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Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy for X-linked Severe Combined Immunodeficiency
NCTID
NCT01410019
(View at clinicaltrials.gov)
Description
X-linked severe combined immunodeficiency (SCID-X1) is an inherited disorder that results in failure of development of the immune system in boys. This trial aims to treat SCID-X1 patients using gene therapy to replace the defective gene.
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Development Status
Inactive
Indication
X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term
DOID:0060013
Compound Name
Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre
Sponsor
Assistance Publique - Hôpitaux de Paris
Funder Type
Other
Recruitment Status
Completed
Enrollment Count
5
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
RV
Editor Type
Dose 1
Median dose: 7.58E6 transduced CD34+ cells/kg (n=11)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2011-06-21
Completion Date
2015-06-16
Last Update
2021-09-28
Participation Criteria
Eligible Age
<=12 Months
Standard Ages
Child
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
France
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Resources/Links
Clinical Publications
Efficacy of gene therapy for X-linked severe combined immunodeficiency
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency
(Abstract) Long-Term Outcome of Gene Therapy for X-Linked Severe Combined Immunodeficiency (SCID-X1) Using an Enhancer-Deleted Self-Inactivating Gammaretroviral Vector - ASH 2022
Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector
Treatment of an infant with X-linked severe combined immunodeficiency (SCID-X1) by gene therapy in Australia
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency
Preclinical Publications
Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency
Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors
(Abstract) Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient - ASH 2011
Related NCTID
Phase Not Applicable: NCT01175239
Phase 1/2: NCT01129544