EUCTR2017-004135-36-AT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 (18 years and older in Germany);
- •- Willing and able to give written informed consent and comply with the study visit schedule. For patients < 18 years of age (or below the age of adulthood under Japanese local law for patients in Japan), 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 copper, zinc, or molybdenum throughout the study duration;
- •- Willing to avoid intake of foods and drinks with high contents of copper 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
排除标准
- •- 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 > 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 (chronic kidney disease stage 5 [CKD 5)]) or creatinine clearance < 30 mL/min
- •Please see study protocol for more details
研究者
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