Safety and Efficacy of Gene Therapy of FHL Type 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
Safety and Efficacy of Gene Therapy of FHL Type 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
Not yet recruiting · NCT06736080 · Registry updated 2026-05-22Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Safety and Efficacy of Gene Therapy of FHL Type 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 | Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3) | Not yet recruiting | Phase 1 / Phase 2 | 2026-05-22 | NCT06736080 |
2 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Assistance Publique - Hôpitaux de Paris | 985 |
| URC-CIC Paris Descartes Necker Cochin | 154 |
1 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Department of Biotherapy, Hopital Necker Enfants Malades | Paris | France | 1 |