Drugs / DTX101
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02618915 | Dec 2015 → Oct 2017 | hemophilia B | Ultragenyx Pharmaceutical Inc | Terminated | Missed primary Stop: Business |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | DTX101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02618915 ↗ |
| Action | Restore | “This study will evaluate the safety and efficacy of the adeno-associated virus (AAV) to deliver human factor IX (hFIX) gene, the healthy gene necessary to make FIX, to the...” NCT02618915 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “DTX101 is a nonreplicating adeno-associated viral serotype rh10 gene transfer vector containing a codon-optimized wild-type human FIX coding sequence.” PMID 40197980 ↗ Jun 2025 |
| Route | Intravenous | “a single peripheral intravenous (IV) infusion of 1.6 x 10\^12 genome copies (GC)/kg DTX101” NCT02618915 ↗ |