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Clinical Trial Report

Gene Therapy Trial Report

Summary

Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease


NCTID NCT02234934 (View at clinicaltrials.gov)
Description
Indication Chronic Granulomatous Disease, X-linked
Compound Name PCCLChimGp91/VSVg lentiviral vector (formerly OTL-102)
Sponsor University of California, Los Angeles
Funder Type Other
Status
Recruiting
Enrollment Count 16

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 VSV-G
Editor Type
Dose 1 Transduced CD34+ cells (6.5-32.6 x 10^6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2014-09-04
Completion Date 2028-12
Last Update 2023-11-18

Participation Criteria


Eligible Age >=23 Months
Standard Ages Child, Adult, Older adult
Eligible Sex MALE

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
Recent Updates Commercial rights are owned by Orchard, which has deprioritized the program

Resources/Links