EUCTR2014-001703-41-GB进行中（未招募）1 期
A Phase 2, Multi-centre, Open-label, Study to Evaluate the Efficacy and Safety of WTX101 Administered for 24 Weeks in Newly Diagnosed Wilson Disease Patients Aged 18 and Older with an Extension Phase of 12 Months - Wilson Disease Patients to evaluate efficacy and safety of WTX101
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 30
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
入选标准
- •Patients must meet all of the following criteria to be eligible for enrolment in this study:
- •1. Willing and able to give informed consent for participation in the study.
- •2. Male or female patients, aged 18 years or older as of signing the ICF.
- •3. Able to understand and willing to comply with study procedures, restrictions and requirements, as judged by the Investigator.
- •4. Newly established diagnosis of Wilson Disease by Leipzig-Score ? 4 (Ferenci et al 2003) documented by testing as outlined in 2012 EASL WD Clinical Practice
- •Guidelines.
- •5. NCC levels above the normal reference range (0.8-2.3 µM).
- •6. Willing to undergo 48 hour washout from current Wilson Disease treatment.
- •7. Adequate venous access to allow collection of a number of blood samples.
- •8. Willing to avoid intake of foods and water with high concentrations of copper
- •throughout the duration of the study.
- •9. Females of childbearing potential will be included if they are either sexually inactive (abstinent) for 14 days prior to the first WTX101 dose continuing through 28 days after the last WTX101 dose, or using one of the following highly effective birth control methods:
- •a. intrauterine device (IUD) (without Cu);
- •b. surgical sterilization of the partner (vasectomy for 6 months minimum);
- •c. combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
- •d. progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
- •e. intrauterine hormone releasing system (IUS);
- •f. bilateral tubal occlusion.
- •Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. In this trial abstinence is only acceptable if in line with the subjects preferred and usual lifestyle.
- •Periodic abstinence (calendar, symptothermal, post-ovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method (LAM) are not acceptable methods of contraception. As well, female condom and male condom should not be used together.
- •10. Females of childbearing potential agree to remain sexually inactive or to keep the same birth control method for at least 28 days following the last dose.
- •11. A female of non-childbearing potential must have undergone one of the following sterilization procedures at least 6 months prior to the first WTX101 dose:
- •a. hysteroscopic sterilization;
- •b. bilateral tubal ligation or bilateral salpingectomy;
- •c. hysterectomy;
- •d. bilateral oophorectomy;
- •or be postmenopausal with amenorrhea for at least 1 year prior to the first WTX101 dose and follicle stimulating hormone (FSH) serum levels consistent with
- •postmenopausal status.
- •12. A non-vasectomized male subject agrees to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study medication and the female part
排除标准
- •1. Treatment for greater than 24 months for Wilson Disease with chelation therapy (i.e.penicillamine, trientine hydrochloride) or zinc therapy.
- •2. Decompensated hepatic cirrhosis.
- •3. Model for End-Stage Liver Disease (MELD) score > 11.
- •4. Modified Nazer score > 6 (Dhawan et al. Liver Transplant 2005).
- •5. GI bleed within past 6 months.
- •6. ALT > 5x upper limit of normal (ULN).
- •7. Marked neurological disease requiring either nasogastric (NG) feeding or intensive in-patient medical care.
- •8. Severe anaemia with a haemoglobin < 9 mg/dL.
- •9. Participation in a clinical trial of an experimental or unapproved/unlicensed therapy at the same time or within the 4 weeks prior to this study.
- •10. History of seizure activity within 6 months of study start.
- •11. Pregnant (or women who are planning to become pregnant) or lactating women.
- •12. Known sensitivity to WTX101, WTX101 excipients (anhydrous di-calcium
- •phosphate, anhydrous sodium carbonate), any of the ingredients contained in
- •WTX101, PPIs or to related compounds.
- •13. Active infection with hepatitis B virus (positive hepatitis B surface antigen) or C virus (patients with positive hepatitis C antibody result would require confirmation of active disease with a positive hepatitis C polymerase chain reaction (PCR) test), seropositivity for human immunodeficiency virus (HIV).
- •14. Any disability acquired from trauma or another illness that, in the opinion of the Investigator, could interfere with evaluation of disability due to WD.
- •15. Previous treatment with tetrathiomolybdate.
- •16. Major systemic disease or other illness that would, in the opinion of the Investigator, compromise patient safety or interfere with the collection or interpretation of study results.
- •17. In the opinion of the Investigator, the patient is likely to be non-compliant or uncooperative during the study.
- •18. Any deviation in laboratory values that are confirmed on re-examination to be
- •clinically significant by the Investigator that would jeopardise the safety of the patient or impact the validity of the study results.
研究者
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