponsor:   Cedars-Sinai Medical Center
Recruiting - verified March 2017

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Extension Study of Drisapersen in DMD Subjects

9th December 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Drisapersen
Sponsor:   BioMarin Pharmaceutical
Terminated - verified July 2016

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The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD

17th November 2015

Condition:   Duchenne Muscular Dystrophy (DMD)
Intervention:   Procedure: Remote Ischemic Preconditioning Training Apparatus
Sponsor:   General Hospital of Chinese Armed Police Forces
Recruiting - verified November 2015

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Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

4th November 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: pamrevlumab (FG-3019)
Sponsor:   FibroGen
Recruiting - verified August 2017

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Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

29th October 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Deflazacort
Sponsors:   PTC Therapeutics;   Parexel;   Dohmen Life Science Services
Available - verified August 2017

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Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy

24th August 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Sustanon (testosterone)
Sponsor:   Newcastle-upon-Tyne Hospitals NHS Trust
Active, not recruiting - verified March 2017

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Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients

10th August 2015

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: SRP-4045;   Drug: Placebo
Sponsor:   Sarepta Therapeutics
Active, not recruiting - verified August 2017

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Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin

3rd August 2015

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Metformin;   Drug: L-Arginine
Sponsor:   University Hospital, Basel, Switzerland
Completed - verified August 2015

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Study of an Investigational Drug, BMS-986089, in Ambulatory Boys With DMD

29th July 2015

Condition:   Muscular Dystrophy (DMD)
Interventions:   Biological: BMS-986089;   Biological: Placebo
Sponsor:   Hoffmann-La Roche
Active, not recruiting - verified August 2017

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HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02

18th July 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: HT-100
Sponsor:   Akashi Therapeutics
Terminated - verified July 2017

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Study of SRP-4045 and SRP-4053 in DMD Patients

14th July 2015

Conditions:   Duchenne Muscular Dystrophy;   DMD;   Muscular Dystrophy
Interventions:   Drug: SRP-4045;   Drug: SRP-4053;   Drug: Placebo
Sponsor:   Sarepta Therapeutics
Recruiting - verified August 2017

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HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)

19th June 2015

Conditions:   Duchenne Muscular Dystrophy;   Cardiomyopathy
Intervention:   Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002)
Sponsor:   Capricor Inc.
Active, not recruiting - verified November 2016

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Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2

16th June 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell
Sponsors:   University of Gaziantep;   Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital
Unknown status - verified June 2015

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Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy

10th June 2015

Condition:   Duchenne / Becker Muscular Dystrophy
Intervention:   Other: Observation
Sponsor:   University Children's Hospital, Zurich
Recruiting - verified October 2016

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Biomechanical and Morphological Changes in Dystrophic Muscle

10th June 2015

Condition:   Duchenne Dystrophy Muscular
Intervention:   Other: No drug and no placebo were used in this study
Sponsor:   University Hospital, Brest
Recruiting - verified October 2016

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Upper Limb Assessment in Duchenne Muscular Dystrophy

30th April 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Catholic University of the Sacred Heart;   Bambino Gesù Hospital;   IRCCS National Neurological Institute "C. Mondino" Foundation;   Azienda Ospedaliera San Giovanni Battista;   University of Messina;   Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta;   University of Naples;   IRCCS Fondazione Stella Maris;   Istituto Giannina Gaslini;   University of Padua;   Maggiore Bellaria Hospital, Bologna;   IRCCS Eugenio Medea;   Nemo Clinical Center Milan
Unknown status - verified May 2015

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Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy

29th April 2015

Condition:   Muscular Dystrophy, Duchenne
Interventions:   Drug: Edasalonexent;   Drug: Edasalonexent;   Drug: Placebo
Sponsor:   Catabasis Pharmaceuticals
Active, not recruiting - verified April 2017

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Sodium Nitrate for Muscular Dystrophy

28th April 2015

Condition:   Becker Muscular Dystrophy
Intervention:   Drug: Sodium Nitrate
Sponsor:   Cedars-Sinai Medical Center
Recruiting - verified October 2016

