Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne muscular dystrophy. Clinical trials are research studies in humans designed to study if an experimental therapy or treatment works. The importance of this research must be balanced with the need for each family to carefully decide if trial participation is right for them.
The following trials are currently actively recruiting.
|Study Name||Therapeutic Approach||Industry/ Institution||Age (years)||Ambulation||Mutation Specific||Steroid Use||Location||Additional Info|
|Cardiac MRI Biomarkers and Genotype-Phenotype Correlations||Observational||UCLA||7-21||Either||No||No specific requirement||United States||Clinical
|Duchenne Registry||Observational||PPMD||All||Either||No||No specific requirement||United States + Other||Duchenne
|Epidys (Givnostat)||Combating Fibrosis||Italfarmaco SpA||6-17||Ambulatory||No||Currently using||United States + Other||Pipeline|
|FIGHTDMD (Ifetroban)||Improving Heart Function||Cumberland Pharmaceuticals||7+||Either||No||No specific requirement||United States||Pipeline|
|Long-Term Outcomes of Ataluren||Restoring or Replacing Dystrophin||PTC Therapeutics||5+||Ambulatory||Yes - Nonsense mutation||Currently using||United States + Other||Pipeline|
|MIS51ON (Safety and Efficacy of High-Dose Eteplirsen)||Restoring or Replacing Dystrophin||Sarepta Therapeutics||7-13||Ambulatory||Yes - Amenable to skipping exon 51||Currently using||United States + Other||Pipeline|
|Molecular Analysis of Patients With Neuromuscular Disease||Observational||Boston Children's Hosptial||All||Either||No||No specific requirement||United States||Clinical
|MOMENTUM (SRP-5051)||Restoring or Replacing Dystrophin||Sarepta Therapeutics||4-21||Either||Yes - Deletion amenable to exon 51 skipping||No specific requirement||United States + Other||Pipeline|
|Monitor Activity Using Wearable Sensors||Observational||Nationwide Children's Hospital||4-12||Ambulatory||No||Currently using||United states||Clinical
|MRI and Biomarkers for Muscular Dystrophy||Observational||University of Florida||5-18||Either||No||No specific requirement||United States||Clinical
|PF-06939926 Gene Therapy||Restoring or Replacing Dystrophin||Pfizer||4-12||Ambulatory||No||Currently using||United States||Pipeline|
|Pilot Trial of Canakinumab||Reducing Inflammation||Children's Research Institute||2-5||Ambulatory||No||Naïve||United States||Pipeline|
|RACER53 (Viltolarsen)||Restoring or Replacing Dystrophin||NS Pharma||4-7||Ambulatory||Yes - Deletion amenable to exon 53 skipping||Currently using||United States + Other||Pipeline|
|Spironolactone Versus Prednisolone||Improving Muscle Growth & Protection||Nationwide Children's Hospital||4-7||Ambulatory||No||Naïve||United States||Pipeline|
|Study to Assess the Efficacy and Safety of Vamorolone||Reducing Inflammation||ReveraGen Biopharma||4-7||Ambulatory||No||Naïve||United States + Other||Pipeline|
|The Burden of Access in DMD||Observational||University of Florida||18+||--||No||--||United States||Clinical
|Wearable Technology to Assess Gait Function||Observational||Columbia University||5+||Ambulatory||No||No specific requirement||United States||Clinical
Not Yet Recruiting
The following trials are not yet recruiting.
|Study Name||Therapeutic Approach||Industry/ Institution||Age (years)||Ambulation Status||Mutation Specific||Steroid Use||Additional Information|
|A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211)||Restoring the Cells Energy||Astellas Pharma||8-16||Either||No||Currently Using||Pipeline|
|A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation||Restoring or Replacing Dystrophin||PTC Therapeutics||6 months - 2 yrs||--||Yes - Nonsense Mutation||Naïve||Pipeline|
|CIFFREO (A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926)||Restoring or Replacing Dystrophin||Pfizer||4-7||Ambulatory||No||Currently Using||Pipeline|
|Weekend Steroids and Exercise as Therapy for DMD||Observational||University of Florida||5-8||Ambulatory||No||Yes||Clinical
The following trials are active, but no longer recruiting participants.
