Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

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Brief Title

Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

Official Title

A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

Brief Summary

      This exploratory study will investigate the effects of ALXN1840 on copper balance in
      participants with Wilson disease (WD).
    

Detailed Description

      Participants who are treatment experienced (which includes standard-of-care therapies or
      ALXN1840) and treatment naïve are eligible for this study.

      Safety will be monitored throughout the study.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Mean Daily Copper Balance

Secondary Outcome

 Change From Baseline In Mean Daily Copper Balance

Condition

Wilson Disease

Intervention

ALXN1840

Study Arms / Comparison Groups

 ALXN1840
Description:  Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39

Publications

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

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

10

Start Date

September 7, 2020

Completion Date

September 2021

Primary Completion Date

September 2021

Eligibility Criteria

        Inclusion Criteria:

          1. Diagnosis of WD by Leipzig Criteria > 4.

          2. Able to reside in the clinical research unit for intensive metabolic monitoring of
             copper and molybdenum.

          3. Participants willing to adhere to copper/molybdenum-controlled diet during the study.

          4. Willing and able to follow protocol-specified contraception requirements.

          5. Capable of giving signed informed consent.

        Exclusion Criteria:

          1. Decompensated cirrhosis or model for end stage liver disease score > 13.

          2. Modified Nazer score > 7.

          3. Clinically significant gastrointestinal bleed within past 3 months.

          4. Alanine aminotransferase > 2 × upper limit of normal.

          5. Hemoglobin less than lower limit of the reference range for age and sex.

          6. Significant medical history (current or past).

          7. Previous treatment with zinc within 30 days prior to the Screening Visit.

          8. Participants in renal failure, defined as in end-stage renal disease on dialysis
             (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, 855-752-2356, [email protected]

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations


NCT ID

NCT04573309

Organization ID

ALXN1840-WD-204

Secondary IDs

2020-001104-41

Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

, , 


Verification Date

September 2020