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Strength Training in Duchenne Muscular Dystrophy

15th 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)
Recruiting - verified August 2017

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2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children

13th April 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Other: speckle tracking (2D strain) echocardiography
Sponsor:   University Hospital, Montpellier
Recruiting - verified January 2016

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Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

10th April 2015

Condition:   Duchenne Muscular Dystrophy (DMD)
Intervention:   Drug: eteplirsen
Sponsor:   Sarepta Therapeutics
Active, not recruiting - verified May 2017

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Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial

24th March 2015

Conditions:   Myocardial Fibrosis;   Muscular Dystrophies
Intervention:   Drug: Enalapril
Sponsors:   InCor Heart Institute;   Federal University of Minas Gerais;   University of Sao Paulo
Completed - verified April 2015

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Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy

26th February 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)
Active, not recruiting - verified November 2016

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Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet

4th February 2015

Condition:   Muscular Dystrophy, Duchenne
Interventions:   Drug: SMT C1100;   Drug: SMT C1100;   Drug: SMT C1100
Sponsor:   Summit Therapeutics
Completed - verified August 2015

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Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy

28th January 2015

Condition:   Muscular Dystrophy, Duchenne
Intervention:   Drug: Translarna
Sponsors:   PTC Therapeutics;   TREAT-NMD;   Cooperative International Neuromuscular Research Group (CINRG)
Recruiting - verified February 2017

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Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy

27th January 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
Active, not recruiting - verified June 2017

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Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

26th January 2015

Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: rAAV1.CMV.huFollistin344
Sponsors:   Jerry R. Mendell;   Duchenne Alliance;   Milo Therapeutics
Active, not recruiting - verified March 2017

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Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

22nd December 2014

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 - verified April 2015

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Phase I/II Study of SRP-4053 in DMD Patients

3rd December 2014

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Placebo;   Drug: SRP-4053
Sponsors:   Sarepta Therapeutics;   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
Active, not recruiting - verified June 2017

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A Device for Rapid, Painless, Bedside Muscle Evaluation of Children

3rd December 2014

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Skulpt, Inc.;   National Institute of Neurological Disorders and Stroke (NINDS)
Completed - verified August 2016

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An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

18th November 2014

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Deflazacort
Sponsor:   Marathon Pharmaceuticals, LLC
Active, not recruiting - verified January 2017

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A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

4th November 2014

Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: PF-06252616;   Drug: Placebo
Sponsor:   Pfizer
Active, not recruiting - verified July 2017

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Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

30th October 2014

Condition:   Muscular Dystrophy, Duchenne
Intervention:   Drug: eteplirsen
Sponsor:   Sarepta Therapeutics
Active, not recruiting - verified June 2017

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Confirmatory Study of Eteplirsen in DMD Patients

25th September 2014

Condition:   Duchenne Muscular Dystrophy (DMD)
Intervention:   Drug: eteplirsen
Sponsor:   Sarepta Therapeutics
Active, not recruiting - verified May 2017

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Functional Muscle Ischemia With Tadalafil Treatment in Becker Muscular Dystrophy

23rd September 2014

Condition:   Becker Muscular Dystrophy
Interventions:   Drug: Tadalafil;   Other: Placebo
Sponsor:   Cedars-Sinai Medical Center
Completed - verified March 2017

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A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

22nd September 2014

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Deflazacort
Sponsor:   PTC Therapeutics
Completed - verified August 2017

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European Home Mechanical Ventilation Registry

6th October 2014

Conditions:   Pulmonary Disease, Chronic Obstructive;   Amyotrophic Lateral Sclerosis;   Spinal Cord Injury;   Muscular Dystrophies;   Obesity Hypoventilation Syndrome;   Kyphoscoliosis;   Congenital Central Hypoventilation Syndrome;   Duchenne Muscular Dystrophy;   Myopathies;   Myotonic Dystrophy
Intervention:  
Sponsors:   ResMed;   Clinical Trial Center North Hamburg Germany;   The Clinical Research Institute Munich Germany
Terminated - verified October 2016

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Stem Cell Therapy in Duchenne Muscular Dystrophy

