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

Description

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.

Conditions

Wilson Disease

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Study Overview

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Study Details

Study overview

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.

An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults With Wilson Disease

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

Condition
Wilson Disease
Intervention / Treatment

-

Contacts and Locations

Los Angeles

University of California Los Angeles, Los Angeles, California, United States, 90095

Redwood City

Stanford University, Redwood City, California, United States, 94063

Sacramento

University of California Davis, Sacramento, California, United States, 95817-1348

Chicago

Northwestern University, Chicago, Illinois, United States, 60611

Indianapolis

Indiana University, Indianapolis, Indiana, United States, 46202

Boston

Massachusetts General Hospital, Boston, Massachusetts, United States, 02114

Ann Arbor

University of Michigan, Ann Arbor, Michigan, United States, 48109

Durham

Duke University Medical Center, Durham, North Carolina, United States, 27710

Cleveland

University Hospitals Cleveland Medical Center, Cleveland, Ohio, United States, 44106

Nashville

Vanderbilt University Medical Center, Nashville, Tennessee, United States, 37212-2700

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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.

Ages Eligible for Study

18 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ultragenyx Pharmaceutical Inc,

Medical Director, STUDY_DIRECTOR, Ultragenyx Pharmaceutical Inc

Study Record Dates

2034-03