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 Info
Cardiac MRI Biomarkers and Genotype-Phenotype CorrelationsObservationalUCLA7-21EitherNoNo specific requirementUnited StatesClinical
Trials.gov
Duchenne RegistryObservationalPPMDAllEitherNoNo specific requirementUnited States + OtherDuchenne
Registry.org
Epidys (Givnostat)Combating FibrosisItalfarmaco SpA6-17AmbulatoryNoCurrently usingUnited States + OtherPipeline
FIGHTDMD (Ifetroban)Improving Heart FunctionCumberland Pharmaceuticals 7+EitherNoNo specific requirementUnited StatesPipeline
Long-Term Outcomes of AtalurenRestoring or Replacing DystrophinPTC Therapeutics5+AmbulatoryYes - Nonsense mutationCurrently usingUnited States + OtherPipeline
MIS51ON (Safety and Efficacy of High-Dose Eteplirsen)Restoring or Replacing DystrophinSarepta Therapeutics7-13AmbulatoryYes - Amenable to skipping exon 51Currently usingUnited States + OtherPipeline
Molecular Analysis of Patients With Neuromuscular DiseaseObservationalBoston Children's HosptialAllEitherNoNo specific requirementUnited StatesClinical
Trials.gov
MOMENTUM (SRP-5051)Restoring or Replacing DystrophinSarepta Therapeutics4-21EitherYes - Deletion amenable to exon 51 skippingNo specific requirementUnited States + OtherPipeline
Monitor Activity Using Wearable Sensors
ObservationalNationwide Children's Hospital4-12AmbulatoryNoCurrently usingUnited statesClinical
Trials.gov
MRI and Biomarkers for Muscular DystrophyObservationalUniversity of Florida5-18EitherNoNo specific requirementUnited StatesClinical
Trials.gov
PF-06939926 Gene TherapyRestoring or Replacing DystrophinPfizer4-12AmbulatoryNoCurrently usingUnited StatesPipeline
Pilot Trial of CanakinumabReducing InflammationChildren's Research Institute2-5AmbulatoryNoNaïveUnited StatesPipeline
RACER53 (Viltolarsen)Restoring or Replacing DystrophinNS Pharma4-7AmbulatoryYes - Deletion amenable to exon 53 skippingCurrently usingUnited States + OtherPipeline
Spironolactone Versus PrednisoloneImproving Muscle Growth & ProtectionNationwide Children's Hospital4-7AmbulatoryNoNaïveUnited StatesPipeline
Study to Assess the Efficacy and Safety of VamoroloneReducing InflammationReveraGen Biopharma4-7AmbulatoryNoNaïveUnited States + OtherPipeline
The Burden of Access in DMDObservationalUniversity of Florida18+--No--United StatesClinical
Trials.gov
Wearable Technology to Assess Gait FunctionObservationalColumbia University5+AmbulatoryNoNo specific requirementUnited StatesClinical
Trials.gov

Not Yet Recruiting

The following trials are not yet recruiting.

Study NameTherapeutic ApproachIndustry/ InstitutionAge (years)Ambulation StatusMutation SpecificSteroid UseAdditional Information
A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211)Restoring the Cells EnergyAstellas Pharma8-16EitherNoCurrently UsingPipeline
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense MutationRestoring or Replacing DystrophinPTC Therapeutics6 months - 2 yrs--Yes - Nonsense MutationNaïvePipeline
CIFFREO (A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926)Restoring or Replacing DystrophinPfizer4-7AmbulatoryNoCurrently UsingPipeline
Weekend Steroids and Exercise as Therapy for DMDObservationalUniversity of Florida5-8AmbulatoryNoYesClinical
Trials.gov

Ongoing

The following trials are active, but no longer recruiting participants.

Study NameTherapeutic ApproachIndustry/ InstitutionAdditional Information
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001Restoring or Replacing DystrophinSareptaPipeline
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular DystrophyRestoring or Replacing DystrophinPTC TherapeuticsPipeline
AAV9 U7snRNA Gene Therapy to Treat DMD Exon 2 DuplicationsRestoring or Replacing DystrophinNationwide Children's HospitalClinical
Trials.gov
Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMDObservationalNationwide Children's HospitalClinical
Trials.gov
ESSENCE (Vyondys/Casimersin)Restoring or Replacing DystrophinSarepta TherapeuticsPipeline
Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2Restoring or Replacing DystrophinNationwide Children's HospitalPipeline
Givinostat in Patients with Becker Muscular DystrophyCombating FibrosisItalfarmaco SpAPipeline
HOPE-2 A Study of CAP-1002 in Ambulatory and Non-Ambulatory PatientsImproving Muscle Growth & ProtectionCapricor TherapeuticsPipeline
IGNITE DMD Microdystrophin Gene Transfer Study in Adolescents and Children With DMDRestoring or Replacing DystrophinSolid BiosciencesPipeline
PolarisDMD (Edasalonexent)Reducing InflammationCatabasis PharmaceuticalsPipeline
Safety and tolerability of PF-06939926Restoring or Replacing DystrophinPfizerPipeline
SIDEROS (Idebenone)Restoring the Cells EnergySanthera PharmaceuticalsPipeline
Study of DS-5141bRestoring or Replacing DystrophinDaiichi SankyoPipeline
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingRestoring or Replacing DystrophinSareptaPipeline
Systemic Gene Delivery Clinical Trial for Duchenne Muscular DystrophyRestoring or Replacing DystrophinSareptaPipeline
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory SubjectsCombating FibrosisFibroGenPipeline
Use of Dynamic Arm Supports to Promote Activities of Daily Living in Individuals With DMDRoboticsUniversity of PittsburghClinical
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.

StudyTherapeutic ApproachIndustry/ InstitutionLocationAdditional Info
A Study to Evaluate Safety, Tolerability, and Efficacy of EteplirsenRestoring or Replacing DystrophinSareptaUnited States + OtherPipeline
An Extension Study to Evaluate Casimersen or GolodirsenRestoring or Replacing DystrophinSareptaUnited States + OtherPipeline
An Open-Label Extension Study for SRP-5051Restoring or Replacing DystrophinSareptaUnited StatesPipeline
Extension Study of NS-065/NCNP-01Restoring or Replacing DystrophinNS PharmaUnited States + OtherPipeline
Galaxy DMD (Open-Label Extension Study of Edasalonexent)Reducing InflammationCatabasisUnited States + OtherPipeline
Givinostat Long-term Safety and Tolerability StudyCombating FibrosisItalfarmaco SpAUnited States + OtherPipeline
Long-term Extension Study to Assess VamoroloneReducing InflammationReveragen BiopharmaUnited States + OtherPipeline
Sideros-E (Idebenone)Restoring the Cells EnergySanthera PharmaceuticalsUnited States + OtherPipeline
Study of Ataluren in Previously Treated ParticipantsRestoring or Replacing DystrophinPTCUnited States + OtherPipeline

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.

Last update June 2020