Study of UX701 Gene Transfer for the Treatment of Wilson Disease
Wilson Disease
About this trial
This is an interventional treatment trial for Wilson Disease
Eligibility Criteria
Key Inclusion Criteria:
- Confirmed diagnosis of Wilson disease
- Ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 12 months at screening, with no medication or dose changes for at least 6 months at screening.
- Stable Wilson disease as evidenced by stable 24-hour urinary copper concentration during the Screening Period
- Ongoing restriction of high copper containing foods for at least 12 months at Screening, 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 12 months prior to Screening.
- History of liver transplant.
- Decompensated hepatic cirrhosis or presence of advanced liver disease as evidenced by portal hypertension, ascites, splenomegaly, esophageal varices, 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.
- 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
Sites / Locations
- University of California Los AngelesRecruiting
- Stanford UniversityRecruiting
- University of California DavisRecruiting
- Northwestern UniversityRecruiting
- Indiana UniversityRecruiting
- Massachusetts General HospitalRecruiting
- University of MichiganRecruiting
- Duke University Medical CenterRecruiting
- Vanderbilt University Medical CenterRecruiting
- University of UtahRecruiting
- University of VirginiaRecruiting
- Seattle Children's HospitalRecruiting
- Gordon and Leslie Diamond Health Care CentreRecruiting
- Centro Hospitalar Universitário Lisboa NorteRecruiting
- Centro Hospitalar Universitário de São JoãoRecruiting
- Hospital Universitario Vall d'Hebron - PPDSRecruiting
- Kings College NHS FoundationRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Experimental
Experimental
Experimental
Experimental
Experimental
Stage 1: UX701 Dose Level 1
Stage 1: UX701 Dose Level 2
Stage 1: UX701 Dose Level 3
Stage 2: UX701 or Placebo
Stage 3: Placebo or UX701
Participants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1.
Participants receive a single, peripheral IV infusion of UX701 at dose level 2.
Participants receive a single, peripheral IV infusion of UX701 at dose level 3.
Participants randomized 2:1 to receive UX701 or Placebo. Participants randomized to UX701 receive a single, peripheral IV infusion of UX701 at a dose selected in Stage 1. Participants randomized to placebo will receive a single, peripheral IV infusion of normal saline (placebo).
Participants randomized in Stage 2 to UX701 will receive a single, peripheral IV infusion of normal saline (placebo). Participants randomized in Stage 2 to placebo will be eligible for a single, peripheral IV infusion of UX701 at the selected dose.