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Gene Therapy for Crigler Najjar Syndrome Type I (AlphaCN)

StatusRecruiting
PhasePhase 1/2
Started
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Record History

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  1. Secondary endpoints: details revised at 1 of 1 entry
    • Study title expanded, about 1 word
    v4
  2. Trial status: Not Yet Recruiting to Recruiting
    • Start date changed from estimate of Nov 1, 2024 to Nov 6, 2024
    • Site details revised at 2 of 2 sites
    v3
  3. Study sites: 1 to 2v2
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT06641154
Lead Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare
Conditions
(from sponsor)		 Crigler-Najjar Syndrome Type I
Related terms Crigler-Najjar Syndrome
Enrollment 5 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov