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oral corticosteroids for Wilson's Disease

University of California Davis, Sacramento, CA
Wilson's Diseaseoral corticosteroids - Drug
Eligibility
18+
All Sexes
What conditions do you have?
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Study Summary

This trial is testing a new drug, UX701, to see if it is safe for people with Wilson Disease and if it helps regulate copper levels.

Eligible Conditions
  • Wilson's Disease

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 1 & 2

Similar Trials

1
High Dose LX2006
1
SAR443122 level 1
1
AT845

Study Objectives

19 Primary · 6 Secondary · Reporting Duration: Up to Week 312

Baseline, Week 52
Stage 1: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52
Stage 1: Change in Ceruloplasmin Activity from Baseline at Week 52
Stage 1: Change in Ceruloplasmin from Baseline at Week 52
Copper
Copper
Stage 1: Change in Total Ceruloplasmin Activity from Baseline at Week 52
Stage 1: Change in Total Ceruloplasmin from Baseline at Week 52
Stage 1: Change in Total Copper from Baseline at Week 52
Stage 1: Change in Total Free Copper from Baseline at Week 52
Stage 1: Change in Total Non-Ceruloplasmin-bound Copper (NCC) from Baseline at Week 52
Stage 2: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52, Evaluated for Superiority
Stage 2: Change in 24-hour Urinary Copper Concentration from Baseline at Week 52, evaluated for superiority
Stage 2: Change in Ceruloplasmin Activity Levels from Baseline at Week 52, Evaluated for Superiority
Stage 2: Change in Liver Copper Concentration Assessed by Liver Biopsy from Baseline at Week 52
Stage 2: Change in WDFRS Clinician Scores from Baseline at Week 52
Stage 2: Change in Wilson Disease Functional Rating Scale (WDFRS) Patient Scores from Baseline at Week 52, Evaluated for Superiority
Up to Week 104
Stage 2: Development of Anti-ATP7B Antibodies
Up to Week 312
Incidence of TEAEs, TESAEs, Treatment-Related TEAEs, and Treatment-Related TESAEs
Therapeutic procedure
Up to Week 52
Incidence of Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), Treatment-Related TEAEs, and Treatment-Related TESAEs
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
Week 52
Stage 1: Number of Consecutive Weeks off SOC Medication at Week 52
Pharmaceutical Preparations
Stage 1: Number of Subjects Who Discontinue SOC Medication by Week 52
Stage 1: Percent Reduction in Standard of Care (SOC) Medication by Week 52
Stage 2: Number of Participants who Discontinue SOC Medication by Week 52
Stage 2: Number of Subjects who Discontinue SOC Medication by Week 52
Pharmaceutical Preparations

Trial Safety

Phase-Based Safety

1 of 3

Similar Trials

1
High Dose LX2006
2
SAR443122 level 1
1
AT845

Awards & Highlights

All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

12 Treatment Groups

Stage 2: UX701 or Placebo
1 of 12
Stage 1: UX701 Dose Level 2
1 of 12
Stage 2: UX701
1 of 12
Stage 3: UX701
1 of 12
Stage 1: UX701 Dose Level 3
1 of 12
Stage 1: UX701 Dose Level 1
1 of 12
Stage 3: Placebo or UX701
1 of 12
Stage 1: Placebo Dose Level 2
1 of 12
Stage 3: Placebo
1 of 12
Stage 1: Placebo Dose Level 3
1 of 12
Stage 1: Placebo for Dose Level 1
1 of 12
Stage 2: Placebo
1 of 12

Experimental Treatment

Non-Treatment Group

78 Total Participants · 12 Treatment Groups

Primary Treatment: oral corticosteroids · Has Placebo Group · Phase 1 & 2

Stage 2: UX701 or PlaceboExperimental Group · 4 Interventions: oral corticosteroids, Placebo for oral corticosteroids, UX701, Placebo · Intervention Types: Drug, Drug, Genetic, Other
Stage 1: UX701 Dose Level 2Experimental Group · 2 Interventions: oral corticosteroids, UX701 · Intervention Types: Drug, Genetic
Stage 2: UX701Experimental Group · 2 Interventions: oral corticosteroids, UX701 · Intervention Types: Drug, Genetic
Stage 3: UX701Experimental Group · 2 Interventions: oral corticosteroids, UX701 · Intervention Types: Drug, Genetic
Stage 1: UX701 Dose Level 3Experimental Group · 2 Interventions: oral corticosteroids, UX701 · Intervention Types: Drug, Genetic
Stage 1: UX701 Dose Level 1Experimental Group · 2 Interventions: oral corticosteroids, UX701 · Intervention Types: Drug, Genetic
Stage 3: Placebo or UX701Experimental Group · 4 Interventions: oral corticosteroids, Placebo for oral corticosteroids, UX701, Placebo · Intervention Types: Drug, Drug, Genetic, Other
Stage 1: Placebo Dose Level 2PlaceboComparator Group · 2 Interventions: Placebo for oral corticosteroids, Placebo · Intervention Types: Drug, Other
Stage 3: PlaceboPlaceboComparator Group · 2 Interventions: Placebo for oral corticosteroids, Placebo · Intervention Types: Drug, Other
Stage 1: Placebo Dose Level 3PlaceboComparator Group · 2 Interventions: Placebo for oral corticosteroids, Placebo · Intervention Types: Drug, Other
Stage 1: Placebo for Dose Level 1PlaceboComparator Group · 2 Interventions: Placebo for oral corticosteroids, Placebo · Intervention Types: Drug, Other
Stage 2: PlaceboPlaceboComparator Group · 2 Interventions: Placebo for oral corticosteroids, Placebo · Intervention Types: Drug, Other

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: up to week 312

Who is running the clinical trial?

Ultragenyx Pharmaceutical IncLead Sponsor
86 Previous Clinical Trials
179,044 Total Patients Enrolled
Medical DirectorStudy DirectorUltragenyx Pharmaceutical Inc
2,668 Previous Clinical Trials
7,959,131 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 0 Total Inclusion Criteria

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References

Frequently Asked Questions

What are the prominent conditions that UX701 is utilized to address?

"UX701 is often used to manage allergic contact dermatitis, but its efficacy also extends to treating nephrotic syndrome, vasculitis, and synovitis." - Anonymous Online Contributor

Unverified Answer

What is the current scope of this trial's presence in clinics?

"This medical experiment is being conducted at Indiana University in Indianapolis, the University of Miami in Florida, and UCLA in California. Additionally, there are 10 other sites participating as well." - Anonymous Online Contributor

Unverified Answer

How many participants are in this clinical trial?

"Affirmative. Clinicaltrials.gov's data reveals that this study, first posted on September 27th 2021 is currently recruiting participants. Approximately 78 volunteers must be registered from 10 clinical sites." - Anonymous Online Contributor

Unverified Answer

Has there been previous research examining UX701?

"UX701 was initially investigated by Karolinska Institutet in 2002, with a total of 18518 trials completed to date. There are 87 clinical experiments presently taking place, predominantly centered around Indianapolis, Indiana." - Anonymous Online Contributor

Unverified Answer

Are there any vacancies within this clinical investigation?

"As confirmed on clinicaltrials.gov, this trial is currently seeking participants and has been since it was published on September 27th 2021 with the most recent update being issued November 2nd 2022." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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