Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
The primary objectives of this study are to evaluate the safety of single IV doses of UX701
in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile
based on the totality of safety and efficacy data and to evaluate the effect of UX701 on
copper regulation.
Phase
Phase 1/Phase 2
Funding Agency/Sponsor
Industry
Disease
Other
Enrollment Eligibility
Key Inclusion Criteria:
- Confirmed diagnosis of Wilson disease
- Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine,
trientine) and/or zinc therapy for at least 6 months at screening, with no medication
or dose changes for at least 6 months at screening.
- Ongoing restriction of high copper containing foods for at least 6 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 6 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