Condition: Severe Combined Immunodeficiency, X Linked · Gene Therapy · Sponsor: David Williams
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
This description comes directly from the study's public registry record.
Colleen Dansereau · 6179197008 · colleen.dansereau@childrens.harvard.edu
Always discuss trial participation with your own doctor first.
| Mattel Children's Hospital - UCLA | Los Angeles, California, United States | Recruiting |
| Emory University/Childrens Healthcare of Atlanta | Atlanta, Georgia, United States | Recruiting |
| Boston Childrens Hospital | Boston, Massachusetts, United States | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati, Ohio, United States | Recruiting |
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Source record: clinicaltrials.gov/study/NCT03311503