Status:
TERMINATED
Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
Lead Sponsor:
Alexion Pharmaceuticals, Inc.
Conditions:
Wilson Disease
Eligibility:
All Genders
12+ years
Phase:
PHASE3
Brief Summary
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 ...
Detailed Description
The study consists of 2 cohorts. Cohort 1: Participants who have received SoC therapy for \> 28 days and Cohort 2: Participants who are treatment-naïve or who have received SoC therapy for ≤ 28 days. ...
Eligibility Criteria
Inclusion
- Key
- Established diagnosis of WD by Leipzig-Score ≥ 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
Exclusion
- 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
- Participants with end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance \< 30 milliliter/minute
Key Trial Info
Start Date :
February 22 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2023
Estimated Enrollment :
214 Patients enrolled
Trial Details
Trial ID
NCT03403205
Start Date
February 22 2018
End Date
June 30 2023
Last Update
June 17 2024
Active Locations (56)
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1
Research Site
Los Angeles, California, United States, 90095
2
Research Site
New Haven, Connecticut, United States, 06510
3
Research Site
Chicago, Illinois, United States, 60611
4
Research Site
Ann Arbor, Michigan, United States, 48109