Gene Therapy for Chronic Granulomatous Disease
Phase 1
- Conditions
- Granulomatous Disease, Chronic
- Registration Number
- NCT00564759
- Lead Sponsor
- Johann Wolfgang Goethe University Hospital
- Brief Summary
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)patients 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- UNKNOWN
- Sex
- Male
- Target Recruitment
- 2
Inclusion Criteria
- x-linked Chronic Granulomatous Disease
- history of life-threatening severe infections
- no HLA-matched related or non-related donor
- therapy resistent life threatening infections/organ dysfunction
- no other treatment options e.g. BMT
Exclusion Criteria
- < 18 years of age
- HIV infection
- life expectancy > 2 years
- infections treatable by conventional therapy (antibiotics, allogeneic granulocytes)
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method safety, toxicity and feasibility 2 years
- Secondary Outcome Measures
Name Time Method Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit 2 years
Trial Locations
- Locations (1)
University Hospital, Hematology
🇩🇪Frankfurt, Germany
University Hospital, Hematology🇩🇪Frankfurt, Germany