Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients

Completed · Phase 2

Conditions studied: Wilson Disease

In brief

The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly diagnosed with Wilson Disease (WD) who were aged 18 and older and who had NCC concentrations within or above the reference range at the time of enrollment in the study. The study consisted of a 24-week Treatment Period, followed by a planned 36-month Extension Period.

Key facts

Study ID

NCT02273596

Run by

Alexion Pharmaceuticals, Inc.

People needed

29

Starts

2014-11-24

Expected to finish

2018-11-07

Last updated by the study team

2021-09-29

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator.
• Newly established diagnosis of WD by Leipzig-Score ≥ 4 documented by testing as outlined in 2012 European Association for the Study of the Liver Wilson Disease Clinical Practice Guidelines.
• NCC levels within or above the normal reference range (0.8 to 2.3 micromole).
• Willing to undergo 48 hour washout from current WD treatment

You may not qualify if…

• Treatment for greater than 24 months for WD with chelation therapy (for example, penicillamine, trientine hydrochloride) or zinc therapy.
• Decompensated hepatic cirrhosis.
• Model for End-Stage Liver Disease score > 11.
• Modified Nazer score > 6.
• Gastrointestinal bleed within past 6 months.
• Alanine aminotransferase > 5 x upper limit of normal.
• Marked neurological disease requiring either nasogastric feeding or intensive in-patient medical care.
• Severe anemia with a hemoglobin < 9 grams/deciliter.

Where it is running

• Clinical Trial Site — Los Angeles, California, United States
• Clinical Trial Site — New Haven, Connecticut, United States
• Clinical Trial Site — Chicago, Illinois, United States
• Clinical Trial Site — Ann Arbor, Michigan, United States
• Clinical Trial Site — Vienna, Austria
• Clinical Trial Site — Heidelberg, Germany
• Clinical Trial Site — Warsaw, Poland
• Clinical Trial Site — Guildford, Surrey, United Kingdom
• Clinical Trial Site — Birmingham, United Kingdom

Full record on ClinicalTrials.gov

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