Status:
COMPLETED
Gene Therapy for X-linked Severe Combined Immunodeficiency
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborating Sponsors:
URC-CIC Paris Descartes Necker Cochin
Conditions:
X-linked Severe Combined Immunodeficiency
Eligibility:
MALE
Up to 12 years
Phase:
PHASE1
PHASE2
Brief Summary
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 t...
Detailed Description
The objective of this protocol is to reinitiate an ex vivo gene therapy clinical protocol to treat patients with SCID-X1 without HLA identical family donor nor HLA identical unrelated donor (bone marr...
Eligibility Criteria
Inclusion
- 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)
Exclusion
Key Trial Info
Start Date :
December 1 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 16 2015
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT01410019
Start Date
December 1 2010
End Date
June 16 2015
Last Update
September 12 2025
Active Locations (1)
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1
Hopital Necker
Paris, France, 75015