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)