Granulomatous Disease, Chronic
Conditions
Keywords
CGD
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.
Interventions
autologous ex-vivo retroviral transduced (SF71-gp91phox) CD34+ cells
Sponsors
German Federal Ministry of Education and Research
Johann Wolfgang Goethe University Hospital
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| safety, toxicity and feasibility | 2 years |
Secondary
| Measure | Time frame |
|---|---|
| Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit | 2 years |
Countries
Germany
Outcome results
None listed