Phase 2
N=29
Efficacy and Safety Study of WTX101 (ALXN1840) in Adult Wilson Disease Patients
Wilson Disease
Bottom Line
View on ClinicalTrials.gov: NCT02273596 ↗Enrolled (actual)
29
Serious AEs
39.3%
Results posted
Sep 2021
Primary outcome: Primary: Percentage Of Participants With Normalized Concentrations Of NCC — 85.7 percentage of participants
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
- Oct 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Of Participants With Normalized Concentrations Of NCC |
85.7 | — |
| SECONDARY Change From Baseline In NCC Concentrations Adjusted For Mo Plasma Concentration At Week 24 |
-2.56 | — |
| SECONDARY Time To Normalization Of NCC Adjusted For Mo Plasma Concentration In Participants With Elevated Baseline NCC |
147.5 | — |
| SECONDARY Change From Baseline In Neurological Status Using The Unified Wilson's Disease Rating Scale (UWDRS) (Neurological Subscore; Part I) At Week 24 |
0.0 | — |
| SECONDARY Change From Baseline In Neurological Status Using The UWDRS (Neurological Subscore; Parts II, III, And Total Score) At Week 24 |
-2.5; -5.75; -8.23 | — |
| SECONDARY Change From Baseline In Psychiatric Status Dimension Using Mini International Neuropsychiatric Interview (M.I.N.I.) Tracking Standardized Scores At Week 24 |
-0.39; -0.07; -0.09; -0.28; -0.58; -0.35 | — |
| SECONDARY Clinical Global Impression-Improvement Scale (CGI-I) At Week 24 |
-0.8 | — |
| SECONDARY Change From Baseline In Clinical Global Impression Severity Scale (CGI-S) At Week 24 |
-0.9 | — |
| SECONDARY Change From Baseline In Quality Of Life (QoL)/Patient Reported Outcome (PRO) Assessed By The European Quality Of Life 5 Dimensions (EQ-5D) Visual Analogue Scale (VAS) At Week 24 |
11.3 | — |
| SECONDARY Change From Baseline In Quality Of Life (QoL)/Patient Reported Outcome (PRO) Assessed By The EQ-5D Descriptive System UK Health Index Scores At Week 24 |
0.0439 | — |
| SECONDARY QoL/PRO Assessed By The 8-Item Medication Adherence Scale (MMAS-8) At Week 24 |
7.59 | — |
| SECONDARY QoL/PRO Assessed By The Treatment Satisfaction Questionnaire For Medication (TSQM-9) At Week 24 |
69.81; 83.57; 75.47 | — |
| SECONDARY Change From Baseline In Hepatic Laboratory Measure Alanine Aminotransferase (ALT) At Week 24 |
-2.0 | — |
| SECONDARY Change From Baseline In Hepatic Laboratory Measure Aspartate Aminotransferase (AST) At Week 24 |
-10.3 | — |
| SECONDARY Change From Baseline In Hepatic Laboratory Measure International Normalized Ratio (INR) At Week 24 |
-0.05 | — |
| SECONDARY Change From Baseline In Hepatic Laboratory Measure Bilirubin At Week 24 |
-0.7 | — |
| SECONDARY Change From Baseline In Exchangeable Cu At Week 24 |
-25.1 | — |
| SECONDARY Change From Baseline In Speciation Profiling (Mo, Cu, And Protein Complex Profiling Using Size Exclusion Chromatography) At Week 24 |
— | — |
| SECONDARY Change From Baseline In 24-Hour Urinary Mo And Cu At Week 24 |
1561.1; -28.1 | — |
| SECONDARY Pharmacokinetics (PK): Area Under The Curve From Time 0 to 24 (AUC0-24) Of Plasma Total Mo |
2872.6; 4796.7; 3138.7; 7873.6; 8836.9; 11342.5 | — |
| SECONDARY PK: Maximum Concentration (Cmax) Of Plasma Total Mo |
202.7; 304.8; 167.6; 393.0; 488.8; 607.3 | — |
| SECONDARY Extension Period: Percentage Of Participants With Normalized Concentrations Of NCC |
92.9 | — |
| SECONDARY Extension Period: Change From Baseline In NCC Levels Adjusted For Mo Plasma Concentration |
-2.92 | — |
Summary
The main purpose of the study was to evaluate the efficacy of ALXN1840 (formerly WTX101) for 24 weeks on non-ceruloplasmin-bound copper (NCC) concentrations adjusted for molybdenum plasma concentration in participants newly diagnosed with Wilson Disease (WD) who were aged 18 and older and who had NCC concentrations within or above the reference range at the time of enrollment in the study. The study consisted of a 24-week Treatment Period, followed by a planned 36-month Extension Period.
Eligibility Criteria
Inclusion Criteria
- Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator.
- Newly established diagnosis of WD by Leipzig-Score ≥ 4 documented by testing as outlined in 2012 European Association for the Study of the Liver Wilson Disease Clinical Practice Guidelines.
- NCC levels within or above the normal reference range (0.8 to 2.3 micromole).
- Willing to undergo 48 hour washout from current WD treatment
Exclusion Criteria
- Treatment for greater than 24 months for WD with chelation therapy (for example, penicillamine, trientine hydrochloride) or zinc therapy.
- Decompensated hepatic cirrhosis.
- Model for End-Stage Liver Disease score > 11.
- Modified Nazer score > 6.
- Gastrointestinal bleed within past 6 months.
- Alanine aminotransferase > 5 x upper limit of normal.
- Marked neurological disease requiring either nasogastric feeding or intensive in-patient medical care.
- Severe anemia with a hemoglobin < 9 grams/deciliter.
Data sourced from ClinicalTrials.gov (NCT02273596). 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.