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Clinical Trial Report

Gene Therapy Trial Report

Summary

Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease


NCTID NCT06325709 (View at clinicaltrials.gov)
Description
Indication Chronic Granulomatous Disease, X-linked
Compound Name Base-edited autologous CD34+ cells
Sponsor National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type NIH
Status
Recruiting
Enrollment Count 10

Therapy Information


Target Gene/Variant CYBB c.676 C>T
Therapy Type Gene editing
Therapy Route Ex-vivo
Mechanism of Action Mutation correction
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Electroporation
Vector Type none
Editor Type ABE8e-SpRY
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-03-21
Completion Date 2032-12-31
Last Update 2024-11-04

Participation Criteria


Eligible Age 18 Years - 75 Years
Standard Ages Adult, Older adult
Eligible Sex MALE

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
Recent Updates First clinical data expected 2025

Resources/Links