# 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

> **NCT06736080** · PHASE1,PHASE2 · NOT_YET_RECRUITING · sponsor: **Assistance Publique - Hôpitaux de Paris** · enrollment: 5 (estimated)

## Conditions studied

- Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)

## Interventions

- **DRUG:** MUNC-CD34
- **DRUG:** MUNC-T3

## Key facts

- **NCT ID:** NCT06736080
- **Lead sponsor:** Assistance Publique - Hôpitaux de Paris
- **Sponsor class:** OTHER
- **Phase:** PHASE1,PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** NOT_YET_RECRUITING
- **Start date:** 2025-01
- **Primary completion:** 2029-01
- **Final completion:** 2029-01
- **Target enrollment:** 5 (ESTIMATED)
- **Last updated:** 2024-12-16


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06736080

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06736080, "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". Retrieved via AI Analytics 2026-06-02 from https://api.ai-analytics.org/clinical/NCT06736080. Licensed CC0.

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