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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
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