Overview

Gene Therapy for Chronic Granulomatous Disease

Status:
Unknown status
Trial end date:
2008-12-01
Target enrollment:
0
Participant gender:
Male
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.
Phase:
Phase 1/Phase 2
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Johann Wolfgang Goethe University Hospital
Collaborator:
German Federal Ministry of Education and Research
Criteria
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)