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

Study Identifier:
WTX101-201
ClinicalTrials.gov Identifier:
NCT02273596
EudraCT Identifier:
N/A
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Study Complete

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: ALXN1840
Date
Nov 2014 - Oct 2016
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and Exclusion 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.
Sex
Female & Male
Age
18+ years

Study Details

Medical Condition
  • Unmapped
Study Drug
  • Drug: ALXN1840
Date
Nov 2014 - Oct 2016
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 18+ years years
Requirements Information
Inclusion and Exclusion 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.

Protocol 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.

Trial Locations

Location
Status
Location
Clinical Trial Site
Los Angeles, California, United States, 90095
Status
N/A
Location
Clinical Trial Site
New Haven, Connecticut, United States, 06519
Status
N/A
Location
Clinical Trial Site
Chicago, Illinois, United States, 60611
Status
N/A
Location
Clinical Trial Site
Ann Arbor, Michigan, United States, 48109
Status
N/A
Location
Clinical Trial Site
Vienna, Austria, 1090
Status
N/A
Location
Clinical Trial Site
Heidelberg, Germany, 69120
Status
N/A
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