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 ClinicalTrials.gov.

RESEARCH COMPANY COMPOUND STATUS INDICATION LOCATION CLINICAL TRIAL
Anti-fibrotic, Anti-Inflammatory, Regenerative Agent Italfarmaco Givinostat Phase 3 Males with Duchenne, 6-17 years old & ambulatory US, EU, CA VIEW DETAILS
Anti-inflammatory Catabasis Edasalonexent Phase 3 Males with Duchenne 4-7 years old US, EU, CA, IL, AU VIEW DETAILS
Children’s Research Institute Ilaris Phase 1/2 Males with Duchenne 2-5 years old & ambulatory Washington DC VIEW DETAILS
Mallinckrodt Cosyntropin Phase 2 Males with Duchenne 4-8 years old US, IL, EU, MX VIEW DETAILS
ReveraGen BioPharma Vamorolone Phase 2b Males with Duchenne 4-7 years old US, EU, CA, IL, AU, SE VIEW DETAILS
Nationwide Children’s Hospital Spironolactone Phase 1 Males with Duchenne 4-7 years old Ohio VIEW DETAILS
Exon Skipping Sarepta Therapeutics Exondys51 Phase 2 Males with Duchenne ages 6 to 48 months, amenable to exon 51 skipping EU VIEW DETAILS
Sarepta Therapeutics Casimersen & Golodirsen Phase 3 Males with Duchenne ages 7-13, amenable to exon 45 or exon 53 skipping, & ambulatory US, EU, CA, IL, AU VIEW DETAILS
Sarepta Therapeutics SRP-5051 Phase 1 Males with Duchenne ages 12 years and older, amenable to exon 51 skipping US, CA VIEW DETAILS
Gene Therapy Nationwide Children’s Hospital GALGT2 Phase 1/2 Males with Duchenne 4 years and older Ohio VIEW DETAILS
Pfizer PF-06939926 Phase 1 Males with Duchenne 5-12 years old US VIEW DETAILS
Sarepta  Therapeutics SRP-9001 Phase 2 Males with Duchenne 4-7 years old Ohio VIEW DETAILS
Solid Biosciences SGT-001 Phase 1/2 Males with Duchenne 4-17 years old Florida VIEW DETAILS
Membrane Sealant Phrixus Pharmaceuticals Carmeseal-MD Phase 1 Males with Duchenne 12-25 years old Ohio VIEW DETAILS
Mitochondrial Function Santhera Idebenone Phase 3 Males with Duchenne 10 years and older US, EU, IL, CH VIEW DETAILS
Muscle Regeneration Roche RO7239361 Phase 2/3 Males with Duchenne 6-11 years old US, EU, JP, AR, AU, CA VIEW DETAILS
Stop Codon Readthrough PTC Therapeutics Ataluren Phase 3 Males with Duchenne 5 Years and older, a nonsense point mutation, & ambulatory US, AU, CA, MX, HK, JP, IN, KR, MY, RU, TW, CN, JO VIEW DETAILS
PTC Therapeutics Ataluren Phase 2 Males with Duchenne ages 2-7 years old, nonsense point mutation US VIEW DETAILS

Chart updated on 5/17/19