Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
NCT ID: NCT02273596
Last Updated: 2021-09-29
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
29 participants
INTERVENTIONAL
2014-11-24
2018-11-07
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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ALXN1840
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.
ALXN1840
Individualized oral doses of ALXN1840.
Interventions
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ALXN1840
Individualized oral doses of ALXN1840.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
ALL
No
Sponsors
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Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Eugene Swenson, MD, PhD
Role: STUDY_DIRECTOR
Alexion Pharmaceuticals, Inc.
Locations
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Clinical Trial Site
Los Angeles, California, United States
Clinical Trial Site
New Haven, Connecticut, United States
Clinical Trial Site
Chicago, Illinois, United States
Clinical Trial Site
Ann Arbor, Michigan, United States
Clinical Trial Site
Vienna, , Austria
Clinical Trial Site
Heidelberg, , Germany
Clinical Trial Site
Warsaw, , Poland
Clinical Trial Site
Guildford, Surrey, United Kingdom
Clinical Trial Site
Birmingham, , United Kingdom
Countries
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References
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Weiss KH, Askari FK, Czlonkowska A, Ferenci P, Bronstein JM, Bega D, Ala A, Nicholl D, Flint S, Olsson L, Plitz T, Bjartmar C, Schilsky ML. Bis-choline tetrathiomolybdate in patients with Wilson's disease: an open-label, multicentre, phase 2 study. Lancet Gastroenterol Hepatol. 2017 Dec;2(12):869-876. doi: 10.1016/S2468-1253(17)30293-5. Epub 2017 Oct 5.
Other Identifiers
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WTX101-201
Identifier Type: -
Identifier Source: org_study_id
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