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

Alexion Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Wilson Disease

Treatments: Drug: ALXN1840

Study type: Interventional

Funder types: Industry

Identifiers

NCT02273596

WTX101-201

Details and patient eligibility

About

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.

Enrollment: 29 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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29 participants in 1 patient group

ALXN1840

Experimental group

Description:

Treatment Period: ALXN1840 at individualized doses ranging from 15 to 60 milligram (mg) per day. Dose increases or dose reductions were dependent on the individual NCC concentrations adjusted for Mo plasma concentration. ALXN1840 may have been administered every other day, once daily, or twice daily, depending on individualized dosing regimen, for 24 weeks. Extension Period: Participants continued the same ALXN1840 daily dose maintained at Week 24 of the Treatment Period and the same dosing regimen. During the Extension Period, no up-titration was made unless NCC concentrations adjusted for Mo plasma concentration did not remain stable within (or below) the reference range. ALXN1840 could have been received for up to 36 months in the Extension Period.

Treatment:

Drug: ALXN1840