Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children (XCGDinChildren)

University of Zurich (UZH)

Status and phase: Completed; Phase 2; Phase 1

Conditions: Chronic Granulomatous Disease

Treatments: Genetic: retroviral SF71-gp91phox transduced CD34+ cells

Study type: Interventional

Funder types: Other

Identifiers

NCT00927134

PedsZürich_GT05

Details and patient eligibility

About

The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.

Enrollment: 2 patients

Sex: Male

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)