Back to resultsCopper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Primary Purpose
Wilson Disease
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
ALXN1840
Sponsored by
About this trial
This is an interventional treatment trial for Wilson Disease focused on measuring Copper Balance, Molybdenum Balance, Wilson Disease, ALXN1840
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of WD by Leipzig Criteria ≥ 4.
- Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
- Participants willing to adhere to copper/molybdenum-controlled diet during the study.
- Willing and able to follow protocol-specified contraception requirements.
- Capable of giving signed informed consent.
Exclusion Criteria:
- Decompensated cirrhosis or model for end stage liver disease score > 13.
- Modified Nazer score > 7.
- Clinically significant gastrointestinal bleed within past 3 months.
- Alanine aminotransferase > 2 × upper limit of normal.
- Hemoglobin less than lower limit of the reference range for age and sex.
- Significant medical history (current or past).
- Previous treatment with zinc within 30 days prior to the Screening Visit.
- Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.
Sites / Locations
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
- Clinical Study Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
ALXN1840
Arm Description
Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39
Outcomes
Primary Outcome Measures
Mean Daily Copper Balance
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Mean Daily Copper Balance
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Secondary Outcome Measures
Change From Baseline In Mean Daily Copper Balance
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound copper (LBC)
Copper will be assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper.
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
Molybdenum balance is defined as the difference between the measured molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine), and will be calculated as the average daily molybdenum balance over the collection period.
Change From Baseline In Total Molybdenum Excretion In Urine And Feces
Mean Daily Molybdenum Balance At Steady State
Molybdenum balance at steady state will be assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum(out) equal to molybdenum(in).
Accumulation Of Molybdenum As Determined By Molybdenum Balance
Molybdenum balance will be assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04573309
Brief Title
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Official Title
A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Study Type
Interventional
2. Study Status
Record Verification Date
November 2022
Overall Recruitment Status
Completed
Study Start Date
September 7, 2020 (Actual)
Primary Completion Date
August 1, 2022 (Actual)
Study Completion Date
September 1, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
Detailed Description
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Wilson Disease
Keywords
Copper Balance, Molybdenum Balance, Wilson Disease, ALXN1840
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ALXN1840
Arm Type
Experimental
Arm Description
Participants will be administered ALXN1840 at a dose of 15 milligrams (mg)/day on Day 1 through Day 28 and then increased to 30 mg/day on Day 29 through Day 39
Intervention Type
Drug
Intervention Name(s)
ALXN1840
Other Intervention Name(s)
formerly WTX101
Intervention Description
Administered orally as tablets.
Primary Outcome Measure Information:
Title
Mean Daily Copper Balance
Description
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Time Frame
Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)
Title
Mean Daily Copper Balance
Description
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Time Frame
Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)
Title
Mean Daily Copper Balance
Description
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Time Frame
Steady state: Day 25 through Day 28 (ALXN1840 15 mg)
Title
Mean Daily Copper Balance
Description
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Time Frame
Steady state: Day 36 through Day 39 (ALXN1840 30 mg)
Secondary Outcome Measure Information:
Title
Change From Baseline In Mean Daily Copper Balance
Description
Copper balance is defined as the difference between the measured copper input in food and drink, and the measured copper elimination in urine and feces, and will be calculated as the average daily copper balance over the collection period.
Time Frame
Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 31 through Day 39 (ALXN1840 30 mg)
Title
Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound copper (LBC)
Description
Copper will be assessed through measurement of copper intake (in food and drink), and copper output (in feces and urine) as well as plasma total and labile bound copper.
Time Frame
Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg; Steady state: Day 25 through Day 28 for ALXN1840 15 mg and Day 36 through Day 39 for ALXN1840 30 mg
Title
Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State
Description
Molybdenum balance is defined as the difference between the measured molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine), and will be calculated as the average daily molybdenum balance over the collection period.
Time Frame
Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Title
Change From Baseline In Total Molybdenum Excretion In Urine And Feces
Time Frame
Accumulation: Baseline, Day 1 through Day 8 (ALXN1840 15 mg) and Day 31 through Day 35 (ALXN1840 30 mg); Steady State: Baseline, Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Title
Mean Daily Molybdenum Balance At Steady State
Description
Molybdenum balance at steady state will be assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine). Steady state is defined as molybdenum(out) equal to molybdenum(in).
Time Frame
Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)
Title
Accumulation Of Molybdenum As Determined By Molybdenum Balance
Description
Molybdenum balance will be assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).
Time Frame
Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of WD by Leipzig Criteria ≥ 4.
Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
Participants willing to adhere to copper/molybdenum-controlled diet during the study.
Willing and able to follow protocol-specified contraception requirements.
Capable of giving signed informed consent.
Exclusion Criteria:
Decompensated cirrhosis or model for end stage liver disease score > 13.
Modified Nazer score > 7.
Clinically significant gastrointestinal bleed within past 3 months.
Alanine aminotransferase > 2 × upper limit of normal.
Hemoglobin less than lower limit of the reference range for age and sex.
Significant medical history (current or past).
Previous treatment with zinc within 30 days prior to the Screening Visit.
Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Eugene S. Swenson, MD, PhD
Organizational Affiliation
Alexion
Official's Role
Study Director
First Name & Middle Initial & Last Name & Degree
Peter Ksenuk, MD
Organizational Affiliation
Alexion
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Study Site
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
Facility Name
Clinical Study Site
City
Lincoln
State/Province
Nebraska
ZIP/Postal Code
68502
Country
United States
Facility Name
Clinical Study Site
City
Grafton
State/Province
Auckland
ZIP/Postal Code
1010
Country
New Zealand
Facility Name
Clinical Study Site
City
London
ZIP/Postal Code
London SE1 1YR
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
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Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
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