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-DK
• Lead Sponsor: Alexion Pharmaceuticals, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-08-05
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• 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, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception starting at least 6 weeks before the Day 1 visit and continuing through 28 days after the last dose of study drug (either ALXN1840 or SoC). Male patients, if heterosexually active, must be willing to follow protocol-specified guidance for highly effective contraception beginning at the Day 1 visit and continuing through 90 days after the last dose of study drug (either ALXN1840 or SoC).
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