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.
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
May 31, 2022
Primary Completion Date
May 31, 2022
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
Eugene S. Swenson, MD, PhD, 855-752-2356, [email protected]
Location Countries
New Zealand
Location Countries
New Zealand
Administrative Informations
NCT ID
NCT04573309
Organization ID
ALXN1840-WD-204
Secondary IDs
2020-001104-41
Responsible Party
Sponsor
Study Sponsor
Alexion Pharmaceuticals
Study Sponsor
Eugene S. Swenson, MD, PhD, Study Director, Alexion Pharmaceuticals
Verification Date
May 2022