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Clinical Trials for Muscular Dystrophies

• Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1
• Phase 2 Study of EDG-5506 in Children and Adolescents With Duchenne Muscular Dystrophy Previously Treated With Gene Therapy (FOX)
• A Study Of PGN-EDO51 In Participants With Duchenne Muscular Dystrophy Amenable To Exon 51-Skipping Treatment
• Open-Label Extension of EDG-5506 in Participants With Becker Muscular Dystrophy
• NS-050/NCNP-03 in Boys With DMD (Meteor50)
• NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
• A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
• Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
• Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
• Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD)
• Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers
• Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
• A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
• A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy
• Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
• Treatment of a Single Patient With CRD-TMH-001
• A Study to Assess the Feasibility of Non-invasive Dried Blood Sampling
• Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
• Once Weekly Infant Corticosteroid Trial for DMD
• Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
• Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy
• Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy
• Tadalafil as Adjuvant Therapy for DMD
• A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD)
• A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
• A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy
• Extension Study of NS-089/NCNP-02 in DMD
• A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-3)
• The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD
• Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
• Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD
• Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
• Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval
• Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
• An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
• Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
• A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
• A Phase 3 Study of TAS-205 in Patients With Duchenne Muscular Dystrophy（REACH-DMD）
• A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
• Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
• Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
• A Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids, in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
• A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
• Weekend Steroids and Exercise as Therapy for DMD
• Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)
• Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
• A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD)
• A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications
• Exploratory Study of NS-089/NCNP-02 in DMD
• Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
• Weekly Steroids in Muscular Dystrophy
• Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
• Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
• Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
• A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)
• A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy
• Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
• Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
• An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
• Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
• A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)
• Bisoprolol in DMD Early Cardiomyopathy
• Spironolactone Versus Prednisolone in DMD
• A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
• Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy
• An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
• Gene Transfer Clinical Trial for LGMD2E (Beta-sarcoglycan Deficiency) Using scAAVrh74.MHCK7.hSGCB
• A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
• A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)
• First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect
• Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E)
• Safety and Efficacy of P-188 NF in DMD Patients
• An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
• Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy
• A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
• A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
• A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
• Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
• Tamoxifen in Duchenne Muscular Dystrophy
• Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
• Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy
• (-)- Epicatechin Becker Muscular Dystrophy
• Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping
• Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
• Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
• Study of Testosterone and rHGH in FSHD
• Tadalafil in Becker Muscular Dystrophy
• Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
• Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents
• Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD)
• Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
• Screening in Oculopharyngeal Muscular Dystrophy
• Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD)
• Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
• Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy
• Treatment Effect of Tamoxifen on Patients With DMD
• Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy
• Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy
• A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
• An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
• A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
• Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
• Rimeporide in Patients With Duchenne Muscular Dystrophy
• Study of DS-5141b in Patients With Duchenne Muscular Dystrophy
• The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy
• Extension Study of Drisapersen in DMD Subjects
• Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
• Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy
• Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
• Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients
• HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
• Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin
• Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
• Study of SRP-4045 and SRP-4053 in Participants With Duchenne Muscular Dystrophy (DMD)
• HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne)
• Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
• Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy
• Sodium Nitrate for Muscular Dystrophy
• Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial
• Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
• Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet
• Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy
• Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy
• Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
• Continuation Protocol to Protocol BBCO-001
• Phase I/II Study of SRP-4053 in DMD Patients
• A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
• An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort
• Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
• Study of Eteplirsen in DMD Patients
• A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
• A Study of TAS-205 for Duchenne Muscular Dystrophy
• Tadalafil in Becker Muscular Dystrophy
• Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy
• An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
• Exploratory Study of NS-065/NCNP-01 in DMD
• A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD)
• A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy
• L-citrulline and Metformin in Becker's Muscular Dystrophy
• Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
• L-citrulline and Metformin in Duchenne's Muscular Dystrophy
• Cardiomyopathy in DMD: Lisinopril vs. Losartan
• Open Label Extension Study of HT-100 in Patients With DMD
• Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA
• A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD)
• Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy
• A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route o
• Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
• Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy
• A Study of Tadalafil for Duchenne Muscular Dystrophy
• Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study)
• Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy
• Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
• Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy
• Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
• A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
• A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
• Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy
• Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy
• Finding the Optimum Regimen for Duchenne Muscular Dystrophy
• PDE Inhibitors in DMD Study (Acute Dosing Study)
• Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada
• CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers
• Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
• Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy
• Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
• To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20
• A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
• Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
• Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
• Tadalafil and Sildenafil for Duchenne Muscular Dystrophy
• Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients
• Safety Study of Flavocoxid in Duchenne Muscular Dystrophy
• A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
• Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
• Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
• Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy
• Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy
• Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
• A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
• Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies
• Tadalafil in Becker Muscular Dystrophy
• Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
• Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)
• Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD)
• Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
• Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
• Ramipril Versus Carvedilol in Duchenne and Becker Patients
• Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)
• Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)
• Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy
• The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy
• Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
• Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
• CoQ10 and Prednisone in Non-Ambulatory DMD
• Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy
• Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy
• Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy
• Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
• High-dose Prednisone in Duchenne Muscular Dystrophy
• Study Evaluating MYO-029 in Adult Muscular Dystrophy
• Pentoxifylline in Duchenne Muscular Dystrophy
• KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
• An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy
• Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)
• A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)
• Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
• Gentamicin Treatment of Muscular Dystrophy
• Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy
• Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy