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 April 2022

Actively Recruiting

The following trials are currently actively recruiting.

Study NameTherapeutic ApproachIndustry/ InstitutionAge (years)AmbulationMutation SpecificSteroid UseLocationAdditional Information
ASP0367Restoring the Cells EnergyAstellas Pharma8-16EithernoCurrently usingUnited StatesPipelineNCT04184882
Ataluren in Participants From ≥6 Months to <2 Years of AgeRestoring or Replacing DystrophinPTC0.5-2EitherYes - Nonsense MutationNo specific requirementUnited StatesPipelineNCT04336826
Cardiac MRI Biomarkers and Genotype-Phenotype CorrelationsObservationalUCLA7-21EitherNoNo specific requirementUnited StatesNCT02834650
Duchenne RegistryObservationalPPMDAllEitherNoNo specific requirementUnited States + OtherDuchenne
Registry.org
NCT02069756
EMBARK (SRP-9001)Restoring or Replacing DystrophinSarepta Therapeutics4-7AmbulatoryYesCurrently usingUnited StatesPipelineNCT05096221
Extracellular RNA Biomarkers of DMDObservationalBoston Children's Hosptial5+EitherNoNaïveUnited StatesNCT05016908
FIGHTDMD (Ifetroban)Improving Heart FunctionCumberland Pharmaceuticals 7+EitherNoNo specific requirementUnited StatesPipelineNCT03340675
Galactic53 (Viltolarsen)Restoring or Replacing DystrophinNS Pharma8+EitherYes - Deletion amenable to exon 53 skippingCurrently usingUnited States + OtherPipelineNCT04956289
IGNITE DMD (SGT-001)Restoring or Replacing DystrophinSolid Biosciences4-17EitherNoCurrently usingUnites StatesPipelineNCT03368742
LENTALOS TWO (Pamrevlumab)Combatting FibrosisFibroGen, Inc.6-11AmbulatorynoCurrently usingUnited States + OtherPipelineNCT04632940
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
MOMENTUM (SRP-5051) Restoring or Replacing DystrophinSarepta Therapeutics4-21EitherYes - Deletion amenable to exon 51 skippingNo specific requirementUnited States + OtherPipelineNCT04004065
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
Wearable Technology to Assess Gait FunctionObservationalColumbia University5+AmbulatoryNoNo specific requirementUnited StatesNCT04193085

Not Yet Recruiting

The following trials are not yet recruiting.

Study NameTherapeutic ApproachIndustry/ InstitutionAge (years)Ambulation StatusMutation SpecificSteroid UseAdditional Information
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
Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular DystrophyCombating FibrosisItalfarmaco SpAPipeline
Epidys (Givnostat)Combating FibrosisItalfarmaco SpAPipeline
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
IGNITE DMD Microdystrophin Gene Transfer Study in Adolescents and Children With DMDRestoring or Replacing DystrophinSolid BiosciencesPipeline
LELANTOS (Pamrevlumab)Combatting FibrosisFibroGen, Inc.Pipeline
Long-Term Outcomes of AtalurenRestoring or Replacing DystrophinPTC TherapeuticsPipeline
MIS51ON (Safety and Efficacy of High-Dose Eteplirsen)Restoring or Replacing DystrophinSarepta TherapeuticsPipeline
MOMENTUM (SRP-5051)Restoring or Replacing DystrophinSarepta TherapeuticsPipeline
PF-06939926 Gene Therapy Restoring or Replacing DystrophinPfizerPipeline
PolarisDMD (Edasalonexent)Reducing InflammationCatabasis PharmaceuticalsPipeline
Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular DystrophyRestoring the Cells EnergyEpirium BioPipeline
Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 SkippingRestoring or Replacing DystrophinSareptaPipeline
Study to Assess the Efficacy and Safety of VamoroloneReducing InflammationSanthera PharmaceuticalsPipeline
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
HOPE-2 A Study of CAP-1002 in Ambulatory and Non-Ambulatory PatientsImproving Muscle Growth & ProtectionCapricor TherapeuticsUnited StatesPipeline
Long-term Extension Study to Assess VamoroloneReducing InflammationReveragen BiopharmaUnited 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.