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Clinical Trial Report
Gene Therapy Trial Report
Summary
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.
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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
Results Posted
Not Available
Therapy Information
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
Editor Type
Dose 1
Transduced CD34+ cells (dose range: 3.6-5.1E6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2009-06-22
Completion Date
2011-09
Last Update
2011-09-27
Participation Criteria
Eligible Age
1 Year - 18 Years
Standard Ages
Child, Adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
1
Locations
Switzerland
Regulatory Information
Has US IND
FDA Designations
Recent Updates
Enrolled 2 patients, 2 patients enrolled under NCT00564759, same vector used in both
Resources/Links
Clinical Publications
Gene Therapy of Chronic Granulomatous Disease: The Engraftment Dilemma
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
Restoration of NET formation by gene therapy in CGD controls aspergillosis
Preclinical Publications
Use of serum-free medium with fibronectin fragment enhanced transduction in a system of gas permeable plastic containers to achieve high levels of retrovirus transduction at clinical scale
Retroviral vector integration in post-transplant hematopoiesis in mice conditioned with either submyeloablative or ablative irradiation
Related NCTID
Phase 1/2: NCT00564759