Status:
RECRUITING
Phase I/II Clinical Trial Stem Cell Gene Therapy in RAG1-Deficient SCID
Lead Sponsor:
Leiden University Medical Center
Collaborating Sponsors:
ZonMw: The Netherlands Organisation for Health Research and Development
Horizon 2020 - European Commission
Conditions:
Severe Combined Immunodeficiency Due to RAG1 Deficiency
Eligibility:
All Genders
8-24 years
Phase:
NA
Brief Summary
This study is a prospective, non-randomized, open-label, two-centre phase I/II intervention study designed to treat children up to 24 months of age with RAG1-deficient SCID with an indication for allo...
Detailed Description
Severe combined immunodeficiency (SCID) is a genetically heterogeneous life-threatening disease characterized by severely impaired T cell development with or without impaired natural killer (NK) and B...
Eligibility Criteria
Inclusion
- RAG1-deficient SCID as confirmed by genetic analysis
- Peripheral blood T cells \< 300/μL and/or naïve T cells \< 1/μL
- Age \< 2 years
- Age at least 8 weeks by the time of busulfan and fludarabine administration
- Lack of an available HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
- Signed informed consent (parental or guardian)
- 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
- Availability of an HLA-matched donor (HLA-identical sibling or 10/10 (A, B, C, DR, DQ) allele-matched (un)related donor)
- RAG1 deficiency with peripheral blood T cells \> 300/μL and/or naïve T cells \> 1/μL
- Omenn syndrome
- Previous allogeneic HSCT
- Significant organ dysfunction/co-morbidity (including but not limited to the ones listed below):
- Mechanical ventilation
- Shortening fraction on echocardiogram \<25%
- Renal failure defined as dialysis dependence
- Uncontrolled seizure disorder
- 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.
- Human immunodeficiency virus (HIV) infection or Human T-cell Leukemia Virus (HTLV) infection
Key Trial Info
Start Date :
July 23 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
December 31 2029
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04797260
Start Date
July 23 2021
End Date
December 31 2029
Last Update
April 18 2024
Active Locations (7)
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1
The Royal Childrens Hospital
Melbourne, Australia, 3052
2
Ospedale Pediatrico Bambino Gesù
Roma, Italy
3
Leiden University Medical Center
Leiden, Netherlands, 2300RC
4
Wroclaw Medical University
Wroclaw, Poland, 50-556