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Clinical Trial Report

Gene Therapy Trial Report

Summary

Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy


NCTID NCT06851767 (View at clinicaltrials.gov)
Description
Development Status Active
Indication X-linked Severe Combined Immunodeficiency (XSCID)
Disease Ontology Term DOID:0060013
Compound Name BE-HSPC-IL2RG
Sponsor National Institute of Allergy and Infectious Diseases (NIAID)
Funder Type NIH
Recruitment Status
Not yet recruiting
Enrollment Count 18
Results Posted Not Available

Therapy Information


Target Gene/Variant IL2RG (p.Q144X)
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 Undisclosed
Vector Type
Editor Type base editor
Dose 1 >5E6 cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2025-02-26
Completion Date 2034-12-31
Last Update 2025-04-23

Participation Criteria


Eligible Age 3 Years - 99 Years
Standard Ages Child, Adult, Older 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

Resources/Links


Resources/Links

No External Links Available.