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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 | FibroGen | Pamrevlumab | Phase 3 | Males with Duchenne, ages 12 years and older, and non-ambulatory | US, EU | VIEW DETAILS |
| FibroGen | Pamrevlumab | Phase 3 | Males with Duchenne 6-11 years old & ambulatory | US | VIEW DETAILS | |
| Anti-inflammatory | Children’s Research Institute | Ilaris | Phase 1/2 | Males with Duchenne 2 years and older & ambulatory | Washington DC | VIEW DETAILS |
| Nationwide Children’s Hospital | Spironolactone | Phase 1 | Males with Duchenne 4-7 years old | Ohio | VIEW DETAILS | |
| Cardiac Function | Cumberland Pharmaceuticals | Ifetroban | Phase 2 | Males with Duchenne, ages 7 years and older | US | VIEW DETAILS |
| Peking Union Medical College Hospital | Bisoprolol Fumarate | Phase 2/3 | Males with Duchenne, ages 7 years and older | CN | VIEW DETAILS | |
| Cell Therapy | CHU de Quebec-Universite Laval | Myoblast transplantation | Phase 1/2 | Males with Duchenne, ages 16 years and older | CA | VIEW DETAILS |
| Exon Skipping | Audentes Therapeutics | AAV9 U7snRNA | Phase 1/2 | Males with Duchenne 6 months – 13 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, CA, JP, EU, CL, KR, MX, NZ, RU, TW, TR | VIEW DETAILS | |
| NS Pharma | NS-089/NCNP-02 | Phase 1/2 | Males with Duchenne 4-17 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 an out of frame duplication of either exon 45, 51, or 53. | Ohio | VIEW DETAILS | |
| Gene Therapy | Pfizer | PF-06939926 | Phase 3 | Males with Duchenne 4-7 years old | US, UK, CA, EU, IL, JP, KR, RU, TW | VIEW DETAILS |
| Sarepta Therapeutics | SRP-9001 | Phase 1 | Males with Duchenne 4-7 years old, & ambulatory | US | VIEW DETAILS | |
| Solid Biosciences | SGT-001 | Phase 1/2 | Males with Duchenne 4-17 years old | US | VIEW DETAILS | |
| Mitochondrial Function | Astellas | ASP0367 | Phase 1b | Males with Duchenne, 8-16 years old | US | VIEW DETAILS |
| Muscle Stabilization | Edgewise Therapeutics | EDG-5506 | Phase 1 | Healthy Volunteers or Males with Becker, 18-55 years old | Texas | VIEW DETAILS |
| Stop Codon Readthrough | PTC Therapeutics | Ataluren | Phase 2 | Males with Duchenne, ages 6 months to 2 years with a nonsense mutation | VIEW DETAILS |
Last updated: 4/15/2021