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Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

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ClinicalTrials.gov Identifier: NCT04573309
Recruitment Status : Recruiting
First Posted : October 5, 2020
Last Update Posted : May 18, 2022
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

Brief Summary:
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).

Condition or disease Intervention/treatment Phase
Wilson Disease Drug: ALXN1840 Phase 2

Detailed Description:
Participants who are treatment experienced (which includes standard-of-care therapies or ALXN1840) and treatment naïve are eligible for this study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Actual Study Start Date : September 7, 2020
Estimated Primary Completion Date : May 31, 2022
Estimated Study Completion Date : May 31, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Wilson Disease
Drug Information available for: Molybdenum

Arm Intervention/treatment
Experimental: ALXN1840
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
Drug: ALXN1840
Administered orally as tablets.
Other Name: formerly WTX101




Primary Outcome Measures :
  1. Mean Daily Copper Balance [ Time Frame: Accumulation: Day 1 through Day 8 (ALXN1840 15 mg) ]
    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.

  2. Mean Daily Copper Balance [ Time Frame: Accumulation: Day 31 through Day 35 (ALXN1840 30 mg) ]
    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.

  3. Mean Daily Copper Balance [ Time Frame: Steady state: Day 25 through Day 28 (ALXN1840 15 mg) ]
    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.

  4. Mean Daily Copper Balance [ Time Frame: Steady state: Day 36 through Day 39 (ALXN1840 30 mg) ]
    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 :
  1. Change From Baseline In Mean Daily Copper Balance [ 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) ]
    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.

  2. Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound copper (LBC) [ 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 ]
    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.

  3. Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State [ Time Frame: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) ]
    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.

  4. 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) ]
  5. Mean Daily Molybdenum Balance At Steady State [ Time Frame: Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg) ]
    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).

  6. Accumulation Of Molybdenum As Determined By Molybdenum Balance [ Time Frame: Accumulation: Day 1 through Day 8 for 15 mg and Day 31 through Day 35 for 30 mg ]
    Molybdenum balance will be assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Diagnosis of WD by Leipzig Criteria ≥ 4.
  2. Able to reside in the clinical research unit for intensive metabolic monitoring of copper and molybdenum.
  3. Participants willing to adhere to copper/molybdenum-controlled diet during the study.
  4. Willing and able to follow protocol-specified contraception requirements.
  5. Capable of giving signed informed consent.

Exclusion Criteria:

  1. Decompensated cirrhosis or model for end stage liver disease score > 13.
  2. Modified Nazer score > 7.
  3. Clinically significant gastrointestinal bleed within past 3 months.
  4. Alanine aminotransferase > 2 × upper limit of normal.
  5. Hemoglobin less than lower limit of the reference range for age and sex.
  6. Significant medical history (current or past).
  7. Previous treatment with zinc within 30 days prior to the Screening Visit.
  8. Participants in renal failure, defined as in end-stage renal disease on dialysis (chronic kidney disease stage 5) or creatinine clearance < 30 milliliters/minute.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04573309


Contacts
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Contact: Alexion Pharmaceuticals Inc. 855-752-2356 clinicaltrials@alexion.com

Locations
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United States, Connecticut
Clinical Study Site Recruiting
New Haven, Connecticut, United States, 06520
United States, Nebraska
Clinical Study Site Recruiting
Lincoln, Nebraska, United States, 68502
New Zealand
Clinical Study Site Recruiting
Grafton, Auckland, New Zealand, 1010
United Kingdom
Clinical Study Site Recruiting
London, United Kingdom, London SE1 1YR
Sponsors and Collaborators
Alexion Pharmaceuticals
Investigators
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Study Director: Eugene S. Swenson, MD, PhD Alexion Pharmaceuticals
Study Director: Peter Ksenuk, MD Alexion Pharmaceuticals
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04573309    
Other Study ID Numbers: ALXN1840-WD-204
2020-001104-41 ( EudraCT Number )
First Posted: October 5, 2020    Key Record Dates
Last Update Posted: May 18, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Copper Balance
Molybdenum Balance
Wilson Disease
ALXN1840
Additional relevant MeSH terms:
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Hepatolenticular Degeneration
Liver Diseases
Digestive System Diseases
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metal Metabolism, Inborn Errors
Metabolic Diseases