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.

Actively Recruiting

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

StudyTherapeutic ApproachIndustry/ InstitutionAge (years)Ambulation StatusMutation SpecificSteroid UseLocation
Assessment of Cardiopulmonary Function Using MRIObservationalUniversity of Florida5-15EitherNoNo specific requirementUnited States
AtalurenNonsense Mutation ReadthroughPTC5+AmbulatoryYes - Nonsense mutationCurrently usingUnited States + Other
CAP-1002
(HOPE-2 trial)
Cardiac Stem CellsCapricor10+EitherNoCurrently usingUnited States
Cardiac MRI Biomarkers and Genotype-Phenotype CorrelationsObservationalNIH7-21EitherNoNo specific requirementUnited States
Eteplirsen - Exon 51 Skipping(Study 4658-102)Exon SkippingSarepta6 mon - 24 monEitherYes - Deletion amenable to exon skippingNo specific requirementEurope
Genetic ModifiersObservationalUCLAAnyEitherNoNo specific requirementUnited States
GivinostatHDAC InhibitorItalfarmaco6-17AmbulatoryNoCurrently usingUnited States + Other
Idebenone
(SIDEROS trial)
Improve Cellular FunctionSanthera10+EitherNoCurrently usingUnited States + Other
MNK-1411
(BRAVE study)
Steroid AlternativeMallinckrodt4-8AmbulatoryNoNever usedUnited States
Nationwide GALGT2 Gene TherapyGene Therapy - GALGT2 UpregulationNationwide Children's Hospital8+AmbulatoryNoCurrently usingUnited States
Nationwide Micro-Dystrophin Gene DeliveryGene Therapy - MicrodystrophinNationwide Children's Hospital3 mon - 7 yrsAmbulatoryYes - OtherNo specific requirementUnited States
PF-06939926 Mini-Dystrophin Gene Therapy
(Formerly BMB-D001)
Gene Therapy - MinidystrophinPfizer5-12AmbulatoryNoCurrently usingUnited States
RG6206 Anti-Myostatin AdnectinMyostatin InhibitionRoche6-11AmbulatoryNoCurrently usingUnited States + Other
SGT-001 Micro-Dystrophin Gene Transfer
(IGNITE DMD trial)
Gene Therapy - MicrodystrophinSolid4-17EitherNoCurrently usingUnited States
SRP-4045 and SRP-4053 - Exons 45 and 53 Skipping
(ESSENCE study)
Exon SkippingSarepta7-13AmbulatoryYes - Deletion amenable to exon skippingCurrently usingOther
SRP-5051 - Exon 51 Skipping
(PPMO Study)
Exon SkippingSarepta12+EitherYes - Deletion amenable to exon skippingNo specific requirementUnited States
Vamorolone
(Formerly VBP15)
Steroid AlternativeReveraGen4-7AmbulatoryNoNever usedUnited States + Other
WVE-210201 - Exon 51 SkippingExon SkippingWAVE Life Sciences5-18EitherYes - Deletion amenable to exon skippingNo specific requirementUnited States + Other

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
Edasalonexent
(Formerly CAT-1004)
Steroid AlternativeCatabasis--------United States + Other
IfetrobanAnti-FibroticCumberland Pharmaceuticals--------United States

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
Domagrozumab
(Formerly PF-06252616)
Myostatin InhibitionPfizer
Ezutromid
(PhaseOut DMD study)
Utrophin UpregulationSummit
Finding the Optimal Steroid Treatment
(FOR-DMD Study)
ObservationalUniversity of Rochester
Natural History of BeckerObservationalCINRG
Natural History of DuchenneObservationalCINRG
NS-065/NCNP-01 - Exon 53 SkippingExon SkippingNS Pharma
Pamrevlumab
(Formerly FG-3019)
Anti-FibroticFibrogen

Preclinical

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

StudyTherapeutic ApproachIndustry/ Institution
ARM210Ryanodine ReceptorsARMGO
AT-300Calcium HomeostasisAkashi
Carmeseal-MDMuscle Cell ProtectionPhrixus
DT-200SARMAkashi
iPS Cell TherapyStem CellsUniversity of Minnesota
Nationwide Exon 2 Skipping for Duplication 2Exon SkippingNationwide Children's Hospital
Recombinant Human Laminin-111Utrophin UpregulationProthelia
TamoxifenAnti FibroticUniversity of Geneva
Wave Exon 53 SkippingExon SkippingWave Life Sciences

Female Carriers

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

StudyTherapeutic ApproachIndustry/ InstitutionAge (years)Ambulation StatusMutation SpecificSteroid UseLocation
Nationwide Carrier StudyObservationalNationwide Children's Hospital18+EitherNoNo specific requirementUnited States