pCCLCHIM-p47 (Lentiviral Vector Transduced CD34 Plus Cells) in Patients With p47 Autosomal Recessive Chronic Granulomatous Disease (AR-CGD)

Inclusion Criteria

• •In order to be eligible to participate in this study, an individual must meet all of the following criteria:
• •Must have confirmed genetic diagnosis of p47 AR-CGD by identification of a mutation in the responsible genes or protein analysis demonstrating lack of P47 expression and supported by laboratory evidence for absent or reduction \> 95% of the biochemical
• •activity of the NADPH-oxidase.
• •Must weigh at least 15 kg.
• •Adult Patient must be willing to sign and date informed consent form.
• •Parent/guardian must be willing to sign and date informed consent form for child and where appropriate, child may sign assent.
• •State willingness to comply with all study procedures and availability for the duration of the study.
• •Male or female must be at least 3 years of age.
• •Do not have an appropriately HLA matched donor (related or unrelated)
• •Must have had at least a history of a prior CGD related infection and/or an ongoing/refractory severe infection requiring hospitalization and/or inflammatory complications requiring hospitalization despite conventional therapy.
• •Patients will be collected as per standard of care mobilization and apheresis procedures as performed at the Clinical Center. Patients will have consented onto the gene therapy study prior to collection; however products collected prior to the study for other protocols may be used as part of the back up.
• •* Patient must weigh at least 15 kilograms body weight.
• •* Normal female donors of childbearing potential may be entered if using effective contraception and having a negative serum pregnancy test within one week of beginning G-CSF administration.
• •Ability to take oral medication and be willing to adhere to the prophylactic regimen.
• •For females of reproductive potential, must agree to use 2 forms of highly effective contraception throughout study participation (for a minimum of 3 months after having received the genetically modified cells but preferably for the first 2 years.)
• •* For females (will be counseled on appropriate combinations for best efficacy):
• •* Condoms, male or female, with or without a spermicide.
• •* Diaphragm or cervical cap with spermicide.
• •* Intrauterine device.
• •* Contraceptive pills or patch, Norplant, Depo-Provera, or other FDAapproved contraceptive method.
• •* Having a male partner who has previously undergone a vasectomy.
• •* For males of reproductive potential: use of condoms or other methods to ensure effective contraception prevention with partner.
• •Agreement to adhere to Lifestyle Considerations throughout study duration.
• •Must provide a durable power of attorney (DPA) for health care decisions to an appropriate adult relative or guardian in accordance to NIH-200 "NIH Advance Directive for Health Care and Medical Research Participation".
• •All patients must be willing to allow storage of blood samples for gene therapy studies.
• •* Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful study completion.