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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
This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene. This method involves transferring a normal copy of the DCLRE1C gene into stem cells of an affected patient. Participants will receive an infusion of stem cells transduced with a self-inactivating lentiviral vector that contains a normal copy of the DCLRE1C gene. Prior to the infusion they will receive sub-ablative, dose-targeted busulfan conditioning. The study will investigate if the procedure is safe, whether it can be done according to the methods described in the protocol, and whether the procedure will provide a normal immune system for the patient. A total of 24 newly diagnosed patients will be enrolled at the University of California San Francisco in this single-site trial and will be followed for 15 years post-infusion. It is hoped that this type of gene transfer may offer improved outcomes for ART-SCID patients who lack a brother or sister who can be used as a donor for stem cell transplantation or who have failed to develop a functioning immune system after a previous stem cell transplant.
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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
Clinical Publications
Lentiviral Gene Therapy for Artemis-Deficient SCID
Preclinical Publications
Characterization of the human artemis promoter by heterologous gene expression in vitro and in vivo
Cytotoxicity associated with artemis overexpression after lentiviral vector-mediated gene transfer
Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency
Role of transgene regulation in ex vivo lentiviral correction of artemis deficiency
Protocol
Clinical Trial Protocol