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, IL VIEW DETAILS
Anti-inflammatory Children’s Research Institute Ilaris Phase 1/2 Males with Duchenne 2-5 years old & ambulatory Washington DC VIEW DETAILS
ReveraGen BioPharma Vamorolone Phase 2b Males with Duchenne 4-7 years old US, EU, CA, IL, AU VIEW DETAILS
Nationwide Children’s Hospital Spironolactone Phase 1 Males with Duchenne 4-7 years old Ohio VIEW DETAILS
University Hospital Tamoxifen Phase 3 Males with Duchenne 6 1/2 – 16 years old EU VIEW DETAILS
Exon Skipping Audentes Therapeutics AAV9 U7snRNA Phase 1/2 Males with Duchenne 6 months – 14 years old, pre-ambulant or ambulant, with a duplication of exon 2. Ohio VIEW DETAILS
NS Pharma Viltolarsen Phase 3 Males with Duchenne 4-7 years old, ambulatory, & amenable to exon 53 skipping. US, JP VIEW DETAILS
NS Pharma NS-089/NCNP-02 Phase 1/2 Males with Duchenne 4-7 years old, ambulatory, & amenable to exon 44 skipping. JP 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 2 Males with Duchenne ages 4-21 years old, amenable to exon 51 skipping US, CA VIEW DETAILS
Sarepta Therapeutics Casimersen, Eteplirsen, & Golodirsen Phase 2 Males with Duchenne 6 months and older who have a single exon duplication of either exon 45, 51, or 53. Ohio VIEW DETAILS
Sarepta Therapeutics Eteplirsen Phase 3 Males with Duchenne ages 7-13, ambulatory, & amenable to exon 51 skipping. CA VIEW DETAILS
Gene Therapy Pfizer PF-06939926 Phase 3 Males with Duchenne 4-7 years old US VIEW DETAILS
Mitochondrial Function Astellas ASP0367 Phase 1 Males with Duchenne, 8-16 years old & ambulatory VIEW DETAILS
Santhera Idebenone Phase 3 Males with Duchenne 10 years and older US, EU, IL 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

Chart updated on 3/4/20