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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
NCTID
NCT06641154
(View at clinicaltrials.gov)
Description
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GT-UGT1A1-AAV8-02 in patients with Crigler-Najjar type 1 aged ≤10 years and requiring phototherapy. Patients will received a single administration of GT-UGT1A1-AAV8-02 and will be followed for safety and efficacy of approximately 60 months (5 years): * a follow-up of approximately 12 months (48 weeks) * a long term follow-up of approximately 48 months (4 years), in order to be in line with the latest EMEA Guideline on follow-up of patients administered with gene therapy medicinal products, released on 22 Oct.2009 by the Committee for medicinal products for human use.
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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.