2024-516431-27-00招募中4 期
Open label, Multicenter, Prospective Study to Characterize the Pharmacokinetics and Pharmacodynamics of Cufence (Trientine Dihydrochloride) and to Investigate the Efficacy and Safety in Wilson’s Disease Patients
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 46
- 试验地点
- 7
- 主要终点
- Serum ceruloplasmin (copper marker)
研究概览
简要总结
To characterize the Cufence dose – exposure – copper markers (24-hr urinary copper excretion (UCE), total serum copper (Cu), ceruloplasmin, non-ceruloplasmin bound copper (NCC)) relationships through population pharmacokinetic-pharmacodynamic (PKPD) modelling in Wilson’s disease patients and to evaluate the influence of patient characteristics on relevant model parameters, such as apparent clearance, apparent volume of distribution and drug potency.
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Patient (or a representative) must provide written, informed consent before the start of any study procedures.
- •Male and female patient aged ≥ 5 years at time of consent.
- •Diagnosis of WD previously determined by the physician based on a Leipzig score ≥
- •Patient (≥ 18 years) has previously been treated with D-penicillamine for WD.
- •Patient (< 18 years) has previously been treated with D-penicillamine or zinc for WD.
- •For female patients of childbearing potential, a negative pregnancy test at the Screening visit and the Baseline visit is required. In addition, a highly effective method (failure rate <1%) of birth control must be used during the study which includes (but is not limited to) the following: - vasectomized partner (at least 6 months prior to dosing); - oral, patch, or injected contraceptives, or vaginal hormonal device (i.e. NuvaRing®), in use for at least 3 consecutive months prior to study dosing and throughout the study duration; - implanted or intrauterine contraceptives in use for at least 6 consecutive months prior to study dosing and throughout the study duration; - abstinence (must agree to use a highly effective method if they become sexually active during the study).
- •Patient is considered to be able to complete study requirements and attend the study visits, in the opinion of the investigator.
排除标准
- •Patient has evidence of uncontrolled liver disease, including but not limited to: a. New Wilson index (Dhawan Index) > 10 b. Alanine aminotransferase (ALT) > 5x upper limit of normal (ULN) c. Aspartate aminotransferase (AST) > 5x ULN d. Model for End-Stage Liver Disease (MELD) score > 13 (only applicable for patients that are ≥ 12 years of age) e. Acute liver failure f. Hepatic malignancy
- •Uncontrolled neurological disease according to the judgement of the physician.
- •Patient has severe anaemia defined as hemoglobin of < 9 g/dL.
- •Patient has a known intolerance, allergy or sensitivity to trientine dihydrochloride, including any component of the study medication.
- •Female patient is pregnant or lactating.
- •Any patients who lack the capacity to consent, including the parent(s) of a paediatric patient.
结局指标
主要结局
Serum ceruloplasmin (copper marker)
Serum ceruloplasmin (copper marker)
Trientine clearance (CL/F) and volume(s) of distribution (V/F)
Trientine clearance (CL/F) and volume(s) of distribution (V/F)
24-hr UCE (copper marker)
24-hr UCE (copper marker)
NCC (copper marker)
NCC (copper marker)
Serum copper (copper marker)
Serum copper (copper marker)
Concentration of trientine in plasma
Concentration of trientine in plasma
Patient characteristics for covariates
Patient characteristics for covariates
次要结局
- Concentration of MAT in plasma
- Concentration of DAT in plasma
- AUC0-t, Cmax and Ctrough for trientine and metabolites. (As secondary endpoint, pre-dose concentrations are reported and summarized using NCA. While the pre-dose PK samples are collected shortly prior to the next dose, the pre-dose concentration is assumed to approximate the Ctrough.)
- Number of AEs including number of AEs leading to discontinuation of treatment with Cufence
- Changes in neurological disease status (Unified Wilson’s Disease Rating Scale (UWDRS))
- Changes in psychiatric symptoms (SCID-5, MMSE, EQ-5D-3L, PHQ-9, PHQ-9-A, CBCL)
- Changes in hepatic disease (full liver panel to determine the APRI, the Child-Pugh score, the FIB4 index and the New Wilson Index (Dhawan Index), and Fibroscan)
研究者
Carlot Kruse
Scientific
Univar Solutions B.V.
研究点 (7)
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