This study examines a gene therapy (retroviral SF71-gp91phox transduced CD34+ cells) for X-linked chronic granulomatous disease in children. It enrolled children with CGD who had a history of life-threatening infections and no HLA-matched donor. The study was conducted in Phase 1/Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * x-linked Chronic Granulomatous Disease * history of life-threatening severe infections * no HLA-matched related or unrelated donor * therapy resistent life threatening infections/organ dysfunction * no other treatment options e.g. HSCT Exclusion Criteria: * \> 18 years of age * HIV infection * life expectancy \> 2 years * infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)