Drugs / rAAV2.5-CMV-minidystrophin
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1 | NCT00428935 | Mar 2006 → Mar 2009 | Duchenne muscular dystrophy | Nationwide Children's Hospital | Completed | No outcome recorded |
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 3 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | rAAV2.5-CMV-minidystrophin | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00428935 ↗ |
| Known as | AAV2.5 | “rAAV2.5-CMV-minidystrophin (d3990)” NCT00428935 ↗ |
| Known as | d3990 | “rAAV2.5-CMV-minidystrophin (d3990)” NCT00428935 ↗ |
| Action | Restore | “We report on delivery of a functional dystrophin transgene to skeletal muscle in six patients with Duchenne's muscular dystrophy.” PMID 20925545 ↗ Oct 2010 |
| Modality | Gene therapy (AAV / viral vector) | “using a recombinant adeno-associated virus” NCT00428935 ↗ |
| Route | Intramuscular | “AAV2.5 vector was injected into the bicep muscle in one arm, with saline control in the contralateral arm.” PMID 22068425 ↗ Nov 2011 |
| Target | DMD | “Recombinant adeno-associated virus (AAV) carrying a truncated human dystrophin gene (mini-dystrophin) expressed from a cytomegalovirus (CMV) promoter.” NCT00428935 ↗ |