Efficacy and Safety of ALXN1840 Administered for 48 Weeks Versus Standard of Care in Participants With Wilson Disease
Study Identifier:
WTX101-301
ClinicalTrials.gov Identifier:
EudraCT Identifier:
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other
Available Documents
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: ALXN1840
- Drug: SoC Therapy
Date
Feb 2018 - Jun 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 12+ years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- 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
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
- Participants with end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance \< 30 milliliter/minute
Sex
Female & Male
Age
12+ years
Study Details
Medical Condition
- Unmapped
Study Drug
- Drug: ALXN1840
- Drug: SoC Therapy
Date
Feb 2018 - Jun 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: 12+ years years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
- 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
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
- Participants with end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance \< 30 milliliter/minute
Protocol 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 in the Primary Evaluation Period. In addition, efficacy and safety will be evaluated during an optional 60-month Extension Period.
Trial Locations
Location
Status
Location
Research Site
Los Angeles, California, United States, 90095
Status
N/A
Location
Research Site
New Haven, Connecticut, United States, 06510
Status
N/A
Location
Research Site
Chicago, Illinois, United States, 60611
Status
N/A
Location
Research Site
Ann Arbor, Michigan, United States, 48109
Status
N/A
Location
Research Site
Houston, Texas, United States, 77030
Status
N/A
Location
Research Site
Seattle, Washington, United States, 98105
Status
N/A
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