|Study Name||Therapeutic Approach||Industry/ Institution||Additional Information|
|A Randomized, Double-blind, Placebo-controlled Study of SRP-9001||Restoring or Replacing Dystrophin||Sarepta||Pipeline|
|A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy||Restoring or Replacing Dystrophin||PTC Therapeutics||Pipeline|
|AAV9 U7snRNA Gene Therapy to Treat DMD Exon 2 Duplications||Restoring or Replacing Dystrophin||Nationwide Children's Hospital||Clinical
|Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD||Observational||Nationwide Children's Hospital||Clinical
|ESSENCE (Vyondys/Casimersin)||Restoring or Replacing Dystrophin||Sarepta Therapeutics||Pipeline|
|Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2||Restoring or Replacing Dystrophin||Nationwide Children's Hospital||Pipeline|
|Givinostat in Patients with Becker Muscular Dystrophy||Combating Fibrosis||Italfarmaco SpA||Pipeline|
|HOPE-2 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients||Improving Muscle Growth & Protection||Capricor Therapeutics||Pipeline|
|IGNITE DMD Microdystrophin Gene Transfer Study in Adolescents and Children With DMD||Restoring or Replacing Dystrophin||Solid Biosciences||Pipeline|
|PolarisDMD (Edasalonexent)||Reducing Inflammation||Catabasis Pharmaceuticals||Pipeline|
|Safety and tolerability of PF-06939926||Restoring or Replacing Dystrophin||Pfizer||Pipeline|
|SIDEROS (Idebenone)||Restoring the Cells Energy||Santhera Pharmaceuticals||Pipeline|
|Study of DS-5141b||Restoring or Replacing Dystrophin||Daiichi Sankyo||Pipeline|
|Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping||Restoring or Replacing Dystrophin||Sarepta||Pipeline|
|Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy||Restoring or Replacing Dystrophin||Sarepta||Pipeline|
|Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects||Combating Fibrosis||FibroGen||Pipeline|
|Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMD||Robotics||University of Pittsburgh||Clinical
Open Label Extension
Open Label Extension Trials typically follow a placebo trial, where all participants are now on drug and can be monitored for an additional period of time. Participating in a previous trial may be a requirement for being enrolled in an Open Label Extension.
|Study||Therapeutic Approach||Industry/ Institution||Location||Additional Info|
|A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen||Restoring or Replacing Dystrophin||Sarepta||United States + Other||Pipeline|
|An Extension Study to Evaluate Casimersen or Golodirsen||Restoring or Replacing Dystrophin||Sarepta||United States + Other||Pipeline|
|An Open-Label Extension Study for SRP-5051||Restoring or Replacing Dystrophin||Sarepta||United States||Pipeline|
|Extension Study of NS-065/NCNP-01||Restoring or Replacing Dystrophin||NS Pharma||United States + Other||Pipeline|
|Galaxy DMD (Open-Label Extension Study of Edasalonexent)||Reducing Inflammation||Catabasis||United States + Other||Pipeline|
|Givinostat Long-term Safety and Tolerability Study||Combating Fibrosis||Italfarmaco SpA||United States + Other||Pipeline|
|Long-term Extension Study to Assess Vamorolone||Reducing Inflammation||Reveragen Biopharma||United States + Other||Pipeline|
|Sideros-E (Idebenone)||Restoring the Cells Energy||Santhera Pharmaceuticals||United States + Other||Pipeline|
|Study of Ataluren in Previously Treated Participants||Restoring or Replacing Dystrophin||PTC||United States + Other||Pipeline|
- Combatting Fibrosis – Reducing the amount of scar tissue development that leads to muscle breakdown.
- Improving Heart Function – Strategies aiming to protect and improve cardiac muscle in Duchenne
- Improving Muscle Growth and Protection – Enhancing muscle growth while minimizing muscle breakdown
- Observational – Studies that aim to strengthen our understanding of the disease, these are studies that do not typically include an investigational product such as a drug.
- Reducing Inflammation – Lessening the chronic inflammation that is an effect of muscle degradation in Duchenne
- Regulating Calcium Balance – Control the flow of calcium in and out of the muscle that is disrupted by the damaged muscle cells
- Restoring or Replacing Dystrophin – Strategies that seek to address the absence of dystrophin protein in muscle.
- Restoring the Cells Energy – Promoting healthy mitochondria cells which can enhance muscle cell functions such as muscle repair
- Robotics – Utilization of devices to improve quality of life and assistance with activities of daily living.
Last update June 2020