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.
Last update August 2022
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 |
|---|---|---|---|---|---|---|---|---|
| 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 |
| Cardiac MRI Biomarkers and Genotype-Phenotype Correlations | Observational | UCLA | 7-21 | Either | No | No specific requirement | United States | NCT02834650 |
| CIFFREO (PF-06939926) | Restoring or Replacing Dystrophin | Pfizer | 4-7 | Ambulatory | No | Currently using | United States + Other | Pipeline NCT04281485 |
| Duchenne Registry | Observational | PPMD | All | Either | No | No specific requirement | United States + Other | Duchenne Registry.orgNCT02069756 |
| EMBARK (SRP-9001) | Restoring or Replacing Dystrophin | Sarepta Therapeutics | 4-7 | Ambulatory | Yes | Currently using | United States | PipelineNCT05096221 |
| 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 |
| HOPE-3 (CAP-1002) | Protecting the Muscle | Capricor | 10+ | Either | No | Currently using | United States | PipelineNCT05126758 |
| LENTALOS TWO (Pamrevlumab) | Combatting Fibrosis | FibroGen, Inc. | 6-11 | Ambulatory | no | Currently using | United States + Other | PipelineNCT04632940 |
| 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 |
| 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 | PipelineNCT04004065 |
| 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.
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Ongoing
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 Trials.gov |
| Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD | Observational | Nationwide Children's Hospital | Clinical Trials.gov |
| Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy | Combating Fibrosis | Italfarmaco SpA | Pipeline |
| Epidys (Givnostat) | Combating Fibrosis | Italfarmaco SpA | Pipeline |
| ESSENCE (Vyondys/Casimersin) | Restoring or Replacing Dystrophin | Sarepta Therapeutics | Pipeline |
| Galactic53 (Viltolarsen) | Restoring or Replacing Dystrophin | NS Pharma | PipelineNCT04956289 |
| 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 |
| IGNITE DMD (SGT-001) | Restoring or Replacing Dystrophin | Solid Biosciences | PipelineNCT03368742 |
| LELANTOS (Pamrevlumab) | Combatting Fibrosis | FibroGen, Inc. | Pipeline |
| Long-Term Outcomes of Ataluren | Restoring or Replacing Dystrophin | PTC Therapeutics | Pipeline |
| MIS51ON (Safety and Efficacy of High-Dose Eteplirsen) | Restoring or Replacing Dystrophin | Sarepta Therapeutics | Pipeline |
| MOMENTUM (SRP-5051) | Restoring or Replacing Dystrophin | Sarepta Therapeutics | Pipeline |
| PF-06939926 Gene Therapy | Restoring or Replacing Dystrophin | Pfizer | Pipeline |
| PolarisDMD (Edasalonexent) | Reducing Inflammation | Catabasis Pharmaceuticals | Pipeline |
| Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy | Restoring the Cells Energy | Epirium Bio | Pipeline |
| Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping | Restoring or Replacing Dystrophin | Sarepta | Pipeline |
| Study to Assess the Efficacy and Safety of Vamorolone | Reducing Inflammation | Santhera Pharmaceuticals | 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 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 |
|---|---|---|---|---|
| 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 |
| HOPE-2 A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients | Improving Muscle Growth & Protection | Capricor Therapeutics | United States | Pipeline |
| Long-term Extension Study to Assess Vamorolone | Reducing Inflammation | Reveragen Biopharma | United States + Other | Pipeline |
| Study of Ataluren in Previously Treated Participants | Restoring or Replacing Dystrophin | PTC | United States + Other | Pipeline |
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.
