Actively Recruiting
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
Led by Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare · Updated on 2025-01-13
5
Participants Needed
2
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients of any gender with severe Crigler-Najjar syndrome type I confirmed by UGT1A1 gene mutations
- Patients requiring phototherapy
- Patients aged at least 3 months and no older than 10 years at consent
- Patients able to provide written informed assent and/or consent
You will not qualify if you...
- Patients who have had a liver transplantation
- Patients with chronic hepatitis B or C
- Patients infected with HIV
- Patients with significant underlying liver disease
- Patients with significant encephalopathy
- Patients participating in another investigational trial during this study
- Patients unable or unwilling to comply with the study protocol requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Alphaviva LLC
Moscow, Russia
Actively Recruiting
2
Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Moscow, Russia
Actively Recruiting
Research Team
D
Denis V. Rebrikov, Dr., Professor,
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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