Clinical Trial

Gene Therapy for Severe Crigler Najjar Syndrome

Study acronym: CareCN
Recruiting
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Record status
This record was last updated March 28, 2023 (before its estimated March 30, 2026 completion). Its status may not reflect the trial's current state.
Summary
This is a Phase 1/2, multinational, open-label, study to evaluate the safety and efficacy of an intravenous infusion of GNT0003 in patients with Crigler-Najjar aged ≥10 years and requiring phototherapy. Patients will received a single administration of GNT0003 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.
Protocol Amendment History 6 amendments
This ClinicalTrials.gov record has been amended 6 times since 2018-03-08; most recent amendment 2023-03-23.
Status change: Not Yet Recruiting → Recruiting 2018-05-15
Trial Details
NCT Number NCT03466463
Lead Sponsor Genethon
Conditions Crigler-Najjar Syndrome
Enrollment 17 participants
Start Date 2018-03-19
Primary Completion 2026-03-30 (estimated)
Study Completion 2030-03-30 (estimated)
Updated on ClinicalTrials.gov 2023-03-28