12th September 2014

Condition:   Duchenne Muscular Dystrophy
Intervention:   Biological: Stem Cell
Sponsor:   Neurogen Brain and Spine Institute
Completed - verified September 2016

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A Study of TAS-205 for Duchenne Muscular Dystrophy

9th September 2014

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: TAS-205;   Drug: Placebo
Sponsor:   Taiho Pharmaceutical Co., Ltd.
Completed - verified September 2015

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Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

8th 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
Active, not recruiting - verified January 2017

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PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular Dystrophy

28th July 2014

Condition:   Becker Muscular Dystrophy
Interventions:   Drug: Tadalafil;   Drug: Placebo
Sponsor:   Cedars-Sinai Medical Center
Completed - verified July 2016

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Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

17th 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
Sponsor:   University of Florida
Recruiting - verified November 2016

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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

16th July 2014

Condition:   Stress
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Completed - verified July 7, 2017

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Muscle MRI in Becker Muscular Dystrophy and in Limb-girdle Muscular Dystrophy Type 2I

11th June 2014

Conditions:   Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy Type 2I
Intervention:  
Sponsor:   Rigshospitalet, Denmark
Completed - verified November 2016

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Magnetic Resonance and Optical Imaging of Dystrophic and Damaged Muscle

6th 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
Recruiting - verified July 2017

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Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients

9th May 2014

Condition:   Duchenne Muscular Dystrophy
Interventions:   Biological: Myoblast transplantation;   Procedure: Saline injection
Sponsor:   CHU de Quebec-Universite Laval
Recruiting - verified March 2017

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Sodium Nitrate to Improve Blood Flow

30th April 2014

Condition:   Becker Muscular Dystrophy
Intervention:   Dietary Supplement: Sodium nitrate
Sponsor:   Cedars-Sinai Medical Center
Completed - verified July 2016

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Evaluation of Muscle miRNA as Biomarkers in Dystrophinopathies

2nd April 2014

Conditions:   Dystrophinopathies,;   Muscular Dystrophies
Intervention:   Other: blood sample
Sponsor:   University Hospital, Montpellier
Recruiting - verified January 2015

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Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy

17th 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
Active, not recruiting - verified May 2017

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Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy

3rd February 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
Active, not recruiting - verified January 2017

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Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

9th January 2014

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   University of Pittsburgh;   Respirtech, Inc.
Recruiting - verified March 2017

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A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy

6th January 2014

Condition:   Duchenne Muscular Dystrophy (DMD)
Interventions:   Drug: Prednisone;   Drug: Placebo
Sponsors:   Ann & Robert H Lurie Children's Hospital of Chicago;   Children's Research Institute
Withdrawn - verified February 2015

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L-citrulline and Metformin in Becker's Muscular Dystrophy

17th December 2013

Condition:   Becker's Muscular Dystrophy (BMD)
Interventions:   Drug: Metformin and Metformin & L-Citrulline;   Drug: L-Citrulline and Metformin & L-Citrulline
Sponsor:   University Hospital, Basel, Switzerland
Completed - verified May 2016

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Prospective Becker-Heart-Study

19th December 2013

Conditions:   Dilated Cardiomyopathy;   Lef Ventricular Dysfunction;   Heart Failure
Intervention:   Procedure: ECG, echocardiography, cardiac MRI, sera biomarkers
Sponsors:   Karim WAHBI;   Institut de Myologie, France
Unknown status - verified March 2015

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L-citrulline and Metformin in Duchenne's Muscular Dystrophy

20th 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 - verified August 2016

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Stacking Exercises Aid the Decline in FVC and Sick Time

14th November 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
Active, not recruiting - verified August 2017

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Open Label Extension Study of HT-100 in Patients With DMD

31st October 2013

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: HT-100
Sponsor:   Akashi Therapeutics
Terminated - verified July 2017

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Cardiomyopathy in DMD: Lisinopril vs. Losartan

29th October 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 - verified January 2017

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Duchenne Muscular Dystrophy < 18y in Norway: Genotype/Phenotype, Growth, Puberty, Bone Health and Quality of Life.

