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Completed

NCT Number: NCT04573309

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

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

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Key information

Conditions

Wilson Disease Basal Ganglia Diseases Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Hepatolenticular Degeneration Heredodegenerative Disorders, Nervous System Liver Diseases Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Movement Disorders Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Research Site, Grafton, New Zealand

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ALXN1840

Drug

Administered orally as tablets.

Other names: formerly WTX101

Primary outcomes

  1. Mean Daily Copper Balance: Day 1 Through Day 8

    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 was calculated as the average daily copper balance over the collection period.

  2. Mean Daily Copper Balance: Day 31 Through Day 35

    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 was calculated as the average daily copper balance over the collection period.

  3. Mean Daily Copper Balance: Day 25 Through Day 28

    Time frame: Accumulation: 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 was calculated as the average daily copper balance over the collection period.

  4. Mean Daily Copper Balance: Day 36 Through Day 39

    Time frame: Accumulation: 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 was calculated as the average daily copper balance over the collection period.

Secondary outcomes

  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 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 was 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 was 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)

    The amount of molybdenum in food, drink, feces and urine is reported in this outcome measure.

  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 ALXN1840 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 was 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 (ALXN1840 15 mg) and Day 31 through Day 35 ((ALXN1840 30 mg)

    Molybdenum balance was assessed through measurement of molybdenum intake (in food, drink, and ALXN1840), and molybdenum output (in feces and urine).

  7. Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum

    Time frame: Day 1 up to Day 39

  8. Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum

    Time frame: Day 39

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

A Phase 2, Open-label Study to Assess Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840

Important dates

Study start
2020
Primary completion
2022
Study completion
2022
First posted
Oct 5, 2020
Registry last updated
Jun 24, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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