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The Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment

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ClinicalTrials.gov Identifier: NCT02763215
Recruitment Status : Active, not recruiting
First Posted : May 5, 2016
Last Update Posted : December 7, 2018
Sponsor:
Information provided by (Responsible Party):
Wilson Therapeutics AB

Brief Summary:

This is a 24-month study to assess copper (Cu) parameters in subjects with Wilson Disease treated with standard of care (SOC) medications. Data will be collected during routinely scheduled Wilson Disease clinic visits at approximately 6-month intervals.

After providing informed consent, subjects meeting all inclusion and no exclusion criteria will be enrolled into the study as outpatients. Subject's routine Wilson Disease clinic visits will be 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, subjects will be receiving SOC therapeutic agents for the treatment of Wilson Disease. If treatment is interrupted or stopped during the course of the study, subjects will continue in the study and biological samples and clinical data will continue to be collected for the full 24-month study period. Dosing with SOC agents, which can include penicillamine, trientine, zinc, or a combination of a Cu chelator and zinc, will be individualized and managed by the treating physician at the study center according to standard clinical practice at the site.


Condition or disease
Wilson Disease

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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment
Actual Study Start Date : May 19, 2016
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease




Primary Outcome Measures :
  1. Plasma Copper (Cu) parameter: Total Copper (Cu) [ Time Frame: 24 months ]
  2. Plasma Copper (Cu) parameter: Total Molybdenum (Mo) [ Time Frame: 24 months ]
  3. Plasma Copper (Cu) parameter: Speciation profiling [ Time Frame: 24 months ]
  4. Plasma Copper (Cu) parameter: Ceruloplasmin (Cp) measured by immune-methodology [ Time Frame: 24 months ]
  5. Plasma Copper (Cu) parameter: Non-ceruloplasmin-bound Cu (NCC) calculated [ Time Frame: 24 months ]
  6. Plasma Copper (Cu) parameter: Plasma Ultrafiltrate (PUF) Cu [ Time Frame: 24 months ]
  7. Plasma Copper (Cu) parameter: Plasma Ultrafiltrate (PUF) Mo [ Time Frame: 24 months ]
  8. Plasma Copper (Cu) parameter: exchangeable Cu (ExCu) [ Time Frame: 24 months ]
  9. Urine Copper (Cu) parameter: 24-hour urinary Cu [ Time Frame: 24 months ]
  10. Urine Copper (Cu) parameter: 24-hour urinary Mo [ Time Frame: 24 months ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Wilson Disease patients receiving standard of care (SOC) therapeutic agents for the treatment of WD. SOC agents can include penicillamine, trientine, zinc, or a combination of a Copper chelator and zinc.
Criteria

Inclusion Criteria:

  • Willing and able to give informed consent for participation in the study.
  • Male or female subjects, aged 18 years or older as of signing the ICF.
  • Receiving Standard of Care therapeutic agents (penicillamine, trientine, zinc, or copper chelators with zinc) for the treatment of Wilson Disease 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 Wilson Disease.
  • Adequate venous access to allow for collection of blood samples.

Exclusion Criteria:

  • Major systemic disease or other illness that would, in the opinion of the Investigator, compromise subject safety or interfere with the collection or interpretation of study results.
  • In the opinion of the Investigator, the subject is likely to be non-compliant or uncooperative during the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02763215


Locations
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United States, Connecticut
Clinical Trial Site
New Haven, Connecticut, United States, 06520
United States, Illinois
Clinical Trial Site
Chicago, Illinois, United States, 60611
United States, Michigan
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
United States, Tennessee
Clinical Trial Site
Nashville, Tennessee, United States, 37232
United States, Washington
Clinical Trial Site
Seattle, Washington, United States, 98105
Austria
Clinical Trial Site
Vienna, Austria, 1090
Germany
Clinical Trial Site
Heidelberg, Germany, 69120
Poland
Clinical Trial Site
Warsaw, Poland, 02-957
United Kingdom
Clinical Trial Site
Guildford, Surrey, United Kingdom, GU27XX
Clinical Trial Site
Birmingham, United Kingdom, B15 2TH
Sponsors and Collaborators
Wilson Therapeutics AB
Investigators
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Study Director: Eugene Swenson, MD, PhD Alexion Pharmaceuticals

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Responsible Party: Wilson Therapeutics AB
ClinicalTrials.gov Identifier: NCT02763215     History of Changes
Other Study ID Numbers: WTX101-203
First Posted: May 5, 2016    Key Record Dates
Last Update Posted: December 7, 2018
Last Verified: November 2018

Additional relevant MeSH terms:
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Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases
Copper
Trace Elements
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs