Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
NCT ID: NCT03403205
Last Updated: 2024-06-17
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE3
214 participants
INTERVENTIONAL
2018-02-22
2023-06-30
Brief Summary
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Detailed Description
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All enrolled participants were randomized by cohort in a 2:1 ratio to treatment with ALXN1840 or SoC (either as continued therapy in Cohort 1 or as continued or initial therapy in Cohort 2).
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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ALXN1840
ALXN1840 was administered orally for 48 weeks at doses ranging from 15 milligrams (mg) every other day (QOD) up to a titrated dose of 60 mg daily.
Participants who completed the Primary Evaluation Period had the option to participate in the up to 60-month Extension Period.
ALXN1840
ALXN1840 administered orally in 15 mg tablets
Standard of Care (SoC) Medication
SoC medication was administered for 48 weeks. Participants who completed the Primary Evaluation Period had the option to participate in the up to 60-month Extension Period.
SoC Therapy
Depending on the site/region, participants randomized to receive SoC treatment will receive trientine, penicillamine, Zinc, or a combination of these medicines, administered according to standard regimens.
Interventions
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ALXN1840
ALXN1840 administered orally in 15 mg tablets
SoC Therapy
Depending on the site/region, participants randomized to receive SoC treatment will receive trientine, penicillamine, Zinc, or a combination of these medicines, administered according to standard regimens.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Female participants of childbearing potential, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception starting at least 6 weeks before the Day 1 visit and continuing through 28 days after the last dose of either ALXN1840 or SoC
* Male participants, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception beginning at Day 1 visit and continuing through 90 days after last dose of either ALXN1840 or SoC
Exclusion Criteria
* MELD score \> 13
* Modified Nazer score \> 7
* Clinically significant gastrointestinal bleed within past 3 months
* Alanine aminotransferase \> 2 X upper limit of normal (ULN) for participants treated for \> 28 days with WD therapy (Cohort 1)
* Alanine aminotransferase \> 5 X ULN for treatment-naïve participants or participants who have been treated for ≤ 28 days (Cohort 2)
* Marked neurological disease requiring either nasogastric feeding or intensive inpatient medical care
* Hemoglobin \< 9 grams/deciliter
* History of seizure activity within 6 months prior to informed consent
* Pregnant (or women who are planning to become pregnant) or breastfeeding women
* Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus or seropositivity for human immunodeficiency virus (HIV)
* Previous treatment with tetrathiomolybdate
* Participants with end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance \< 30 milliliter/minute
12 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 S. Swenson, M.D., Ph.D.
Role: STUDY_DIRECTOR
Alexion Pharmaceuticals, Inc.
Locations
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Research Site
Los Angeles, California, United States
Research Site
New Haven, Connecticut, United States
Research Site
Chicago, Illinois, United States
Research Site
Ann Arbor, Michigan, United States
Research Site
Houston, Texas, United States
Research Site
Seattle, Washington, United States
Research Site
Adelaide, , Australia
Research Site
Concord, , Australia
Research Site
Parkville, , Australia
Research Site
Parkville, , Australia
Research Site
Graz, , Austria
Research Site
Innsbruck, , Austria
Research Site
Vienna, , Austria
Research Site
Toronto, Ontario, Canada
Research Site
Prague, , Czechia
Research Site
Århus N, , Denmark
Research Site
Bron, , France
Research Site
Paris, , France
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Hamburg, , Germany
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Heidelberg, , Germany
Research Site
Leipzig, , Germany
Research Site
Hong Kong, , Hong Kong
Research Site
Budapest, , Hungary
Research Site
Jerusalem, , Israel
Research Site
Ramat Gan, , Israel
Research Site
Chiba, , Japan
Research Site
Kumamoto, , Japan
Research Site
Kurume-shi, , Japan
Research Site
Matsuyama, , Japan
Research Site
Meguro-ku, , Japan
Research Site
Sapporo, , Japan
Research Site
Takatsuki-shi, , Japan
Research Site
Yokohama, , Japan
Research Site
Grafton, , New Zealand
Research Site
Warsaw, , Poland
Research Site
Warsaw, , Poland
Research Site
Moscow, , Russia
Research Site
Moscow, , Russia
Research Site
Moscow, , Russia
Research Site
Nizhny Novgorod, , Russia
Research Site
Saint Petersburg, , Russia
Research Site
Belgrade, , Serbia
Research Site
Singapore, , Singapore
Research Site
Daegu, , South Korea
Research Site
Barcelona, , Spain
Research Site
Málaga, , Spain
Research Site
Sabadell, , Spain
Research Site
Taipei, , Taiwan
Research Site
Taoyuan, , Taiwan
Research Site
Ankara, , Turkey (Türkiye)
Research Site
Istanbul, , Turkey (Türkiye)
Research Site
Istanbul, , Turkey (Türkiye)
Research Site
Izmir, , Turkey (Türkiye)
Research Site
Edgbaston, , United Kingdom
Research Site
Guildford, , United Kingdom
Research Site
London, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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Other Identifiers
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2017-004135-36
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
WTX101-301
Identifier Type: -
Identifier Source: org_study_id
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