NCT06736080View on ClinicalTrials.gov

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

PHASE1/PHASE2Not yet recruitingINTERVENTIONAL
Enrollment

5

Participants

Timeline

Start Date

January 31, 2025

Primary Completion Date

January 31, 2029

Study Completion Date

January 31, 2029

Conditions
Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)
Interventions
DRUG

MUNC-CD34

"* Dosage: ≥ 2 x10e6 CD34/kg after thawing, dose limit: 20x10e6 CD34+ cells/kg~* Route of administration: intravenous, on D0"

DRUG

MUNC-T3

"* Dosage: \[1.10e4; 5.10e6\] T-CD3+/kg after thawing,~* Route of administration: intravenous, on D14 post-GT +/- D28 In case of persistent circulating T-cell after the HLH remission at inclusion, the MUNC-CD34 will be completed by MUNC-T3 infusion"

Trial Locations (1)

75015

Hôpital Necker Enfant Malades, Paris

All Listed Sponsors
lead

Assistance Publique - Hôpitaux de Paris

OTHER

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 | Biotech Hunter | Biotech Hunter