12th October 2013

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Oslo University Hospital;   The Research Council of Norway;   University Hospital of North Norway
Enrolling by invitation - verified April 2017

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DuchenneConnect Registry

23rd September 2013

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention:  
Sponsors:   DuchenneConnect;   Parent Project Muscular Dystrophy
Recruiting - verified April 2016

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Whole Body Vibration Therapy in Boys With Duchenne Muscular Dystrophy

13th September 2013

Condition:   Duchenne Muscular Dystrophy
Intervention:   Device: Whole Body Vibration Therapy
Sponsor:   Children's Hospital of Eastern Ontario
Active, not recruiting - verified February 2017

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Mother-caregivers of Children With Duchenne Muscular Dystrophy

8th August 2013

Condition:   Other Diseases or Conditions
Interventions:   Biological: sleep parameters;   Biological: Hormonal profile;   Biological: Inflammatory profile;   Biological: Cardiovascular profile;   Biological: Metabolic profile
Sponsor:   Monica Levy Andersen
Completed - verified February 2016

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Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy

1st August 2013

Condition:   Muscular Dystrophy, Duchenne
Interventions:   Drug: NPC-14;   Drug: Placebo
Sponsors:   Kobe University;   Japan Medical Association;   Nobelpharma
Active, not recruiting - verified September 2015

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A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)

2nd July 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 - verified June 2015

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Duchenne Muscular Dystrophy Clinical Trial

7th June 2013

Condition:   Duchenne Muscular Dystrophy
Intervention:   Device: VECTTOR
Sponsor:   Alan Neuromedical Technologies, LLC
Completed - verified April 2015

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A Study of Tadalafil for Duchenne Muscular Dystrophy

24th May 2013

Condition:   Muscular Dystrophy, Duchenne
Interventions:   Drug: Tadalafil;   Drug: Placebo
Sponsor:   Eli Lilly and Company
Terminated - verified January 2017

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Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)

9th May 2013

Condition:   Becker Muscular Dystrophy
Intervention:   Drug: (-)-epicatechin
Sponsors:   Craig McDonald, MD;   Cardero Therapeutics, Inc.
Recruiting - verified March 2017

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Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy

2nd May 2013

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: HT-100
Sponsor:   Akashi Therapeutics
Terminated - verified July 2017

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Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients

4th April 2013

Condition:   Muscular Dystrophy, Duchenne
Interventions:   Dietary Supplement: EPA and DHA;   Dietary Supplement: Placebo Comparator: Sunflower oil
Sponsors:   Coordinación de Investigación en Salud, Mexico;   Instituto Nacional de Rehabilitacion
Enrolling by invitation - verified January 2016

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Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy

20th March 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 - verified June 2015

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Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy

26th March 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 - verified May 2017

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A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects

28th February 2013

Condition:   Muscular Dystrophies
Interventions:   Drug: Drisapersen;   Drug: Drisapersen;   Drug: Drisapersen
Sponsor:   BioMarin Pharmaceutical
Terminated - verified February 2016

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Duchenne Muscular Dystrophy Tissue Bank for Exon Skipping

17th January 2013

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsor:   Cooperative International Neuromuscular Research Group
Unknown status - verified July 2015

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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

20th December 2012

Condition:   Duchenne Muscular Dystrophy (DMD)
Intervention:   Drug: Givinostat
Sponsor:   Italfarmaco
Active, not recruiting - verified March 2017

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A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

13th December 2012

Condition:   Duchenne Muscular Dystrophy
Intervention:   Other: Observational study
Sponsor:   BioMarin Pharmaceutical
Terminated - verified June 2015

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Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study

19th October 2012

Condition:   Carrier of Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Hospital Rudolfstiftung;   Oesterreichische Muskelforschung
Completed - verified October 2016

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Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy

20th 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 - verified December 2016

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Pediatric Radio Frequency Coils Generic

21st June 2012

Conditions:   Duchenne Muscular Dystrophy;   Musculoskeletal Abnormalities
Intervention:  
Sponsor:   Children's Hospital Medical Center, Cincinnati
Recruiting - verified August 2016

