Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

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Brief Title

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Official Title

Clinical Evaluation and Assessment of Instruments and Biomarkers in Subjects With Wilson Disease

Brief Summary

      The primary objective of the study is to determine the relevance and appropriateness of
      outcome assessments, including biomarkers, within the Wilson disease population to inform
      study design and endpoint selection for future clinical studies.
    

Detailed Description

      Given the limited information on the frequency and spectrum of disease manifestations and
      clinical course of Wilson disease, the UX701-CL001 study aims to assess the utility and
      feasibility of various assessments and biomarkers to inform endpoint selection for future
      clinical studies, better understand the relationship between biomarkers and potential
      clinical outcomes, and characterize the clinical presentation of Wilson disease. UX701-CL001
      is a clinical survey study. Subjects will complete assessments at the study site and at home
      to evaluate the clinical manifestations of Wilson disease in clinical and real-world
      environments.
    


Study Type

Observational


Primary Outcome

Clinical manifestation of Wilson Disease under study: demographics


Condition

Wilson Disease



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

35

Start Date

October 2020

Completion Date

January 2021

Primary Completion Date

January 2021

Eligibility Criteria

        Inclusion Criteria:

          1. Male or female ≥ 12 years of age at the time written informed consent is provided.

          2. Confirmed diagnosis of Wilson disease.

          3. Have a documented history of copper chelator (ie, penicillamine, trientine) and/or
             zinc therapy or be ≥ 1 year post liver transplant with no active associated
             complications.

          4. Willing and able to comply with all study procedures and requirements. If < 18 years
             of age (or as required by region), have a caregiver who is willing and able to assist
             with study requirements if needed.

          5. Willing and able to provide written informed consent after the study has been
             explained and before any study-related data are collected or study-related procedures
             are performed. If < 18 years of age (or as required by region), willing and able to
             provide written assent and have a legally authorized representative who is willing and
             able to provide written informed consent after the study has been explained and before
             any study-related data are collected or study-related procedures are performed.

        Exclusion Criteria:

          1. History of liver disease due to a medical condition unrelated to Wilson disease.

          2. Liver fibrosis stage F3 or F4.

          3. Decompensated hepatic cirrhosis and/or evidence of portal hypertension.

          4. Marked neurological disease requiring either nasogastric feeding or intensive
             inpatient medical care.

          5. Female subject who is pregnant or breastfeeding or who plans to become pregnant at any
             time during the study.

          6. Female subject of childbearing potential who has a positive urine pregnancy test on
             Day 1 or is unwilling to have additional pregnancy tests during the study.

          7. Current or previous participation in a gene transfer study.

          8. Presence or history of any disease or condition that, in the Investigator's opinion,
             would interfere with the subject's safety or ability to participate in the study or
             significantly affect interpretation of study results.
      

Gender

All

Ages

12 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Ultragenyx Medical Director, 1-415-483-8800, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04531189

Organization ID

UX701-CL001


Responsible Party

Sponsor

Study Sponsor

Ultragenyx Pharmaceutical Inc


Study Sponsor

Ultragenyx Medical Director, Study Director, Ultragenyx Pharmaceutical


Verification Date

September 2020