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Clinical Trial Report

Gene Therapy Trial Report

Summary

ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID


NCTID NCT00598481 (View at clinicaltrials.gov)
Description
Development Status Approved
Indication Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Disease Ontology Term DOID:5810
Compound Name STRIMVELIS
Compound Alias GSK2696273
Compound Description CD34+ cells transduced with MND-ADA retroviral vector
Sponsor Fondazione Telethon
Funder Type Other
Recruitment Status
Completed
Enrollment Count 12
Results Posted View Results

Therapy Information


Target Gene/Variant ADA
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
Delivery System Viral transduction
Vector Type MoMLV (RV)
Editor Type
Dose 1 Median dose: 11.6E6 CD34+ cells/kg (19 patients)
Dose 2 Median dose: 9.2E6 CD34+ cells/kg (22 patients)
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase2
Submit Date 2008-01-10
Completion Date 2019-06-19
Last Update 2024-01-29

Participation Criteria


Eligible Age <=17 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 2
Locations Italy,Israel

Regulatory Information


Has US IND
FDA Designations
Recent Updates Product was initially developed by SR-TIGET, in-licensed by GlaxoSmithKline in 2010. EMA approval in May 2016. GSK sold Strimvelis in March 2018 to Orchard Therapeutics. Orchard discontinued the program in March 2022. Product license was transferred to Fondazione Telethon in July 2023

Resources/Links