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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
Development Status Active
Indication Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency
Disease Ontology Term DOID:0090012
Compound Name AProArt
Compound Description autologous hematopoietic cells transduced with a lentiviral vector, AProArt (hDCLRE1Cp.hDCLRE1C)
Sponsor University of California, San Francisco
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 25
Results Posted Not Available

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 none
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 2025-01-13

Participation Criteria


Eligible Age >=2 Months
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links