Clinical trial
A Phase I/II Open Label Non Randomized Study, Monocentric, Single Arm, Evaluating Safety and Efficacy of Gene Therapy of FHL 3 Caused by Mutations in the Human UNC13D Gene by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced ex Vivo With the UNC13D LV Vector Expressing the UNC13D cDNA
NCT06736080 is registered in ClinicalTrials.gov with Assistance Publique - Hôpitaux de Paris as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Phase I/II Open Label Non Randomized Study, Monocentric, Single Arm, Evaluating Safety and Efficacy of Gene Therapy of FHL 3 Caused by Mutations in the Human UNC13D Gene by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced ex Vivo With the UNC13D LV Vector Expressing the UNC13D cDNA
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Phase
- Phase 1/2
- Overall status
- Not yet recruiting
- Start date
- May 31, 2026
- Completion date
- January 31, 2030
- Enrollment
- 5 (estimated)
- Sites
- 1
Conditions
As the registry lists them; a condition with its own page is linked.
familial hemophagocytic lymphohistiocytosis type 3 (fhl 3)