Trial · NCT04884815
A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
NCT04884815, Phase 1, Phase 2, Active not recruiting, Ultragenyx Pharmaceutical Inc
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease. Trials, record NCT04884815. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04884815
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease
- NCT number
- NCT04884815
- Status
- Active not recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 82 participants, estimated
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Wilson Disease
- Interventions
- UX701, Standard of Care (SOC)
- First posted
- 2021-05-13
- Last updated
- 2026-09-25
- Other registries' IDs
- eudract:2020-005266-34
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | 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 | |
| Primary | Stage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Change in Total Copper from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Change in Ceruloplasmin-bound Copper from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Change in Ceruloplasmin from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Change in Non-Ceruloplasmin-bound Copper (NCC) from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Change in Free Copper from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Change in Ceruloplasmin Activity from Baseline at Week 52 | Baseline, Week 52 | |
| Primary | Stage 1: Percent Reduction in Standard of Care (SOC) Medication by Week 52 | Week 52 | |
| Primary | Stage 1: Number of Participants Who Discontinue SOC Medication by Week 52 | Week 52 |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04884815
GET https://conductscience.org/api/trials/v1/NCT04884815/registered