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This study examines UX701, a gene therapy, compared with standard of care in adults with Wilson disease. It enrolls adults with a diagnosis confirmed by ATP7B gene mutation who have been stable on copper chelator and/or zinc therapy for at least two months. The study is being conducted in Phase 1/Phase 2 and is active but not currently recruiting.
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.
Key Inclusion Criteria: * Confirmed diagnosis of Wilson disease based on genetic confirmation of heterozygous or homozygous biallelic ATP7B mutation. * Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 2 months at screening, with no medication or dose changes for at least 2 months at screening. * Ongoing restriction of high copper containing foods for at least 2 months at Screening and continued through study participation. * Willing and able to comply with all study procedures and requirements, including frequent blood collection, total urine collection over a 24-hour period, patient-reported outcome assessments, and long-term follow-up Key Exclusion Criteria: * Detectable pre-existing antibodies to the AAV9 capsid. * Stage 1 only: History of copper chelator or zinc therapy noncompliance, in the Investigator's judgment, within 6 months prior to Screening. * History of liver transplant. * Active decompensated hepatic cirrhosis or history of hepatic encephalopathy. * Significant hepatic inflammation as evidenced by laboratory abnormalities. * Model for End-Stage Liver Disease (MELD) score \> 13. * Hemoglobin \< 9 g/dL * Presence of Stage 3 or higher chronic kidney disease based on estimated glomerular filtration rate \< 60 mL/min/1.73 m2. * Marked neurological deficit or compromise that, in the Investigator's opinion, would interfere with the subject's safety or ability to participate in the study. * Moderate to severe depression, recent or active suicidal ideation with intent or suicidal behavior, psychosis, or unstable psychiatric illness. * Known hypersensitivity to UX701 or its excipients, copper chelators, zinc, rituximab, tacrolimus, corticosteroids, or eculizumab that, in the Investigator's judgement, places the participant at increased risk for adverse events. * Participation in another gene transfer study or use of another gene transfer product before or during study participation. * Subjects with known hypersensitivity to amide-containing local anesthetics are excluded from participating in the optional liver biopsy substudy. Note: Other protocol defined Inclusion/ Exclusion criteria may apply