A Phase 3, Randomised, Rater-Blinded, Multi-Centre Study to Evaluate the Efficacy and Safety of WTX101 Administered for 48 Weeks Versus Standard of Care in Wilson Disease Subjects Aged 18 and Older with an Extension Phase of up to 60 Months

Wilson Therapeutics AB0 个研究点目标入组 102 人开始时间: 2018年1月15日最近更新: 2019年6月30日

研究概览

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•- Willing and able to give informed consent for participation in the study;
•- Male or female subjects, aged 18 years or older as of signing the informed consent form (ICF);
•- Able to understand and willing to comply with study procedures, restrictions, and requirements, as judged by the Investigator;
•- 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;
•- Treatment for >28 days for WD with chelation therapy (ie, penicillamine, trientine hydrochloride), Zn therapy, or a combination of a chelator and Zn; OR Treatment naïve or treatment for = 28 days for WD with chelation therapy (ie, penicillamine, trientine hydrochloride), Zn therapy, or a combination of a chelator and Zn;
•- Willing to undergo = 48-hour washout from current WD treatment;
•- Willing to avoid use of vitamins and/or minerals containing Cu, Zn, or Mo throughout the study duration
•- Willing to avoid intake of foods and drinks with high contents of Cu throughout the study duration;
•please see the study protocol for details
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 102
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 3

•- 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;
•- Severe anaemia with a haemoglobin 9 g/dL;
•- 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
•- 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);
•please see study protocol for more details

发起方