This study investigates GT-UGT1A1-AAV8-02, a gene therapy, in patients with severe Crigler-Najjar syndrome type I (an inherited disorder in which the liver cannot process bilirubin). Patients aged 3 months to 10 years with a confirmed mutation in the UGT1A1 gene who require phototherapy can take part. It is being conducted in Phase 1/Phase 2 and is still enrolling participants.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
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 3 months (no more 10 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