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NCT04884815 · ClinicalTrials.gov registry record · Phase 1
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
A Phase 1 study of Wilson Disease, sponsored by Ultragenyx Pharmaceutical.
- Active
- Registry status
- Phase 1
- Development phase
- 82
- Enrollment target
- 16
- Study locations
NCT04884815: Active Phase 1 study of Wilson Disease, sponsored by Ultragenyx Pharmaceutical.
NCT04884815 is a Phase 1 study of Wilson Disease that is active but no longer recruiting, run by Ultragenyx Pharmaceutical. The registered enrollment target is 82 participants, below the 106-participant average among 4 other Wilson Disease trials with a reported enrollment target (23% lower). The trial reports 16 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04884815, a Phase 1 study of Wilson Disease, is active but no longer recruiting, sponsored by Ultragenyx Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 82 participants
- Enrollment target
- 16
- Study locations
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.
Conditions Studied
Interventions
- DRUG Standard of Care (SOC)
- GENETIC UX701
Study Locations (16)
California
- University of California Los Angeles - Los Angeles
- Stanford University - Redwood City
- University of California Davis - Sacramento
Illinois
- Northwestern University - Chicago
Indiana
- Indiana University - Indianapolis
Massachusetts
- Massachusetts General Hospital - Boston
Michigan
- University of Michigan - Ann Arbor
North Carolina
- Duke University Medical Center - Durham
Tennessee
- Vanderbilt University Medical Center - Nashville
Utah
- University of Utah - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 82 participants |
| Start Date | 2021-09-27 |
| Est. Completion | 2034-03 |
| Phase | Phase 1 |
What the registry record for NCT04884815 still lists
NCT04884815 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 82 participants, a relatively small participant target, below the 106-participant average among 4 other Wilson Disease trials with a reported enrollment target (23% lower).
The record links to 1 condition, with Wilson Disease appearing as the primary indexed condition, and to 2 interventions - of which Standard of Care (SOC) is the first listed.
NCT04884815 lists 16 locations in 14 states (California, Illinois, Indiana).
Frequently Asked Questions
What is clinical trial NCT04884815 about?
NCT04884815 is a clinical study titled "A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease". 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.
What is the current status of trial NCT04884815?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 82 participants. The study started on 2021-09-27. Estimated completion is 2034-03.
What conditions does trial NCT04884815 study?
This clinical trial studies the following conditions: Wilson Disease.
What interventions are being tested in trial NCT04884815?
The interventions under investigation include: Standard of Care (SOC) (DRUG), UX701 (GENETIC).
Who is sponsoring clinical trial NCT04884815?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04884815 being conducted?
This trial has 16 study locations across California, Illinois, Indiana, Massachusetts, Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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