Contact SCGE




Gene Therapy Trial Report

Summary

Gene Therapy for X-linked Severe Combined Immunodeficiency


NCTID NCT01410019 (View at clinicaltrials.gov)
Description
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 (ACTUAL)
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
Dose 1 Median dose: 7.58E6 transduced CD34+ cells/kg (n=11)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2011-06-21
Completion Date 2015-06-16
Last Update 🔄 2026-03-11

Participation Criteria


Eligible Age <=12 Months
Standard Ages Child
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion criteria : * Boys diagnosed during the first year of life * Diagnosis of classical SCID-X1 based on immunophenotype (absent, or reduced numbers of non-functional T lymphocytes) and confirmed by DNA sequencing * No HLA identical family donor and no HLA identical unrelated donor (10/10 antigens) found in the 6 weeks following the beginning of the search. This period could be shortened if the probability to find a donor is low or if the clinical situation (gravity) required * Presence of a severe infection: pneumonitis and / or chronic diarrhea, or infection with herpes viruses or parainfluenza type 3 or adenovirus, or disseminated BCG infection, or presence of severe diarrhea and a severe compromise of the general state with denutrition * Or failure of a HLA HAPLO-identical bone marrow transplant within 10 years after transplantation * In all cases: * No family background of cancer in childhood. * No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia of children * Parental/guardian voluntary consent Exclusion criteria : * Atypical health with autologous T\> 500/ml3 * Infection by HIV 1 or 2 * Allogeneic HSC completed (excluding situations of failure) * Existence of an HLA identical family donor or HLA identical unrelated donor * No severe infections in a child with a preserved general state * Family background of cancer in childhood * Detection of cytogenetic abnormality and / or rearrangement associated with acute leukemia of children * No affiliation to a social security scheme (beneficiary or assignee)
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations France

Regulatory Information


Has US IND
FDA Designations
Recent Updates

Resources/Links