Efficacy and Safety Study of WTX101 in Adult Wilson Disease Patients

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Brief Title

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

Official Title

A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older With an Extension Phase of 36 Months

Brief Summary

      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.

Study Phase

Phase 2

Study Type


Primary Outcome

Percentage Of Participants With Normalized Concentrations Of NCC

Secondary Outcome

 Change From Baseline In NCC Concentrations Adjusted For Mo Plasma Concentration At Week 24


Wilson Disease



Study Arms / Comparison Groups

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.


* 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


Estimated Enrollment


Start Date

November 24, 2014

Completion Date

November 7, 2018

Primary Completion Date

October 27, 2016

Eligibility Criteria

        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.




18 Years - N/A

Accepts Healthy Volunteers



Eugene Swenson, MD, PhD, , 

Location Countries


Location Countries


Administrative Informations



Organization ID


Responsible Party


Study Sponsor

Alexion Pharmaceuticals

Study Sponsor

Eugene Swenson, MD, PhD, Study Director, Alexion Pharmaceuticals

Verification Date

September 2021