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

NCT02763215 | Completed Results

• 1 other identifier: WTX101-203
• Study Type: observational
• Target: 64
• Locations: 5 countries
• Sites: 10

Brief 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. 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

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  Baseline through Last Assessment (ranging from 1 to 24 months)

• Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment

  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

  Baseline, up to 24 months

• Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment

  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

  Baseline through Last Assessment (ranging from 1 to 24 months)

Study Arms (1)

Total

Drug: Standard of Care Medications

Interventions

Standard of Care Medications DRUG

Standard of care medications could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc.

Total

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (10)

Clinical Trial Site

New Haven, Connecticut, 06504, United States

Location

Clinical Trial Site

Chicago, Illinois, 60611, United States

Location

Clinical Trial Site

Ann Arbor, Michigan, 48109, United States

Location

Clinical Trial Site

Nashville, Tennessee, 37240, United States

Location

Clinical Trial Site

Seattle, Washington, 98105, United States

Location

Clinical Trial Site

Vienna, 1090, Austria

Location

Clinical Trial Site

Heidelberg, 69120, Germany

Location

Clinical Trial Site

Warsaw, 02-957, Poland

Location

Clinical Trial Site

Birmingham, B15 2GW, United Kingdom

Location

Clinical Trial Site

Guildford, GU2 7XX, United Kingdom

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

Only AEs associated with study-related interventional testing or assessments (that is, venipuncture) were collected.

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Study Officials

• Alexion Pharmaceuticals, Inc.

  Alexion Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2016
• First Posted: May 5, 2016
• Study Start: May 19, 2016
• Primary Completion: January 21, 2019
• Study Completion: January 21, 2019
• Last Updated: December 1, 2020
• Results First Posted: October 26, 2020

Record last verified: 2020-11

Locations

GB (2); US (5); AU (1); DE (1); PL (1)