Gene Therapy for X-linked Severe Combined Immunodeficiency
Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Aug 3, 2011
Trial Information
Current as of May 01, 2025
Completed
Keywords
ClinConnect Summary
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 marrow and cord blood) available in an adequate time with the clinical conditions of the patient at diagnosis (approximately 6 weeks). This clinical protocol No. 2 of SCID-X1 must be as efficient than the previous one but must involve a risk of insertional mutagenesis significantly reduced as compared to the first protocol.
The main purpose of the study is the study of toxicity: tolerance and inci...
Gender
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)
About Assistance Publique Hôpitaux De Paris
Assistance Publique - Hôpitaux de Paris (AP-HP) is a leading public hospital system in France, renowned for its commitment to healthcare excellence and innovative medical research. As a prominent clinical trial sponsor, AP-HP plays a pivotal role in advancing medical knowledge and improving patient care through rigorous scientific investigations across a wide range of therapeutic areas. With a focus on collaboration and interdisciplinary approaches, AP-HP leverages its extensive network of hospitals and expert clinicians to facilitate high-quality clinical trials that adhere to the highest ethical and regulatory standards, ultimately aiming to translate research findings into tangible health benefits for diverse patient populations.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Paris, , France
Patients applied
Trial Officials
Alain Fischer, MD, PhD
Study Director
Assistance Publique - Hôpitaux de Paris
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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