Clinical Trials in the USA / NCT04884815
Active, not recruiting
Phase 1/2
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
NCT04884815 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Wilson Disease
Investigational drug(s) / intervention(s)
UX701Standard of Care (SOC)
UX701: Nonreplicating, recombinant gene transfer vector
Standard of Care (SOC): SOC treatment (i.e., copper chelators and/or zinc) administered according to standard regimens.
Study 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.
Eligibility
Key Inclusion 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
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.
* 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
Primary outcome measure(s)
- Stage 1: Incidence of Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Adverse Events of Special Interest (AESIs), Treatment-Related TEAEs, and Treatment-Related TESAEs — Up to Week 52
- Stage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Change in Total Copper from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Change in Ceruloplasmin-bound Copper from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Change in Ceruloplasmin from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Change in Non-Ceruloplasmin-bound Copper (NCC) from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Change in Free Copper from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Change in Ceruloplasmin Activity from Baseline at Week 52 — Baseline, Week 52
- Stage 1: Percent Reduction in Standard of Care (SOC) Medication by Week 52 — Week 52
- Stage 1: Number of Participants Who Discontinue SOC Medication by Week 52 — Week 52
- Stage 1: Number of Consecutive Weeks off SOC Medication at Week 52 — Week 52
- Stage 2: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52, Evaluated for Superiority — Baseline, Week 52
- Stage 2: Percent Reduction in SOC Medication by Week 52, Evaluated for Superiority — Week 52
Trial sites (16)
| Facility | City | Region | Status |
|---|---|---|---|
| University of California Los Angeles | Los Angeles | California | |
| Stanford University | Redwood City | California | |
| University of California Davis | Sacramento | California | |
| Northwestern University | Chicago | Illinois | |
| Indiana University | Indianapolis | Indiana | |
| Massachusetts General Hospital | Boston | Massachusetts | |
| University of Michigan | Ann Arbor | Michigan | |
| Duke University Medical Center | Durham | North Carolina | |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | |
| Vanderbilt University Medical Center | Nashville | Tennessee | |
| University of Utah | Salt Lake City | Utah | |
| Seattle Children's Hospital | Seattle | Washington | |
| Centro Hospitalar Universitário Lisboa Norte | Lisbon | Lisbon District | |
| Centro Hospitalar Universitário de São João | Porto | Porto District | |
| Hospital Universitario Vall d'Hebron - PPDS | Barcelona | Spain | |
| Kings College NHS Foundation | London | Surrey |
More Ultragenyx Pharmaceutical Inc trials in the USA
Setrusumab vs Placebo for Osteogenesis Imperfecta
Active, not recruiting
Tumor-induced Osteomalacia Disease Monitoring Program
Active, not recruiting
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04884815 on ClinicalTrials.gov ↗ ← All trials in the USA