This trial is testing a new way to treat SCID-X1, a genetic disorder that prevents the development of a functioning immune system. The new method, called lentiviral gene transfer, involves transferring a normal copy of the common gamma chain gene into the patient's bone marrow stem cells. The investigators want to determine if the procedure is safe and effective.
5 Primary · 0 Secondary · Reporting Duration: up to 10 years post gene transfer
28 Total Participants · 1 Treatment Group
Primary Treatment: CL20-i4-EF1α-hγc-OPT · No Placebo Group · Phase 1 & 2
Age < 18 · Male Participants · 8 Total Inclusion CriteriaMark “Yes” if the following statements are true for you: