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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy for Chronic Granulomatous Disease


NCTID NCT00394316 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication X-Linked Chronic Granulomatous Disease
Disease Ontology Term DOID:0070195
Compound Name MFGS-gp91phox
Sponsor National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type NIH
Recruitment Status
Terminated
Enrollment Count 3
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 MFGS RV
Editor Type
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Early phase1
Submit Date 2006-10-31
Completion Date 2014-04-08
Last Update 2018-07-05

Participation Criteria


Eligible Age 3 Years - 55 Years
Standard Ages Child, Adult
Sexes Eligible for Study MALE

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Enrolled 3/6 planned patients, study was terminated in April 2014

Resources/Links