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-ambulatoryUS, EUVIEW DETAILS
FibroGenPamrevlumabPhase 3Males with Duchenne 6-11 years old & ambulatoryUSVIEW DETAILS
Anti-inflammatoryChildren’s Research InstituteIlarisPhase 1/2Males with Duchenne 2 years and older & ambulatoryWashington DCVIEW DETAILS
Nationwide Children’s HospitalSpironolactonePhase 1Males with Duchenne 4-7 years oldOhioVIEW 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 – 13 years old, pre-ambulant or ambulant, with a duplication of exon 2OhioVIEW DETAILS
NS PharmaViltolarsenPhase 3Males with Duchenne 4-7 years old, ambulatory, & amenable to exon 53 skipping.US, CA, JP, EU, CL, KR, MX, NZ, RU, TW, TRVIEW 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 an out of frame duplication of either exon 45, 51, or 53.OhioVIEW DETAILS
Gene TherapyPfizerPF-06939926Phase 3Males with Duchenne 4-7 years oldUS, UK, CA, EU, IL, JP, KR, RU, TWVIEW DETAILS
Sarepta TherapeuticsSRP-9001Phase 1Males with Duchenne 4-7 years old, & ambulatoryUSVIEW DETAILS
Solid BiosciencesSGT-001Phase 1/2Males with Duchenne 4-17 years oldUSVIEW DETAILS
Mitochondrial FunctionAstellasASP0367Phase 1bMales with Duchenne, 8-16 years oldUSVIEW DETAILS
Muscle StabilizationEdgewise TherapeuticsEDG-5506Phase 1Healthy Volunteers or Males with Becker, 18-55 years oldTexasVIEW DETAILS
Stop Codon ReadthroughPTC TherapeuticsAtalurenPhase 2
Males with Duchenne, ages 6 months to 2 years with a nonsense mutation

Last updated: 4/15/2021

Share This Page

Make an Impact

You can advance the care, treatment and cure for Duchenne muscular dystrophy. Contributions in any amount can truly make a difference and can be credited to a fundraiser or event from the list below.