A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease

Launched by ULTRAGENYX PHARMACEUTICAL INC · May 7, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called UX701 for Wilson Disease, a condition where the body has trouble managing copper, leading to serious health issues. The main goals of the study are to check how safe a single dose of UX701 is, figure out the best dose to use, and see how it helps control copper levels in the body.

To participate in this trial, individuals need to have a confirmed diagnosis of Wilson Disease and have been stable on their current medications for at least six months. They should also be following a diet that limits copper intake. Participants will need to commit to regular blood tests and other assessments throughout the study. It’s important to note that not everyone will qualify; for example, those with serious liver problems or certain mental health conditions may be excluded. Overall, this trial is an important step in finding new ways to manage Wilson Disease and improve patients' health.

Gender

ALL

Eligibility criteria

• 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