Status:
ACTIVE_NOT_RECRUITING
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
Lead Sponsor:
Ultragenyx Pharmaceutical Inc
Conditions:
Wilson Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief 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 to...
Detailed Description
Stage 1 (Phase 1/2) is an open-label safety and dose-finding stage designed to evaluate the safety and efficacy of 4 dose levels of UX701 to establish initial safety of UX701 and select a safe and eff...
Eligibility Criteria
Inclusion
- Key
- 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
- 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
Key Trial Info
Start Date :
September 27 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2034
Estimated Enrollment :
82 Patients enrolled
Trial Details
Trial ID
NCT04884815
Start Date
September 27 2021
End Date
March 1 2034
Last Update
December 12 2025
Active Locations (16)
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1
University of California Los Angeles
Los Angeles, California, United States, 90095
2
Stanford University
Redwood City, California, United States, 94063
3
University of California Davis
Sacramento, California, United States, 95817-1348
4
Northwestern University
Chicago, Illinois, United States, 60611