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 (Mo)
      plasma concentration in participants newly diagnosed with Wilson Disease (WD) aged 18 and
      older and with 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
      36-month Extension Period.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Percentage Of Participants With Normalized Concentrations Of NCC

Secondary Outcome

 Time To Normalization Of NCC Adjusted For Mo Plasma Concentration

Condition

Wilson Disease

Intervention

ALXN1840

Study Arms / Comparison Groups

 ALXN1840
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. Up-titration was permitted if NCC concentrations adjusted for Mo plasma concentration did not remain stable within (or below) the reference range. ALXN1840 was administered for up to 36 months.

Publications

* 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

Drug

Estimated Enrollment

28

Start Date

November 24, 2014

Completion Date

November 7, 2018

Primary Completion Date

October 27, 2016

Eligibility Criteria

        Inclusion Criteria:

          -  Willing and able to give informed consent for participation in the study.

          -  Male or female participants, aged 18 years or older as of signing the informed consent
             form.

          -  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 (MELD) score > 11.

          -  Modified Nazer score > 6.

          -  Gastrointestinal bleed within past 6 months.

          -  Alanine aminotransferase > 5 x upper limit of normal (ULN).

          -  Marked neurological disease requiring either nasogastric feeding or intensive
             in-patient medical care.

          -  Severe anemia with a hemoglobin < 9 gram/deciliter (g/dL).
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Eugene Swenson, MD, PhD, , 

Location Countries

Austria

Location Countries

Austria

Administrative Informations


NCT ID

NCT02273596

Organization ID

WTX101-201


Responsible Party

Sponsor

Study Sponsor

Alexion Pharmaceuticals


Study Sponsor

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


Verification Date

October 2020