Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
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
The investigators propose to replace HLA- partially compatible allogeneic Hematopoietic Stem Cell Transplantation (HSCT) for FHL type 3 patients, with autologous transplantation of immunoselected gene-modified CD34+ cells, combined with transduced autologous T-cell each time this is possible and also to propose this alternative treatment as salvage in case of failure of a previous allogeneic HSCT. This approach should avoid the severe immunological complications (failure to engraft, acute or chronic graft versus host disease (GVHD)) and conditioning toxicities such as severe Veno-Occlusive Disease (VOD). As the clinical manifestations of FHL type 3 patients are triggered by opportunistic viral infections (often EBV) and can be poorly controlled or only transiently controlled by the available drugs , providing the patient after the conditioning with immediately functional autologous cytotoxic T-cells could be key to maintain the control of the viral infection and hopefully its eradication awaiting for the hematopoietic reconstitution . This procedure should avoid any reactivation of the viral infection and thus improving the patients' overall survival and event-free survival while clearing the ongoing triggering infections.
(Show More)
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.