Status:
COMPLETED
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Wilson Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE2
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 concentratio...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
November 24 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 7 2018
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT02273596
Start Date
November 24 2014
End Date
November 7 2018
Last Update
September 29 2021
Active Locations (9)
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1
Clinical Trial Site
Los Angeles, California, United States, 90095
2
Clinical Trial Site
New Haven, Connecticut, United States, 06519
3
Clinical Trial Site
Chicago, Illinois, United States, 60611
4
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109