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Clinical Trial Report

Gene Therapy Trial Report

Summary

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


NCTID NCT06736080 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Familial Hemophagocytic Lymphohistiocytosis Type 3 (FHL 3)
Disease Ontology Term DOID:0110923
Compound Name Autologous Cells Transduced Ex Vivo with LV-UNC13D
Sponsor Assistance Publique - Hôpitaux de Paris
Funder Type Other
Recruitment Status
Not yet recruiting
Enrollment Count 5
Results Posted Not Available

Therapy Information


Target Gene/Variant UNC13D
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells/T-CD3+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 2-20E6 CD34+ cells/kg
Dose 2 1.1E4 - 5.1E6 T-CD3+ cells/kg (as needed)
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-12-04
Completion Date 2029-01
Last Update 2024-12-16

Participation Criteria


Eligible Age 3 Months - 17 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.