Multi-Center Study for the Assessment of Copper Parameters in Wilson Disease Subjects on Standard of Care Treatment
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 64
- 试验地点
- 1
- 主要终点
- Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6 Months Of Treatment
研究概览
简要总结
This was a 24-month study to assess copper parameters in participants with Wilson disease (WD) treated with standard of care (SoC) medications.
After providing informed consent, participants meeting all inclusion and no exclusion criteria were enrolled into the study as outpatients. The participants' routine clinic visits were scheduled according to the standard clinical practice at the study center and at the discretion of the treating physician at approximate 6-month intervals.
At the time of enrollment, participants were receiving SoC medications for the treatment of WD, which could include penicillamine, trientine, zinc, or a combination of a copper chelator and zinc. If treatment was interrupted or stopped during the course of the study, participants continued in the study and biological samples and clinical data were continued to be collected for the full 24-month study period. Dosing with SoC agents was individualized and managed by the treating physician at the study center according to standard clinical practice at the site.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Willing and able to give informed consent for participation in the study.
- •Male or female participants, aged 18 years or older as of signing the informed consent form.
- •Receiving SoC medications (penicillamine, trientine, zinc, or copper chelators with zinc) for the treatment of WD at the time of enrollment and for no more than 60 months prior to enrollment.
- •Able to understand and willing to comply with study procedures and requirements, as judged by the Investigator.
- •Established diagnosis of WD.
- •Adequate venous access to allow for collection of blood samples.
排除标准
- •Major systemic disease or other illness that would, in the opinion of the Investigator, compromise participant safety or interfere with the collection or interpretation of study results.
- •In the opinion of the Investigator, the participant was likely to be non-compliant or uncooperative during the study.
研究组 & 干预措施
Total
干预措施: Standard of Care Medications (Drug)
结局指标
主要结局
Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of Non-ceruloplasmin-bound Copper (NCC) Or Reached A Reduction Of ≥ 25% In NCC During 6 Months Of Treatment
时间窗: Baseline through Month 6
To achieve a normalized NCC concentration, participants must have had 2 consecutive measures within (or below) the normal concentration range (0.8 to 2.3 micromolar \[μM\]). Two consecutive measurements required that the measurements occurred on separate dates and were assigned to 2 different visits. Non-ceruloplasmin-bound copper was calculated by subtracting the amount of copper bound to the ceruloplasmin from the total plasma copper concentration.
次要结局
- Change From Baseline In Serum Total Ceruloplasmin (Nephelometry) Used To Calculate NCC At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In NCC Concentrations At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Plasma Total Molybdenum At Month 6 And Month 24(Baseline, Month 6, Month 24)
- Time To Normalization Of NCC If Above The Reference Range At The Time Of Enrollment(Baseline, up to 24 months)
- Change From Baseline In 24-Hour Urinary Copper At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Percentage Of Participants Who Achieved Or Maintained Normalized Concentrations Of NCC Or Reached A Reduction Of ≥ 25% In NCC Through Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Exchangeable Copper At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Copper Plasma Ultrafiltrate At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Plasma Total Copper Used To Calculate NCC At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Molybdenum Plasma Ultrafiltrate At Month 6 And Month 24(Baseline, Month 6, Month 24)
- Change From Baseline In 24-Hour Urinary Molybdenum At Month 6 And Month 24(Baseline, Month 6, Month 24)
- Change From Baseline In Hepatic Laboratory Measures For Alanine Aminotransferase (ALT) At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Hepatic Laboratory Measures For Aspartate Aminotransferase (AST) At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Hepatic Laboratory Measures For International Normalized Ratio (INR) At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- CGI Scale Item 2 (Global Improvement) At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Hepatic Laboratory Measures For Bilirubin At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))
- Change From Baseline In Clinical Global Impression (CGI) Scale Item 1 (Severity Of Illness) At Month 6, Month 24, And Last Assessment(Baseline through Last Assessment (ranging from 1 to 24 months))