Phase 2
N=9
Copper and Molybdenum Balance in Participants With Wilson Disease Treated With ALXN1840
Wilson Disease
Bottom Line
View on ClinicalTrials.gov: NCT04573309 ↗Enrolled (actual)
9
Serious AEs
0.0%
Results posted
Oct 2023
Primary outcome: Primary: Mean Daily Copper Balance: Day 1 Through Day 8 — 0.8025; 0.3062 milligrams/day
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ALXN1840 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Alexion Pharmaceuticals, Inc.
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Mean Daily Copper Balance: Day 1 Through Day 8 |
0.8025; 0.3062 | — |
| PRIMARY Mean Daily Copper Balance: Day 31 Through Day 35 |
0.9156; 0.6613 | — |
| PRIMARY Mean Daily Copper Balance: Day 25 Through Day 28 |
0.7109; 0.7072 | — |
| PRIMARY Mean Daily Copper Balance: Day 36 Through Day 39 |
0.9975; 0.5662 | — |
| SECONDARY Change From Baseline In Mean Daily Copper Balance |
-0.3780; -0.2060; -0.4697; 0.1950; -0.2650; 0.1492 | — |
| SECONDARY Copper Quantified In Food, Drink, Feces, And Urine, Including Plasma Total And Labile Bound Copper (LBC) |
1.6423; 1.3523; 1.8269; 1.5637; 1.8938; 1.6022 | — |
| SECONDARY Molybdenum Specified In ALXN1840 Doses Given And Quantified In Food, Drink, Feces, And Urine, Including Plasma At Steady State |
0.3324; 0.3905; 0.1923; 0.1724; 0.0006; 0.0018 | — |
| SECONDARY Change From Baseline In Total Molybdenum Excretion In Urine And Feces |
0.7102; 0.9510; 1.0146; 0.9736; 1.0484; 1.7188 | — |
| SECONDARY Mean Daily Molybdenum Balance At ALXN1840 Steady State |
1.3618; 2.1087; 2.7599; 3.6581 | — |
| SECONDARY Accumulation Of Molybdenum As Determined By Molybdenum Balance |
2.2458; 2.0876; 2.6265; 4.0775 | — |
| SECONDARY Maximum Observed Plasma Concentration (Cmax) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum |
430.451; 1271.551; 13.546; 17.823 | — |
| SECONDARY Area Under The Concentration Time Curve (AUC0-inf) of Total Molybdenum and Plasma Ultrafiltrate (PUF) Molybdenum |
13532.591; 65694.679; 292.737; 476.960 | — |
Summary
This exploratory study will investigate the effects of ALXN1840 on copper balance in participants with Wilson disease (WD).
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.
Data sourced from ClinicalTrials.gov (NCT04573309). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.