EUCTR2017-004135-36-PT进行中（未招募）1 期
A Phase 3, Randomized, Rater-Blinded, Multi-Center Study To Evaluate The Efficacy And Safety Of ALXN1840 Administered For 48 Weeks Versus Standard Of Care In Patients With Wilson Disease Aged 12 Years And Older With An Extension Period Of Up To 60 Months
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 180
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •- 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 and the patient must be willing to give written informed assent (if applicable as determined by the central or local Institutional Review Board [IRB]/ Institutional (or Independent) Ethics Committee [IEC]). If allowable per local regulations, a patients’ Legally Acceptable Representative may provide informed consent if a patient is unable to do so.
- •-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
排除标准
- •- Decompensated hepatic cirrhosis;
- •- MELD score greater than 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
研究者
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