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 NameTherapeutic ApproachIndustry/ InstitutionAge (years)AmbulationMutation SpecificSteroid UseLocationAdditional Information
AFFINITY DUCHENNE (RGX-202)Restoring or Replacing DystrophinREGENXBIO4-11AmbulatoryYes - Exon 18-79Currently usingUnited StatesPipelineNCT05693142
Ataluren in Participants From ≥6 Months to <2 Years of AgeRestoring or Replacing DystrophinPTC0.5-2EitherYes – Nonsense MutationNo specific requirementUnited StatesPipelineNCT04336826
Canyon
(EDG-5506 in Becker)
Protecting the MuscleEdgewise Therapeutics12-50AmbulatoryYes – BeckerNo specific requirementUnited StatesNCT05291091
DELIVER (DYNE-251)Restoring or Replacing DystrophinDyne Therapeutics4-16EitherYes - Amenable to skipping exon 51Currently usingUnited States + OtherPipeline NCT05524883
Duchenne RegistryObservationalPPMDAllEitherNoNo specific requirementUnited States + OtherDuchenne
Registry.org
NCT02069756
Extracellular RNA Biomarkers of DMDObservationalBoston Children's Hosptial5+EitherNoNaïveUnited StatesNCT05016908
FIGHTDMD (Ifetroban)Improving Heart FunctionCumberland Pharmaceuticals 7+EitherNoNo specific requirementUnited StatesPipelineNCT03340675
Fordadistrogene Movaparvovec in Early Stage DuchenneRestoring or Replacing DystrophinPfizer2-4Not RequiredYesNaïveUnited StatesPipelineNCT05429372
HOPE-3
(CAP-1002)
Protecting the MuscleCapricor10+EitherNoCurrently usingUnited StatesPipelineNCT05126758
LYNX (EDG-5506)Improving Muscle Growth & ProtectionEdgewise Therapeutics4-9AmbulatoryNoCurrently usingUnited States + OtherPipeline NCT05540860
MIS51ON (Safety and Efficacy of High-Dose Eteplirsen)Restoring or Replacing DystrophinSarepta Therapeutics7-13AmbulatoryYes – Amenable to skipping exon 51Currently usingUnited States + OtherPipelineNCT03992430
Molecular Analysis of Patients With Neuromuscular DiseaseObservationalBoston Children's HosptialAllEitherNoNo specific requirementUnited StatesNCT00390104
MRI and Biomarkers for Muscular DystrophyObservationalUniversity of Florida5-18EitherNoNo specific requirementUnited StatesNCT01484678
Pilot Trial of Canakinumab (Ilaris)Reducing InflammationChildren's Research Institute2-5AmbulatoryNoCurrently usingUnited StatesPipelineNCT03936894
RACER53 (Viltolarsen)Restoring or Replacing DystrophinNS Pharma4-7AmbulatoryYes – Deletion amenable to exon 53 skippingCurrently usingUnited States + OtherPipelineNCT04060199
Sleep Intervention in Young Boys With DMDObservationalUniversity of Pittsburgh6-18EitherNoNo specific requirementUnited StatesNCT04529707
Tadalafil as Adjuvant Therapy for DMDImproving Muscle Growth & ProtectionUniversity of Florida7-13AmbulatoryNoNo specific requirementUnited StatesNCT05195775
Twice Weekly Steroids and Exercise as Therapy for DMDObservationalUniversity of Florida5-8AmbulatoryNoNo specific requirementUnited StatesNCT04322357
Vamorolone in BeckerReducing InflammationReveraGen18-64AmbulatoryYes – BeckerNo specific requirementUnited StatesPipelineNCT05166109
Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 YearsReducing InflammationReveraGen2 to 17EitherNoVaries by age cohortUnited States + OtherPipelineNCT05185622
Wearable Technology to Assess Gait FunctionObservationalColumbia University5+AmbulatoryNoNo specific requirementUnited StatesNCT04193085

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 NameTherapeutic ApproachIndustry/ InstitutionAdditional Information

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.

StudyTherapeutic ApproachIndustry/ InstitutionLocationAdditional 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.