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

Alexion Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Wilson Disease

Treatments: Drug: ALXN1840

Study type: Interventional

Funder types: Industry

Identifiers

NCT04573309

2020-001104-41 (EudraCT Number)

ALXN1840-WD-204

Details and patient eligibility

About

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

Full description

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

Enrollment: 9 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

ALXN1840

Experimental group

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

Treatment:

Drug: ALXN1840