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Clinical Trial Report

Gene Therapy Trial Report

Summary

Autologous Gene Therapy for Artemis-Deficient SCID


NCTID NCT03538899 (View at clinicaltrials.gov)
Description
Indication Severe Combined Immunodeficiency, DCLRE1C Deficient
Compound Name AProArt (endogenous Artemis promotor)
Sponsor University of California, San Francisco
Funder Type Other
Status
Recruiting
Enrollment Count 25

Therapy Information


Target Gene/Variant DCLRE1C
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 LV
Editor Type
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2018-05-03
Completion Date 2038-06
Last Update 2024-02-12

Participation Criteria


Eligible Age >=2 Months
Standard Ages Child, Adult, Older adult
Eligible Sex ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
Recent Updates

Resources/Links