Drugs / rAAV1.CMV.huFollistatin344
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT01519349 | Jan 2012 → Oct 2017 | Becker muscular dystrophy, inclusion body myositis | Nationwide Children's Hospital | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02354781 | Jan 2015 → Nov 2017 | Duchenne muscular dystrophy | Jerry R. Mendell | Completed | No outcome recorded |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rAAV1.CMV.huFollistatin344 | “Six DMD patients will receive rAAV1.CMV.huFollistatin344 to both limbs by multiple injections to gluteal muscles, quadriceps and tibialis anterior muscles.” NCT02354781 ↗ |
| Action | Inhibit | “a potent myostatin antagonist, follistatin (FS), was used to inhibit the myostatin pathway.” PMID 25322757 ↗ Oct 2014 |
| Modality | Gene therapy (AAV / viral vector) | “The gene will be carried into the muscle by a virus called adeno-associated virus (AAV).” NCT01519349 ↗ |
| Route | Intramuscular | “administered via intramuscular injection” NCT01519349 ↗ |
| Target | MSTN | “a potent myostatin antagonist, follistatin (FS), was used to inhibit the myostatin pathway.” PMID 25322757 ↗ Oct 2014 |