EUCTR2016-003876-29-GB进行中（未招募）1 期
CHELATE STUDY: Trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s disease - CHELATE STUDY GMPO-131-002
gmp-orphan SA0 个研究点目标入组 55 人开始时间: 2017年4月4日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 55
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
- •1. Patient is able to provide and has provided written informed consent
- •2. Written documentation has been obtained in accordance with the relevant country and local privacy requirements, where applicable, including for EU sites, Data Protection Consent
- •3. Male or female, aged = 18 and = 75 years old at time of consent
- •4. Patient has a diagnosis of Wilson’s disease, as defined by a prior or current Leipzig score of = 4
- •5. Patient’s Wilson’s disease is clinically stable, in the opinion of the investigator, and being treated with penicillamine for at least 1 year (52 weeks) prior to the screening/enrolment visit
- •6. Patient is on a stable dose and regimen of penicillamine for at least 4 months (16 weeks) prior to the screening/enrolment visit (other prescribed treatments for Wilson’s disease not permitted during this period)
- •7. No anticipated need that patient will require additional pharmacological therapies other than study medication, including prescribed zinc therapy, for the management of copper levels during the study
- •8. Patient must be willing to maintain stable diet throughout the study, and avoid foods with high copper content, including the Penicillamine Baseline period
- •9. Patient considered suitable to receive therapy with both TETA 4HCl and penicillamine administered twice a day
- •10. Negative central laboratory tests for HIV and viral hepatitis (results will be available after start of run-in period)
- •11. For female patients of childbearing potential, negative urine pregnancy test (at screening/enrolment visit and prior to randomization)
- •12. For females of childbearing potential, use of a reliable form of contraceptive
- •13. Patient is considered as able to complete study requirements and attend the study visits, in he opinion of the investigator
- •The Clinical Adjudication Committee will be responsible for reviewing
- •clinical study data provided by the sites to confirm that the
- •penicillamine-treated Wilson's disease patients enrolled into the
- •Penicillamine Baseline Period are well-controlled, clinically stable, and
- •are suitable for randomisation into the study at Week 12 visit.
- •Information associated with the Inclusion and Exclusion criteria
- •demonstrating that patients are adequately controlled and tolerating
- •penicillamine defined by serum NCC, 24-hour urinary copper excretion,
- •ALT and any other laboratory or clinical findings per protocol will be
- •used for this assessment prior to the Week 12 visit. Patient and site
- •identification will be anonymized to the adjudicators and additionally for
- •the evaluations post-randomisation the adjudicators will be blinded to
- •the treatment allocation.
- •Three adjudication members will review the data independently
- •according to the protocol criteria. A majority assessment of two or more
- •will determine the decision. The recommendation by the Clinical
- •Adjudication Committee on whether or not to include (randomise) the
- •patient will be final.
- •The details of the process and communication with the Investigator will
- •be detailed in a Charter for the Clinical Adjudication Committee
- •Are the trial subjects under 18? no
- •Number of subjects for this age range: 0
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 50
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1. Patient is in ‘de-coppering’ phase of treatment for Wilson’s disease, in the opinion of the investigator
- •2. Patient evidence of uncontrolled liver disease, including but not limited to:
- •a. Modified Nazer score of > 4 (result may not be available until after start of run-in period since based on lab results*)
- •b. decompensated cirrhosis
- •c. acute hemolytic anemia
- •d. acute hepatitis
- •e. hepatic malignancy
- •f. evidence of acute liver failure
- •3. Cause of patient’s liver disease is due to another condition, in the investigator’s opinion
- •4. Patient has severe anemia defined as hemoglobin of = 9 g/dL (result will be available after start of run-in period*)
- •5. Patient has experienced a gastrointestinal bleed within 6 months (24 weeks) prior to screening/enrolment visit
- •6. Patient has renal impairment defined as creatinine clearance of = 30 mL/min (result may not be available until after start of run-in period*), or patient has nephritis or nephrotic syndrome, in the opinion of the investigator
- •7. Patient has neurological disease that prevents swallowing of study medication (e.g. requires a nasogastric feeding tube) or requires intensive in-patient medical care
- •8. Patient is currently taking medication containing trientine for management of Wilson’s disease or has taken it within 4 months (16 weeks) of screening/enrolment visit
- •9. Patient is currently receiving prescribed zinc therapy for management of Wilson’s disease or has taken it within 4 months (16 weeks) of screening/enrolment visit
- •10. Patient is taking any of the following concomitant therapies: gold therapy, antimalarial therapy, cytotoxic drugs, oxyphenbutazone, phenyl butazone
- •11. Patient has a known intolerance, allergy or sensitivity to penicillamine (that is uncontrolled) or to TETA 4HCl, including any component of the study medication
- •12. For female patients of childbearing potential, planning a pregnancy during study period or currently nursing
- •13. For female patients of childbearing potential, unable or unwilling to use a reliable form of contraceptive throughout the study
- •14. Patient is currently participating in another therapeutic study, or has previously participated in a therapeutic study within 30 days of screening/enrolment visit (or longer, if local requirements specify this)
- •15. Patient has any condition or in any situation which, in the investigator’s opinion, puts the patient at significant risk, could confound study results, or may interfere significantly with the patient’s participation in the study
- •*Samples for screening/enrolment labs can be taken within ± 7 days of visit. In the event that lab value is above the specified threshold, it can be repeated. If repeated including at the Week 4 and Week 8 visits, with the result available prior to ramdomization at Week 12.
研究者
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