A Phase 3 clinical study to Evaluate the Efficacy and Safety of ALXN1840 in adult and adolescent patients who suffer from Wilson Disease.

Phase 1

• Conditions: Wilson Disease; MedDRA version: 20.0Level: LLTClassification code 10047988Term: Wilson's diseaseSystem Organ Class: 100000004850; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2017-004135-36-PL
• Lead Sponsor: Alexion Pharmaceuticals, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-04-03
• Last Update Posted: 9 October 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 180

Inclusion Criteria

- Established diagnosis of WD by Leipzig-Score > 4 documented by testing as outlined in the 2012 European Association for the Study of Liver WD Clinical Practice Guidelines;
-12 years of age or older at time of informed consent/assent;
- Willing and able to give written informed consent and comply with the study visit schedule. For patients < 18 years of age patient`s legal guardian must be willing and able to give written informed consent.
-Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator.;
-Willing to withhold treatment with SoC for > 48 hours immediately prior to first study assessment on Day 1;
-Adequate venous access to allow collection of required blood samples;
- Willing to avoid use of vitamins and/or minerals containing Cu, Zn, or molybdenum (Mo) throughout the study duration;
-Willing to avoid intake of foods and drinks with high contents of Cu throughout the study duration;
-Female patients of childbearing potential must be willing to follow guidance for highly effective contraception;

please see the study protocol for details
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 160
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

- Decompensated hepatic cirrhosis;
- MELD score> 13;
- Participation in a clinical study of an experimental or unapproved/unlicensed therapy at the same time or within the 4 weeks prior to this Screening Visit;
- Pregnant (or women who are planning to become pregnant) or lactating women;
- Major systemic disease or other illness that would, in the opinion of the Investigator, compromise subject safety or interfere with the collection or interpretation of study results;
- Modified Nazer score >7
- Hemoglobine < 9 g/dL
- Alanine aminotransferase >2 × ULN for subjects treated for >28 days with WD therapy (Cohort 1);
- Alanine aminotransferase Alanine aminotransferase > 5 × ULN for treatment naïve subjects or subjects who have been treated for = 28 days (Cohort 2);
- Patients with end-stage renal disease on dialysis (CKD 5) or creatinine clearance < 30 mL/min
please see study protocol for more details

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified