The following clinical trial and research study FAQ sheets are family-friendly summaries of clinical trials and research studies for people with Duchenne and Becker, as well as female carriers. There are also FAQ sheets for current pre-clinical research that is soon to be in clinical trial. These FAQ sheets are written in consultation directly with the Researchers for the program book of PPMD’s Annual Conference (held every June). Although we try to include the majority of studies in the United States, this is not intended to be a comprehensive list. Additional information may be found at ClinicalTrials.gov.

Updated May 2019

Actively Recruiting

The following trials are currently actively recruiting. Click any title to go to the study’s FAQ page.

StudyTherapeutic ApproachIndustry/ InstitutionAge (years)AmbulationMutation SpecificSteroid UseLocation
Ataluren - Long-Term Outcomes (041)Restoring or Replacing DystrophinPTC Therapeutics5+AmbulatoryYes - Nonsense mutationCurrently usingUnited States + Other
Ataluren - The Dystrophin Study (046) in participants currently taking atalurenRestoring or Replacing DystrophinPTC TherapeuticsAnyAmbulatoryYes - Nonsense mutationNo specific requirementUnited States
Cardiac MRI Biomarkers and Genotype-Phenotype CorrelationsObservationalNIH7-21EitherNoNo specific requirementUnited States
Dynamic Arm SupportRoboticsUniversity of Pittsburgh14+NonambulatoryNoNo specific requirementUnited States
Edasalonexent
(PolarisDMD trial)
Reducing InflammationCatabasis Pharmaceuticals4-7AmbulatoryNoNone in past 6 monthsUnited States + Other
Genetic ModifiersObservationalUCLAAnyEitherNoNo specific requirementUnited States
GivinostatImproving Muscle Growth & ProtectionItalfarmaco SpA6-17AmbulatoryNoCurrently usingUnited States + Other
Idebenone
(SIDEROS trial)
Restoring the Cells EnergySanthera Pharmaceuticals 10+EitherNoCurrently usingUnited States + Other
MNK-1411
(BRAVE study)
Reducing InflammationMallinckrodt Pharmaceuticals4-8AmbulatoryNoNever usedUnited States
PF-06939926 Mini-Dystrophin Gene TherapyRestoring or Replacing DystrophinPfizer5-12AmbulatoryNoCurrently usingUnited States
SGT-001 Micro-Dystrophin Gene Transfer
(IGNITE DMD trial)
Restoring or Replacing DystrophinSolid Biosciences4-11AmbulatoryNoCurrently usingUnited States
Spironolactone vs. PrednisoloneImproving Heart FunctionNationwide Children's Hospital4-7EithernonaïveUnited states
SRP-4045 and SRP-4053 - Exons 45 and 53 Skipping
(ESSENCE study)
Restoring or Replacing DystrophinSarepta Therapeutics7-13AmbulatoryYes - Deletion amenable to exon 45 & 53 SkippingCurrently usingUnited States + Other
SRP-9001 Micro-Dystrophin Gene TransferRestoring or Replacing DystrophinSarepta Therapeutics4-7AmbulatoryNoCurrently usingUnited States
VamoroloneReducing InflammationReveraGen Biopharma4-7AmbulatoryNoNever usedUnited States

Not Yet Recruiting

The following trials are not yet recruiting. Click any title to go to the study’s FAQ page.

StudyTherapeutic ApproachIndustry/ InstitutionAge (years)Ambulation StatusMutation SpecificSteroid UseLocation
IfetrobanImproving Heart FunctionCumberland Pharmaceuticals7+EitherNoStableUnited States
Suvodirsen - Exon 51 Skipping
(DYSTANCE 51 study)
Restoring or Replacing DystrophinWave Life Sciences5-12AmbulatoryYes - Deletion amenable to exon 51 skippingCurrently usingUnited States + Other

Ongoing

The following trials are active, but no longer recruiting participants. Click any title to go to the study’s FAQ page.

StudyTherapeutic ApproachIndustry/ Institution
Ataluren - The Dystrophin Study (045)Restoring or Replacing DystrophinPTC Therapeutics
CAP-1002
(HOPE-2 trial)
Improving Muscle Growth & ProtectionCapricor Therapeutics
Carrier StudyObservationalNationwide Children's Hospital
Duchenne Natural History StudyObservationalCINRG
Finding the Optimal Steroid Treatment
(FOR-DMD study)
ObservationalUniv of Rochester
PamrevlumabCombating FibrosisFibroGen
RG6206
(SPITFIRE study)
Improving Muscle Growth & ProtectionRoche/Genentech
Viltolarsen (Formerly NS-065/NCNP-01) - Exon 53 SkippingRestoring or Replacing DystrophinNS Pharma

Preclinical

The following are pre-clinical research. Click any title to go to the study’s FAQ page.

StudyTherapeutic ApproachIndustry/Institution
ARM210Regulating Calcium BalanceARMGO
Recombinant Human Laminin-111Improving Muscle Growth & ProtectionProthelia
iPS Cell TherapyImproving Muscle Growth & ProtectionUniversity of Minnesota
Wave Preclinical Exon SkippingRestoring or Replacing DystrophinWave Life Sciences