Study of UX701 Gene Transfer for the Treatment of Wilson Disease
C
Craig Lammert, MD
Primary Investigator
Enrolling By Invitation
18 years and older
All
Phase
1/2
78 participants needed
1 Location
Brief description of study
The primary objectives of this study are to evaluate the safety of single IV doses of UX701with 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 ongulation.
Detailed description of study
This study is a randomized, double-blind, placebo-controlled, seamless, adaptive Phase 1/2/3udy of UX701 in patients with Wilson disease.
Stage 1 (Phase 1/2) is a nonrandomized, open-label safety and dose-finding stage designed tovaluate the safety and efficacy of 3 dose levels of UX701 to establish initial safety of701 and select a safe and efficacious dose for further evaluation. Stage 2 (Phase 3) is adomized, double-blind, placebo-controlled stage designed to evaluate the safety andy of UX701 using the dose selected in Stage 1. Stage 3 is designed to evaluate theg-term safety, efficacy, and clinical benefit of UX701. All participants will be followed5 years from the time of UX701 administration.
Participants who receive UX701 will receive prophylactic oral corticosteroids. Participants
who receive placebo will receive placebo oral corticosteroids to maintain the study blind.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Wilson Disease
-
Age: 18 Years
-
Gender: All
Key Inclusion Criteria:
- Confirmed diagnosis of Wilson disease
- Ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at12 months at screening, with no medication or dose changes for at least 6 monthsg.
- Stable Wilson disease as evidenced by stable 24-hour urinary copper concentrationduring the Screening Period
- Ongoing restriction of high copper containing foods for at least 12 months atg, continued through study participation.
- Willing and able to comply with all study procedures and requirements, includingquent blood collection, total urine collection over a 24-hour period,d 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 thevestigator's judgment, within 12 months prior to Screening.
- History of liver transplant.
- Decompensated hepatic cirrhosis or presence of advanced liver disease as evidenced byhypertension, ascites, splenomegaly, esophageal varices, hepatichalopathy.
- 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< 60 mL/min/1.73 m2.
- Marked neurological deficit or compromise that, in the Investigator's opinion, wouldwith the subject's safety or ability to participate in the study.
- Moderate to severe depression, recent or active suicidal ideation with intent oruicidal behavior, psychosis, or unstable psychiatric illness.
- Participation in another gene transfer study or use of another gene transfer productbefore or during study participation.
- Subjects with known hypersensitivity to amide-containing local anesthetics areuded from participating in the optional liver biopsy substudy.
Note: Other protocol defined Inclusion/ Exclusion criteria may apply
Updated on
01 Aug 2024.
Study ID: UX701-CL301
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