Chronic Granulomatous Disease
Conditions
Keywords
Chronic Granulomatous Disease in children (=or< 18 years)
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) children 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 transduced (SF71-gp91phox)CD34+ cells
Sponsors
Goethe University
University of Zurich
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
1 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* x-linked Chronic Granulomatous Disease * history of life-threatening severe infections * no HLA-matched related or unrelated donor * therapy resistent life threatening infections/organ dysfunction * no other treatment options e.g. HSCT
Exclusion criteria
* \> 18 years of age * HIV infection * life expectancy \> 2 years * infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| eradication of pre-existing therapy refractory bacterial and/or fungal infections | 6 months |
Secondary
| Measure | Time frame |
|---|---|
| Reconstitution of ROS production by peripheral blood cells | 1 month |
Countries
Switzerland
Outcome results
None listed