Wilson DiseaseNCT02763215
The Assessment of Copper Parameters in Wilson Disease Participants on Standard of Care Treatment
An observational study of Wilson Disease, run by Alexion Pharmaceuticals, Inc. at 10 sites in 5 countries.
Trial summary
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications. After providing informed consent, participants meeting all inclusion and no exclusion criteria were enrolled into the study as outpatients. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Wilson Disease
- How many people
- 64 participants
- Where
- 10 sitesin 5 countries
- Study dates
- 19 May 2016to 21 Jan 2019
- Phase
- Observational
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Wilson disease participants receiving SoC medications for the treatment of WD. Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Who may be able to join
- Willing and able to give informed consent for participation in the study.
- Male or female participants, aged 18 years or older as of signing the informed consent form.
- Receiving SoC medications (penicillamine, trientine, zinc, or copper chelators with zinc) for the treatment of WD at the time of enrollment and for no more than 60 months prior to enrollment.
- Able to understand and willing to comply with study procedures and requirements, as judged by the Investigator.
- Established diagnosis of WD.
- Adequate venous access to allow for collection of blood samples.
Who cannot join
- Major systemic disease or other illness that would, in the opinion of the Investigator, compromise participant safety or interfere with the collection or interpretation of study results.
- In the opinion of the Investigator, the participant was likely to be non-compliant or uncooperative during the study.
Trial locations and contacts
10 sites in 5 countries.
10 sites in 5 countries
United States5 sites
Clinical Trial Site
Ann Arbor, Michigan, 48109
Clinical Trial Site
Chicago, Illinois, 60611
Clinical Trial Site
Nashville, Tennessee, 37240
Clinical Trial Site
New Haven, Connecticut, 06504
Clinical Trial Site
Seattle, Washington, 98105
United Kingdom2 sites
Clinical Trial Site
Birmingham, B15 2GW
Clinical Trial Site
Guildford, GU2 7XX
About this study
Official title: Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
After providing informed consent, participants meeting all inclusion and no exclusion criteria were enrolled into the study as outpatients. The participants' routine clinic visits were scheduled according to the standard clinical practice at the study center and at the discretion of the treating physician at approximate 6-month intervals.
At the time of enrollment, participants were receiving SoC medications for the treatment of WD, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc. If treatment was interrupted or stopped during the course of the study, participants continued in the study and biological samples and clinical data were continued to be collected for the full 24-month study period. Dosing with SoC agents was individualized and managed by the treating physician at the study center according to standard clinical practice at the site.
- Conditions
- Wilson Disease
- Being tested
- Standard of Care Medications
- Medical subject headings
- Hepatolenticular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard of Care MedicationsDrug
Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
| Study group | Role | What they receive |
|---|---|---|
| Total | Not described Receives: Drug: Standard of Care Medications |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6 Months Of Treatment
To achieve a normalized NCC concentration, participants must have had 2 consecutive measures within (or below) the normal concentration range (0.8 to 2.3 micromolar [μM]). Two consecutive measurements required that the measurements occurred on separate dates and were assigned to 2 different visits. Non-ceruloplasmin-bound copper was calculated by subtracting the amount of copper bound to the ceruloplasmin from the total plasma copper concentration.
Measured: Baseline through Month 6
Secondary outcomes (17)
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of NCC Or Reached A Reduction Of ≥ 25% In NCC Through Last Assessment
To achieve a normalized NCC concentration, participants must have 2 consecutive measures within (or below) the normal range (0.8 to 2.3 μM). Two consecutive measurements required that the measurements occurred on separate dates and were assigned to 2 different visits. Non-ceruloplasmin-bound copper was calculated by subtracting the amount of copper bound to the ceruloplasmin from the total plasma copper concentration. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment
Evaluation of NCC concentrations over time are reported as micromoles/liter (μmol/L). Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Time To Normalization Of NCC If Above The Reference Range At The Time Of Enrollment
The time to normalization of NCC was measured by Kaplan-Meier analysis.
Measured: Baseline, up to 24 months
Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment
The change in exchangeable copper was evaluated over time and is reported as nanograms/milliliter (ng/mL). Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment
Change in copper plasma ultrafiltrate was measured over time and is reported as ng/mL. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment
Plasma total copper was measured over time and is reported as ng/mL. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment
The total serum (nephelometry) was measured over time and is reported as milligrams (mg)/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment
Evaluation of 24-hour urinary copper over time is reported as micrograms (μg)/day. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24
The plasma total molybdenum was measured over time and is reported in ng/mL.
Measured: Baseline, Month 6, Month 24
Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24
The molybdenum plasma ultrafiltrate was measured over time and is reported as ng/mL.
Measured: Baseline, Month 6, Month 24
Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24
Evaluation of 24-hour urinary molybdenum over time is reported as μg/day.
