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Gene Therapy for Severe Crigler Najjar Syndrome

RecruitingNAAges 9 Years - anyAllGenethon
NCT03466463 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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.

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📋 Eligibility rules (as published)
Inclusion Criteria: * Patients with severe Crigler-Najjar syndrome resulting from a molecular confirmation of mutations in the UGT1A1 gene and requiring phototherapy * Male or female at least 9 years at the date of signature of informed consent * Patient able to give informed assent and/or consent in writing Exclusion Criteria: * Patients who underwent liver transplantation * Patients with chronic hepatitis B or C * Patients infected with Human immunodeficiency virus (HIV) * Patients with significant underlying liver disease * Patients with significant encephalopathy * Participation in any other investigational trial during this trial * Patients unable or unwilling to comply with the protocol requirements
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What it tests

  • GNT0003
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Where

4 sites
Hopital Antoine BECLERE
Clamart, France
ASST Papa Giovanni XXIII
Bergamo, Italy
Azienda Ospedaliera Universitaria Federico II
Naples, Italy
AMC
Amsterdam, Netherlands
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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