Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
David Williams
Summary
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
Description
This is an open labeled, multi-center, phase I/II, cohort study involving a single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID in up to 10 patients with X-linked SCID (SCID-X1) at Boston Children's Hospital, UCLA Mattel Children's Hospital, Cincinnatti Childrens Hospital Medical Center, Children's Healthcare of Atlanta. Patients will receive transduced cells after low dose targeted busulfan pre-conditioning (n=12). Enrolled subjects will be followed for 2 years after infusion on this protocol. Required long-term monitoring for a tota…
Eligibility
- Age range
- 0–5 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: \- 1. Diagnosis of SCID-X1 based on immunophenotype and lack of T cell function (proliferation to PHA \<10% of the lower limit of normal for the laboratory) AND confirmed by a mutation in IL2RG 2. Lack of an HLA identical (A, B, C, DR, DQ) related donor 3. Age 5 years old or younger 4. Signed informed consent 5. Documentation of willingness to follow up for 15 years post-infusion as currently required by the FDA 6. If the patient has previously undergone allogeneic transplant, lack of donor T cell engraftment must be documented. 7\. Age at least 8 weeks by the time of bus…
Interventions
- Biologicalautologous CD34+ cell transduced with G2SCID vector
single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) lentiviral vector G2SCID
Locations (4)
- Mattel Children's Hospital - UCLALos Angeles, California
- Emory University/Childrens Healthcare of AtlantaAtlanta, Georgia
- Boston Childrens HospitalBoston, Massachusetts
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio