Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children

NCTID	NCT00927134 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Inactive
Indication	X-Linked Chronic Granulomatous Disease
Disease Ontology Term	DOID:0070195
Compound Name	Retroviral SF71-gp91phox transduced CD34+ cells
Sponsor	University of Zurich
Funder Type	Other
Recruitment Status	Completed
Enrollment Count	2 (ACTUAL)
Results Posted	Not Available

Target Gene/Variant	CYBB
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	SFFV
Dose 1	Transduced CD34+ cells (dose range: 3.6-5.1E6 cells/kg)

Eligible Age	1 Year - 18 Years
Standard Ages	Child, Adult
Sexes Eligible for Study	MALE
Eligibility Criteria	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) View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	1
Locations	Switzerland

Has US IND
FDA Designations
Recent Updates	Enrolled 2 patients, 2 patients enrolled under NCT00564759, same vector used in both