CompletedPhase 2

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

United States, New Zealand, United Kingdom9 participantsStarted 2020-09-07

Plain-language summary

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

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Diagnosis of WD by Leipzig Criteria ≥ 4.
✓. Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
✓. Participants willing to adhere to copper/molybdenum-controlled diet during the study.
✓. Willing and able to follow protocol-specified contraception requirements.
✓. Capable of giving signed informed consent.

✕. Decompensated cirrhosis or model for end stage liver disease score \> 13.
✕. Modified Nazer score \> 7.
✕. Clinically significant gastrointestinal bleed within past 3 months.
✕. Alanine aminotransferase \> 2 × upper limit of normal.
✕. Hemoglobin less than lower limit of the reference range for age and sex.
✕. Significant medical history (current or past).
✕. Previous treatment with zinc within 30 days prior to the Screening Visit.
✕. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance \< 30 milliliters/minute.

Mean Daily Copper Balance: Day 1 Through Day 8

Timeframe: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)

Mean Daily Copper Balance: Day 31 Through Day 35

Timeframe: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)

Mean Daily Copper Balance: Day 25 Through Day 28

Timeframe: Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)

Mean Daily Copper Balance: Day 36 Through Day 39

Timeframe: Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)

NCT IDNCT04573309

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2022-06-07

Results submitted2023-07-31

Who can participate