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Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy

8th June 2012

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Ketamine;   Drug: Dexmedetomidine
Sponsor:   Nationwide Children's Hospital
Completed - verified February 2015

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Finding the Optimum Regimen for Duchenne Muscular Dystrophy

3rd April 2012

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Prednisone;   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 - verified January 2017

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Study of Ataluren for Previously Treated Patients With nmDBMD in Europe, Israel, Australia, and Canada

15th March 2012

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Dystrophinopathy
Intervention:   Drug: Ataluren
Sponsor:   PTC Therapeutics
Active, not recruiting - verified July 2016

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Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

23rd February 2012

Condition:   Duchenne Muscular Dystrophy (DMD)
Intervention:   Drug: AVI-4658 (Eteplirsen)
Sponsor:   Sarepta Therapeutics
Active, not recruiting - verified November 2016

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Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

22nd February 2012

Condition:   Becker Muscular Dystrophy
Intervention:  
Sponsor:   Cooperative International Neuromuscular Research Group
Active, not recruiting - verified January 2017

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Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy

26th January 2012

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: eplerenone;   Drug: placebo
Sponsors:   Subha Raman;   Ballou Skies
Completed - verified October 2016

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Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis

23rd 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)
Active, not recruiting - verified January 2017

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Double Push Acoustic Radiation Force (DP ARF) Ultrasound for Monitoring Degeneration in Duchenne Muscular Dystrophy

22nd December 2011

Condition:   Muscular Dystrophy, Duchenne
Intervention:  
Sponsors:   University of North Carolina, Chapel Hill;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified January 2017

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Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy

5th December 2011

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Boston Children’s Hospital;   Beth Israel Deaconess Medical Center
Completed - verified March 2016

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Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

23rd November 2011

Condition:   Muscular Dystrophies
Intervention:   Drug: GSK2402968
Sponsor:   GlaxoSmithKline
Terminated - verified March 2017

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Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

10th October 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 - verified January 2017

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Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular Dystrophy

8th October 2011

Conditions:   Muscular Dystrophy;   Muscular Disease
Intervention:  
Sponsor:   National Institute of Neurological Disorders and Stroke (NINDS)
Completed - verified April 21, 2017

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A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)

3rd 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 - verified April 2014

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Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

8th July 2011

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: AVI-4658 (Eteplirsen);   Other: Placebo
Sponsor:   Sarepta Therapeutics
Completed - verified July 2015

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Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

29th June 2011

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Genethon;   Institute of Myology
Recruiting - verified March 2016

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The Role of Family Functioning in Promoting Adaptation in Siblings of Individuals With Duchenne Muscular Dystrophy (DMD)

30th June 2011

Conditions:   Genetic Disease;   Communication
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Terminated - verified January 7, 2016

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Research of Biomarkers in Duchenne Muscular Dystrophy Patients

23rd June 2011

Condition:   Duchenne Muscular Dystrophy (DMD)
Intervention:  
Sponsors:   Genethon;   Institute of Myology
Completed - verified August 2016

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Study of Ataluren for Previously Treated Patients With nmDBMD in the US

22nd November 2010

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention:   Drug: Ataluren
Sponsor:   PTC Therapeutics
Enrolling by invitation - verified May 2017

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A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

21st October 2010

Condition:   Muscular Dystrophies
Intervention:   Drug: GSK2402968 6mg/kg/week
Sponsor:   GlaxoSmithKline
Completed - verified March 2017

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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

7th September 2010

Condition:   Osteoporosis
Intervention:   Drug: zoledronic acid
Sponsor:   Novartis Pharmaceuticals
Recruiting - verified April 2017

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Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy

17th August 2010

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: Epigallocatechin-Gallate;   Drug: Placebo
Sponsor:   Charite University, Berlin, Germany
Active, not recruiting - verified August 2017

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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

13th August 2010

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Completed - verified June 24, 2013

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A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

20th 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 - verified July 2017

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Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies

18th 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
Enrolling by invitation - verified June 2016

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Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

2nd 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 - verified March 2017

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This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy

1st 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 - verified January 2017

