Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
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...
Brief Summary
Official Title: “Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children”
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.
- Study Type: Interventional
- Study Design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
- Study Primary Completion Date: December 2010
Intervention(s) in this Clinical Trial
- Genetic: retroviral SF71-gp91phox transduced CD34+ cells
- autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells
Outcome Measures for this Clinical Trial
Primary Measures
- eradication of pre-existing therapy refractory bacterial and/or fungal infections
- Time Frame: 6 months
Safety Issue?: No
- Time Frame: 6 months
Secondary Measures
- Reconstitution of ROS production by peripheral blood cells
- Time Frame: 1 month
Safety Issue?: No
- Time Frame: 1 month
Criteria for Participation in this Clinical Trial
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)
Gender Eligibility for this Clinical Trial: Male
Minimum Age for this Clinical Trial: 1 Year
Maximum Age for this Clinical Trial: 18 Years
Are Healthy Volunteers Accepted for this Clinical Trial?: No
Clinical Trial Investigator Information
Lead Investigator: University of Zurich Other
Overall Clinical Trial Officials and Contacts
Reinhard Seger, Prof Dr med Principal Investigator University Children's Hospital Zürich
Related Publications
References
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kühlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Lüthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006 Apr;12(4):401-9. Epub 2006 Apr 2.
Additional Information
Information obtained from ClinicalTrials.gov on February 02, 2012
Link to the current ClinicalTrials.gov record. http://clinicaltrials.gov/show/NCT00927134
Study ID Number: PedsZürich_GT05
ClinicalTrials.gov Identifier: NCT00927134
Health Authority: Switzerland: Federal Office of Public Health
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The URL of this page is:
http://clinicaltrialsfeeds.org/clinical-trials/show/NCT00927134
