This trial is active, not recruiting.

Condition granulomatous disease, chronic
Treatment retroviral sf71-gp91phox transduced cd34+ cells
Phase phase 1/phase 2
Sponsor Johann Wolfgang Goethe University Hospitals
Collaborator German Federal Ministry of Education and Research
Start date January 2004
End date December 2008
Trial size 2 participants
Trial identifier NCT00564759, 58/59, DeReG 31, KSG 31


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation non-randomized
Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label

Primary Outcomes

safety, toxicity and feasibility
time frame: 2 years

Secondary Outcomes

Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
time frame: 2 years

Eligibility Criteria

Male participants at least 18 years old.

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)

Additional Information

Official title Phase I/II Gene Therapy Study for X-Linked Chronic Granulomatous Disease
Principal investigator Dieter Hoelzer, MD, PhD
Trial information was received from ClinicalTrials.gov and was last updated in November 2007.
Information provided to ClinicalTrials.gov by Johann Wolfgang Goethe University Hospitals.