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Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)

21st December 2009

Condition:   Duchenne Muscular Dystrophy
Interventions:   Drug: PRO044 SC;   Drug: PRO044 IV
Sponsor:   BioMarin Pharmaceutical
Completed - verified March 2015

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Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

8th December 2009

Conditions:   Muscular Dystrophy, Duchenne;   Ambulatory Care
Interventions:   Drug: Placebo;   Drug: Idebenone
Sponsor:   Santhera Pharmaceuticals
Completed - verified September 2015

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Study of Ataluren (PTC124®) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)

5th November 2009

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention:   Drug: Ataluren (PTC124)
Sponsors:   PTC Therapeutics;   Genzyme, a Sanofi Company
Terminated - verified May 2017

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Safety Study of Transvenous Limb Perfusion in Human Muscular Dystrophy

1st April 2009

Conditions:   Muscular Dystrophies;   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Limb-Girdle Muscular Dystrophy
Intervention:   Other: Retrograde high pressure transvenous perfusion with normal saline
Sponsors:   University of North Carolina, Chapel Hill;   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);   University of North Carolina
Completed - verified January 2015

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Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

16th February 2009

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Intervention:   Drug: Ataluren (PTC124)
Sponsors:   PTC Therapeutics;   Genzyme, a Sanofi Company
Terminated - verified May 2017

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Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders

6th February 2009

Conditions:   Duchenne Muscular Dystrophy;   Amyotrophic Lateral Sclerosis;   Neuromuscular Diseases
Interventions:   Device: mechanical insufflation - exsufflation;   Device: Standard respiratory physiotherapy
Sponsor:   Assistance Publique - Hôpitaux de Paris
Terminated - verified May 2015

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Ramipril Versus Carvedilol in Duchenne and Becker Patients

8th January 2009

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Interventions:   Drug: carvedilol;   Drug: ramipril
Sponsor:   Catholic University, Italy
Unknown status - verified January 2009

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Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

24th December 2008

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: AVI-4658 for Injection
Sponsors:   Sarepta Therapeutics;   British Medical Research Council
Completed - verified September 2015

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A Research Study of Zoledronic Acid in Children and Adolescents With Osteoporosis

26th November 2008

Condition:   Osteoporosis
Interventions:   Drug: Zoledronic acid;   Drug: Placebo
Sponsor:   Novartis Pharmaceuticals
Recruiting - verified April 2017

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Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

23rd September 2008

Condition:   Duchenne Muscular Dystrophy
Intervention:   Drug: Ataluren
Sponsors:   PTC Therapeutics;   Genzyme, a Sanofi Company
Terminated - verified May 2017

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Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

1st January 2008

Conditions:   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy
Interventions:   Drug: PTC124 High Dose;   Drug: PTC124 Low Dose;   Drug: Placebo
Sponsor:   PTC Therapeutics
Completed - verified June 2017

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Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)

1st May 2007

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsors:   Cooperative International Neuromuscular Research Group;   U.S. Department of Education;   National Institutes of Health (NIH);   United States Department of Defense;   Parent Project Muscular Dystrophy
Active, not recruiting - verified April 2016

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Molecular Analysis of Patients With Neuromuscular Disease

17th October 2006

Conditions:   Limb-girdle Muscular Dystrophy;   Duchenne Muscular Dystrophy;   Becker Muscular Dystrophy;   Facioscapulohumeral Muscular Dystrophy
Intervention:  
Sponsors:   Boston Children’s Hospital;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified July 2017

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Biomechanical Analysis of Gait in Individuals With Duchenne Muscular Dystrophy

5th April 2006

Condition:   Duchenne Muscular Dystrophy
Intervention:  
Sponsor:   Shriners Hospitals for Children
Completed - verified May 2015

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Test-Retest Reliability of Pulmonary Function Tests in Patients With Duchenne's Muscular Dystrophy

13th September 2005

Conditions:   Duchenne's Muscular Dystrophy;   Scoliosis
Intervention:  
Sponsor:   Children's Healthcare of Atlanta
Withdrawn - verified February 2015

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12 Apr 2017