This study examines a gene therapy in which the patient's own stem cells are genetically modified with a lentiviral approach and returned, in patients with chronic granulomatous disease. Eligible participants have a diagnosis confirmed by DNA sequencing and at least one severe infection or inflammatory complication requiring hospitalization. It is being conducted as a study without a formal phase and is currently recruiting participants.
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: 1. CGD patients \>= 0 years of age 2. Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or significantly reduced biochemical activities of the NADPH-oxidase 3. Karnofsky-Index \> =70% 4. At least one prior, ongoing or refractory severe infection and/or inflammatory complications requiring hospitalization despite drug intervention 5. Written informed consent for adult patient, and assent for pediatric subjects seven years or older Exclusion Criteria: 1. Contraindication for leukapheresis (anaemia Hb \<8g/dl, cardiovascular instability, severe coagulopathy) or for administration of conditioning medication 2. Female patients who are pregnant or lactating as determined by history and/or positive pregnancy test