Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 2:16 AM
Ignite Modification Date:
2025-12-25 @ 2:16 AM
NCT ID:
NCT04797260
Eligibility Criteria:
Inclusion Criteria:
1. RAG1-deficient SCID as confirmed by genetic analysis
2. Peripheral blood T cells \< 300/μL and/or naïve T cells \< 1/μL
3. Age \< 2 years
4. Age at least 8 weeks by the time of busulfan and fludarabine administration
5. Lack of an available HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
6. Signed informed consent (parental or guardian)
7. Able to return to the study centre for follow-up (per protocol) during the 2-year study and the 15-year long-term off study review
Exclusion Criteria:
1. Availability of an HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
2. RAG1 deficiency with peripheral blood T cells \> 300/μL and/or naïve T cells \> 1/μL
3. Omenn syndrome
4. Previous allogeneic HSCT
5. Significant organ dysfunction/co-morbidity (including but not limited to the ones listed below):
1. Mechanical ventilation
2. Shortening fraction on echocardiogram \<25%
3. Renal failure defined as dialysis dependence
4. Uncontrolled seizure disorder
6. Any other condition that the investigator considers is a contraindication to collection and/or infusion of trans-duced cells for that individual or indicate patient's inability to follow the protocol, for example contraindication f to busulfan, major congenital abnormalities, ineligible to receive anaesthesia, or documented refusal or inability of the family to return for scheduled visits.
7. Human immunodeficiency virus (HIV) infection or Human T-cell Leukemia Virus (HTLV) infection
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
8 Weeks
Maximum Age:
24 Months
Study:
NCT04797260
Study Brief:
Protocol Section:
NCT04797260