Clinical Trials List
Protocol NumberWTX101-301
NCT Number(ClinicalTrials.gov Identfier)NCT03403205
2019-07-01 - 2023-04-25
Phase III
Recruiting1
A PHASE 3, RANDOMIZED, RATER-BLINDED, MULTI-CENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF ALXN1840 ADMINISTERED FOR 48 WEEKS VERSUS STANDARD OF CARE IN PATIENTS WITH WILSON DISEASE AGED 12 YEARS AND OLDER WITH AN EXTENSION PERIOD OF UP TO 60 MONTHS
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Trial Applicant
Syneos Health
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Sponsor
Alexion Pharmaceuticals, Inc.
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Linkou Chang Gung Medical Foundation
Taiwan National PI
黃錦章
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
WILSON DISEASE
Objectives
The study will evaluate the efficacy and safety of ALXN1840 (formerly called WTX101) administered for 48 weeks compared to standard of care (SoC) in Wilson Disease (WD) participants aged 12 and older in the Primary Evaluation Period. In addition, efficacy and safety will be evaluated during an optional 60-month Extension Period.
Test Drug
ALXN1840
Active Ingredient
ALXN1840
Dosage Form
tablet
Dosage
15
Endpoints
Daily Mean Area Under The Effect-time Curve (AUEC) Of Directly Measured Non-ceruloplasmin-bound Copper (dNCC) [ Time Frame: Baseline to 48 weeks ]
Inclution Criteria
Criteria
Key Inclusion Criteria:
Established diagnosis of WD by Leipzig-Score ≥ than 4
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
Key Inclusion Criteria:
Established diagnosis of WD by Leipzig-Score ≥ than 4
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
Key Exclusion Criteria:
Decompensated hepatic cirrhosis
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
Decompensated hepatic cirrhosis
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
The Estimated Number of Participants
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Taiwan
0 participants
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Global
180 participants
