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.
Actively Recruiting
The following trials are currently actively recruiting.
| Study Name | Therapeutic Approach | Industry/ Institution | Age (years) | Ambulation | Mutation Specific | Steroid Use | Location | Additional Information |
|---|---|---|---|---|---|---|---|---|
| AFFINITY DUCHENNE (RGX-202) | Restoring or Replacing Dystrophin | REGENXBIO | 4-11 | Ambulatory | Yes - Exon 18-79 | Currently using | United States | PipelineNCT05693142 |
| Ataluren in Participants From ≥6 Months to <2 Years of Age | Restoring or Replacing Dystrophin | PTC | 0.5-2 | Either | Yes – Nonsense Mutation | No specific requirement | United States | PipelineNCT04336826 |
| Canyon (EDG-5506 in Becker) | Protecting the Muscle | Edgewise Therapeutics | 12-50 | Ambulatory | Yes – Becker | No specific requirement | United States | NCT05291091 |
| DELIVER (DYNE-251) | Restoring or Replacing Dystrophin | Dyne Therapeutics | 4-16 | Either | Yes - Amenable to skipping exon 51 | Currently using | United States + Other | Pipeline NCT05524883 |
| Duchenne Registry | Observational | PPMD | All | Either | No | No specific requirement | United States + Other | Duchenne Registry.orgNCT02069756 |
| Extracellular RNA Biomarkers of DMD | Observational | Boston Children's Hosptial | 5+ | Either | No | Naïve | United States | NCT05016908 |
| FIGHTDMD (Ifetroban) | Improving Heart Function | Cumberland Pharmaceuticals | 7+ | Either | No | No specific requirement | United States | PipelineNCT03340675 |
| Fordadistrogene Movaparvovec in Early Stage Duchenne | Restoring or Replacing Dystrophin | Pfizer | 2-4 | Not Required | Yes | Naïve | United States | PipelineNCT05429372 |
| HOPE-3 (CAP-1002) | Protecting the Muscle | Capricor | 10+ | Either | No | Currently using | United States | PipelineNCT05126758 |
| INSPIRE DUCHENNE (SGT-003) | Restoring or Replacing Dystrophin | Solid Biosciences | 4-7 | Ambulatory | Yes – Deletion mutation in exons 1-11 or 42-45 | Currently using | United States | PipelineNCT06138639 |
| LYNX (EDG-5506) | Improving Muscle Growth & Protection | Edgewise Therapeutics | 4-9 | Ambulatory | No | Currently using | United States + Other | Pipeline NCT05540860 |
| 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 | PipelineNCT03992430 |
| Molecular Analysis of Patients With Neuromuscular Disease | Observational | Boston Children's Hosptial | All | Either | No | No specific requirement | United States | NCT00390104 |
| MRI and Biomarkers for Muscular Dystrophy | Observational | University of Florida | 5-18 | Either | No | No specific requirement | United States | NCT01484678 |
| Pilot Trial of Canakinumab (Ilaris) | Reducing Inflammation | Children's Research Institute | 2-5 | Ambulatory | No | Currently using | United States | PipelineNCT03936894 |
| RACER53 (Viltolarsen) | Restoring or Replacing Dystrophin | NS Pharma | 4-7 | Ambulatory | Yes – Deletion amenable to exon 53 skipping | Currently using | United States + Other | PipelineNCT04060199 |
| Sleep Intervention in Young Boys With DMD | Observational | University of Pittsburgh | 6-18 | Either | No | No specific requirement | United States | NCT04529707 |
| Tadalafil as Adjuvant Therapy for DMD | Improving Muscle Growth & Protection | University of Florida | 7-13 | Ambulatory | No | No specific requirement | United States | NCT05195775 |
| Twice Weekly Steroids and Exercise as Therapy for DMD | Observational | University of Florida | 5-8 | Ambulatory | No | No specific requirement | United States | NCT04322357 |
| Vamorolone in Becker | Reducing Inflammation | ReveraGen | 18-64 | Ambulatory | Yes – Becker | No specific requirement | United States | PipelineNCT05166109 |
| Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years | Reducing Inflammation | ReveraGen | 2 to 17 | Either | No | Varies by age cohort | United States + Other | PipelineNCT05185622 |
| Wearable Technology to Assess Gait Function | Observational | Columbia University | 5+ | Ambulatory | No | No specific requirement | United States | NCT04193085 |
Not Yet Recruiting
The following trials are not yet recruiting.
no trials to display
Ongoing
The following trials are active, but no longer recruiting participants.
| Study Name | Therapeutic Approach | Industry/ Institution | Additional Information |
|---|---|---|---|
| Canakinumab | Reducing Inflammation | Children's National Hospital | Clinical Trials.gov |
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 |
Definitions
- 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.
