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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)


NCTID NCT06641154 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Crigler-Najjar Syndrome Type I
Disease Ontology Term DOID:3803
Compound Name GT-UGT1A1-AAV8-02
Compound Description rAAV2/8.TBG.UGT1A1
Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Funder Type Other gov
Recruitment Status
Recruiting
Enrollment Count 5
Results Posted Not Available

Therapy Information


Target Gene/Variant UGT1A1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Target Tissue/Cell Liver
Delivery System Viral transduction
Vector Type rAAV2/8
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2024-10-11
Completion Date 2029-11-01
Last Update 2025-01-13

Participation Criteria


Eligible Age 3 Months - 10 Years
Standard Ages Child
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 2
Locations Russian Federation

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links


Resources/Links

No External Links Available.