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
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 with Low Dose Targeted Busulfan Conditioning
NCTID
NCT03311503
(View at clinicaltrials.gov)
Description
This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized
(Show More)
Development Status
Active
Indication
X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term
DOID:0060013
Compound Name
G2SCID lentiviral vector transduced CD34+ cells
Compound Description
rHIV_IL2RGcoG2SCID
Sponsor
David Williams
Funder Type
Other
Recruitment Status
Recruiting
Enrollment Count
12
Results Posted
Not Available
Therapy Information
Target Gene/Variant
IL2RG
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
HIV
Editor Type
none
Dose 1
Transduced CD34+ cells (minimum dose: 2.5E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2017-10-12
Completion Date
2028-10-01
Last Update
2025-01-06
Participation Criteria
Eligible Age
0 Years - 5 Years
Standard Ages
Child
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
4
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Recent Updates
Resources/Links
Clinical Publications
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency
Preclinical Publications
Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning
Correction of SCID-X1 using an enhancerless Vav promoter
Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression
Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells
Related NCTID
Phase 1: NCT03601286