Clinical Trials

The table below contains those trials currently enrolling Duchenne patients and involving active drugs, as of the date indicated.

A more complete listing of Duchenne trials may be found at

ResearchCompanyCompoundStatusIndicationLocationClinical Trial
Anti-fibroticFibrogenPamrevlumabPhase 3Males with Duchenne, ages 12 years and older, and non-ambulatoryUSVIEW DETAILS
Anti-inflammatoryChildren’s Research InstituteIlarisPhase 1/2Males with Duchenne 2-5 years old & ambulatoryWashington DCVIEW DETAILS
Nationwide Children’s HospitalSpironolactonePhase 1Males with Duchenne 4-7 years oldOhioVIEW DETAILS
University HospitalTamoxifenPhase 3Males with Duchenne 6 1/2 – 16 years oldEUVIEW DETAILS
Cardiac FunctionCumberland PharmaceuticalsIfetrobanPhase 2
Males with Duchenne, ages 7 years and older
Peking Union Medical College HospitalBisoprolol FumaratePhase 2/3
Males with Duchenne, ages 7 years and older
Cell TherapyCHU de Quebec-Universite LavalMyoblast transplantationPhase 1/2Males with Duchenne, ages 16 years and olderCAVIEW DETAILS
Exon SkippingAudentes TherapeuticsAAV9 U7snRNAPhase 1/2Males with Duchenne 6 months – 14 years old, pre-ambulant or ambulant, with a duplication of exon 2.OhioVIEW DETAILS
NS PharmaViltolarsenPhase 3Males with Duchenne 4-7 years old, ambulatory, & amenable to exon 53 skipping.US, JPVIEW DETAILS
NS PharmaNS-089/NCNP-02Phase 1/2Males with Duchenne 4-17 years old, ambulatory, & amenable to exon 44 skipping.JPVIEW DETAILS
Sarepta TherapeuticsCasimersen & GolodirsenPhase 3Males with Duchenne ages 7-13, amenable to exon 45 or exon 53 skipping, & ambulatoryUS, EU, CA, IL, AUVIEW DETAILS
Sarepta TherapeuticsSRP-5051Phase 2Males with Duchenne ages 4-21 years old, amenable to exon 51 skippingUS, CAVIEW DETAILS
Sarepta TherapeuticsCasimersen, Eteplirsen, & GolodirsenPhase 2Males with Duchenne 6 months and older who have a single exon duplication of either exon 45, 51, or 53.OhioVIEW DETAILS
Sarepta TherapeuticsEteplirsenPhase 3Males with Duchenne ages 7-13, ambulatory, & amenable to exon 51 skipping.CAVIEW DETAILS
Gene TherapyPfizerPF-06939926Phase 3Males with Duchenne 4-7 years oldUSVIEW DETAILS
Mitochondrial FunctionAstellasASP0367Phase 1bMales with Duchenne, 8-16 years oldUSVIEW DETAILS
Stop Codon ReadthroughPTC TherapeuticsAtalurenPhase 3Males with Duchenne 5 Years and older, a nonsense point mutation, & ambulatoryUS, AU, CA, MX, HK, JP, IN, KR, MY, RU, TW, CN, JOVIEW DETAILS
PTC TherapeuticsAtalurenPhase 2
Males with Duchenne, ages 6 months to 2 years with a nonsense mutation

Chart Updated 11/25/2020

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