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 for the program book of the PPMD Annual Conference (held every June) in consultation directly with the Researchers. Although we try to include the majority of studies in the United States, this is not intended to be a comprehensive list.

Preclinical

Click any title to go to the study’s FAQ page (when available).

StudyTherapeutic ApproachIndustry/Institution
ARM210Ryanodine ReceptorsARMGO
AT-300Calcium HomeostasisAkashi
BiglycanUtrophin UpregulationTivorsan
Carmeseal-MDMuscle Cell ProtectionPhrixus
DT-200SARMAkashi
Exon 2 Skipping for Dup2 PatientsExon SkippingNationwide Children's Hospital
iPS Cell TherapyStem CellsDr. Rita Perlingeiro (University of Minnesota)
Laminin-111Utrophin UpregulationProthelia
MA-0211Improve Cellular FunctionMitobridge/Astellas
NBD PeptideAnti-fibroticsDr. Denis Guttridge (Ohio State University)
RTC13Nonsense Mutation ReadthroughDr. Carmen Bertoni (UCLA)
TamoxifenMuscle Growth & ProtectionUniversity of Geneva
WAVE Exon 51 and 53 SkippingExon SkippingWAVE Life Sciences

Enrolling

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

StudyTherapeutic ApproachIndustry/ InstitutionAge (years)Ambulation StatusMutation SpecificSteroid UseLocation
Assessment of Cardiopulmonary FunctionObservationalUniversity of Florida5-15EitherNoNoUnited States
AtalurenStop Codon ReadthroughPTC5+AmbulatoryYes - Nonsense mutationCurrently usingUnited States + Other
Cardiac MRI BiomarkersObservationalNIH7-21EitherNoNo specific requirementUnited States
Exon 45 and 53 Skipping/ESSENCEExon SkippingSarepta7-13AmbulatoryYes - Deletion amenable to exon skippingCurrently usingUnited States + Other
Exon 51 SkippingExon SkippingSarepta12+EitherYes - Deletion amenable to exon skippingNo specific requirementUnited States
FG-3019Anti-FibroticFibrogen12+Non-ambulatoryNoCurrently usingUnited States
GALGT2 Gene TransferGALGT2 UpregulationNationwide Children's Hospital8+AmbulatoryNoCurrently usingUnited States
Genetic ModifiersObservationalUCLAAnyEitherNoNo specific requirementUnited States
GivinostatHDAC Inhibitor Italfarmaco6-17AmbulatoryNoCurrently usingUnited States + Other
Idebenone/SIDEROSImprove Cellular FunctionSanthera10+EitherNoCurrently usingUnited States + Other
Micro-dystrophin Gene TherapyGene TherapyNationwide Children's Hospital3 mon - 7 yrsAmbulatoryYes - OtherNo specific requirementUnited States
Micro-dystrophin Gene TherapyGene TherapySolid4-17EitherNoCurrently usingUnited States
Mini-dystrophin Gene TherapyGene TherapyPfizer5-12AmbulatoryNoCurrently usingUnited States
MNK-1411/BRAVEMallinckrodt4-8AmbulatoryNoNever usedUnited States
MRI and BiomarkersObservationalUniversity of Florida5-18EitherNoNo specific requirementUnited States
Peak Cough Flow and Cough ClearanceObservationalRespirtech and University of Pittsburgh6-21EitherNoNo specific requirementUnited States
Skeletal Muscle Blood Flow (Becker)PDE5 InhibitorCedars-Sinai Medical Center18-45EitherNoNo specific requirementUnited States
VamoroloneSteroid ReplacementReveraGen4-7AmbulatoryNoNever usedUnited States
(-)- Epicatechin (Becker)Mitochondrial BiogenesisCardero18-70AmbulatoryNoNo specific requirementUnited States

Ongoing

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

StudyTherapeutic ApproachIndustry/Institution
Aldosterone InhibitionTraditional Cardiac DrugsOhio State University
Ataluren Extension StudyStop Codon ReadthroughPTC
Ataluren in 2-5 year oldsStop Codon ReadthroughPTC
BMS-986089Myostatin InhibitionRoche
Confirmatory Study of Eteplirsen (PROMOVI)Exon SkippingSarepta
Eteplirsen (Advanced Stage Duchenne)Exon SkippingSarepta
Eteplirsen (Early Stage Duchenne)Exon SkippingSarepta
Exon 45 Skipping (Advanced Stage Duchenne)Exon SkippingSarepta
Ezutromid (PhaseOut DMD)Utrophin UpregulationSummit
FOR-DMDObservationalUniversity of Rochester
HOPE-DuchenneCardiac Stem CellsCapricor
MoveDMDSteroid ReplacementCatabasis
Natural History of BeckerObservationalCINRG
Natural History of DuchenneObservationalCINRG
NS-065/NCNP-01 Exon 53 SkippingExon SkippingNS Pharma
PF-06252616Myostatin InhibitionPfizer
Strength Training in DuchenneObservationalUniversity of Florida

Female Carriers

Click any title to go to the study’s FAQ page (when available).

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