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Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)

NCT06641154 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2025-01-13

No results posted yet for this study

Summary

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

  • Crigler-Najjar Syndrome Type I

Interventions

DRUG

GT-UGT1A1-AAV8-02

Intravenous infusion, single dose

Sponsors & Collaborators

  • Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare

    lead OTHER_GOV

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Months
Max Age
10 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-06
Primary Completion
2027-11-01
Completion
2029-11-01

Countries

  • Russia

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06641154 on ClinicalTrials.gov