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

NCT04573309 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: ALXN1840-WD-2042020-001104-41
• Study Type: interventional
• Target: 9
• Locations: 3 countries
• Sites: 3

Brief Summary

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

Conditions

Wilson Disease

Keywords

Copper Balance; Molybdenum Balance; Wilson Disease; ALXN1840

Outcome Measures

Primary Outcomes (4)

• Mean Daily Copper Balance: Day 1 Through Day 8

  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.

  Accumulation: Day 1 through Day 8 (ALXN1840 15 mg)

• Mean Daily Copper Balance: Day 31 Through Day 35

  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.

  Accumulation: Day 31 through Day 35 (ALXN1840 30 mg)

• Mean Daily Copper Balance: Day 25 Through Day 28

  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.

  Accumulation: Day 25 through Day 28 (ALXN1840 15 mg)

• Mean Daily Copper Balance: Day 36 Through Day 39

  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.

  Accumulation: Day 36 through Day 39 (ALXN1840 30 mg)

Secondary Outcomes (8)

• Change From Baseline In Mean Daily Copper Balance

  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 Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC)

  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

• Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State

  Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)

• Change From Baseline In Total Molybdenum Excretion In Urine And Feces

  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)

• Mean Daily Molybdenum Balance At ALXN1840 Steady State

  Steady state: Day 25 through Day 28 (ALXN1840 15 mg) and Day 36 through Day 39 (ALXN1840 30 mg)

Study Arms (1)

ALXN1840

EXPERIMENTAL

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

Interventions

ALXN1840 DRUG

Administered orally as tablets.

Also known as: formerly WTX101

ALXN1840

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (3)

Research Site

New Haven, Connecticut, 06510, United States

Location

Research Site

Grafton, 1010, New Zealand

Location

Research Site

London, SE1 1YR, United Kingdom

Location

MeSH Terms

Conditions

Hepatolenticular Degeneration

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc.
• Organization: Alexion Pharmaceuticals, Inc.

clinicaltrials@alexion.com

855-752-2356

Study Officials

• Eugene S. Swenson, MD, PhD

  Alexion Pharmaceuticals, Inc.

  STUDY DIRECTOR

• Peter Ksenuk, MD

  Alexion Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 28, 2020
• First Posted: October 5, 2020
• Study Start: September 7, 2020
• Primary Completion: June 7, 2022
• Study Completion: June 7, 2022
• Last Updated: June 24, 2024
• Results First Posted: October 27, 2023

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); NZ (1); GB (1)