Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

This is a phase I/II open label multi-center study in which patients will receive low dose targeted busulfan followed by infusion of autologous CD34+ selected bone marrow or mobilized peripheral blood cells transduced with the G2SCID vector. Subjects will be enrolled over 3 years and be followed for 2 years post-infusion on this protocol, then followed long-term on a separate long-term follow-up protocol. Enrollment of subjects will be agreed upon by representatives of both sites. Data will be collected uniformly from both sites through an electronic capture system and key laboratory studies will be centralized. Harvest, cellular manufacturing and infusion will occur at each site using the same SOPs. Key aspects of cellular product characterization will be centralized

NCT Number	NCT03311503
Lead Sponsor	David Williams
Conditions	Severe Combined Immunodeficiency, X Linked, Gene Therapy
Enrollment	12 participants
Start Date	2018-02-26
Primary Completion	2028-01-01 (estimated)
Study Completion	2028-01-01 (estimated)
Updated on ClinicalTrials.gov	2025-12-11