Measured: Baseline, Month 6, Month 24
Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment
The ALT levels were measured over time and are reported as units (U)/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment
The AST levels were measured over time and are reported as U/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment
Bilirubin was measured over time and is reported as μmol/L. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment
The INR was measured over time. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment
The CGI scale item 1 (severity of illness) rating scale is a commonly used measure of symptom severity, treatment response, and the efficacy of treatments in treatment studies of participants with mental disorders. It uses the Clinical Global Impression - Severity Scale (CGI-S), a 7-point scale that requires the Investigator to rate the severity of the participant's illness at the time of assessment, relative to the Investigator's past experience with participants who had the same diagnosis. Considering total clinical experience, a participant was assessed on severity of illness at the time of rating as: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill. A decrease in score indicates improvement in disease severity. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment
The CGI scale item 2 (global improvement) is a rating scale commonly used measure of symptom severity, treatment response, and the efficacy of treatments in treatment studies of participants with mental disorders. It uses the Clinical Global Impression - Improvement Scale (CGI-I), a 7-point scale that requires the Investigator to assess how much the participant's illness has improved or worsened relative to the Baseline state at the beginning of the study and rate as: 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. A decrease in score indicates improvement in disease severity. Last Assessment was the last available post-baseline result for each participant (ranging from 1 to 24 months).
Measured: Baseline through Last Assessment (ranging from 1 to 24 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2016 (estimated)
- Study start19 May 2016
- Primary completion21 Jan 2019
- Study completion21 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Wilson DiseaseStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alexion Pharmaceuticals, Inc., Study director, Alexion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02763215
- Sponsor's protocol ID
- WTX101-203
- Status last verified
- Nov 2020
- First submitted
- 3 May 2016
- Last updated
- 1 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment
- ClinicalTrials.gov ID
- NCT02763215
- Acronym / sponsor ID
- WTX101-203
- Conditions
- Wilson Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 May 2016 (actual)
- Primary completion
- 21 Jan 2019 (actual)
- Study completion
- 21 Jan 2019 (actual)
- First posted
- 5 May 2016 (estimated)
- Results
- Posted 26 Oct 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
After providing informed consent, participants meeting all inclusion and no exclusion criteria were enrolled into the study as outpatients. The participants' routine clinic visits were scheduled according to the standard clinical practice at the study center and at the discretion of the treating physician at approximate 6-month intervals.
At the time of enrollment, participants were receiving SoC medications for the treatment of WD, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc. If treatment was interrupted or stopped during the course of the study, participants continued in the study and biological samples and clinical data were continued to be collected for the full 24-month study period. Dosing with SoC agents was individualized and managed by the treating physician at the study center according to standard clinical practice at the site.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Total | – | Drug: Standard of Care Medications |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Wilson disease participants receiving SoC medications for the treatment of WD. Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.
Key inclusion criteria (6 listed)
- Willing and able to give informed consent for participation in the study.
- Male or female participants, aged 18 years or older as of signing the informed consent form.
- Receiving SoC medications (penicillamine, trientine, zinc, or copper chelators with zinc) for the treatment of WD at the time of enrollment and for no more than 60 months prior to enrollment.
- Able to understand and willing to comply with study procedures and requirements, as judged by the Investigator.
- Established diagnosis of WD.
Key exclusion criteria (2 listed)
- Major systemic disease or other illness that would, in the opinion of the Investigator, compromise participant safety or interfere with the collection or interpretation of study results.
- In the opinion of the Investigator, the participant was likely to be non-compliant or uncooperative during the study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Standard of Care MedicationsStandard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc. | Drug | Total |
Primary outcome
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6 Months Of Treatment
Time frame: Baseline through Month 6
To achieve a normalized NCC concentration, participants must have had 2 consecutive measures within (or below) the normal concentration range (0.8 to 2.3 micromolar [μM]). Two consecutive measurements required that the measurements occurred on separate dates and were assigned to 2 different visits. Non-ceruloplasmin-bound copper was calculated by subtracting the amount of copper bound to the ceruloplasmin from the total plasma copper concentration.
17 secondary outcomes
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of NCC Or Reached A Reduction Of ≥ 25% In NCC Through Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Time To Normalization Of NCC If Above The Reference Range At The Time Of Enrollment
Time frame: Baseline, up to 24 months
Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24
Time frame: Baseline, Month 6, Month 24
Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24
Time frame: Baseline, Month 6, Month 24
Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24
Time frame: Baseline, Month 6, Month 24
Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment
Time frame: Baseline through Last Assessment (ranging from 1 to 24 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexion Pharmaceuticals, Inc. study director
Alexion Pharmaceuticals, Inc.
10 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Wilson Disease, run by Alexion Pharmaceuticals, Inc. at 10 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, United Kingdom, Austria, Germany, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Oct 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6 Months Of Treatment (time frame: Baseline through Month 6). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Dec 2020.