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