A Prospective, Longitudinal, Multi-center, Observational Study of Acoramidis In Patients With Wild-type or Variant Transthyretin Amyloid Cardiomyopathy (ATTR-CM): MOSAIC-TTR Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 500
- 试验地点
- 1
- 主要终点
- Patient Reported Outcome Measure: Amylo-AFFECT-QOL score
研究概览
简要总结
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive heart disease caused by the buildup of an abnormal protein, called transthyretin (TTR), in the heart. This buildup can make it harder for the heart to pump blood and may lead to worsening symptoms over time. Acoramidis is a medicine approved for the treatment of adults with wild-type or hereditary (variant) ATTR-CM. While its benefits have been demonstrated in clinical trials, more information is needed about how it is used and how patients do in everyday medical practice.
The MOSAIC-TTR study is an observational study in France. Participants will receive acoramidis as part of their usual medical care. No experimental treatments or additional medical procedures will be required. The study will collect information directly from participating hospitals and from the Healthcare European Amyloidosis Registry (HEAR; NCT05101304). The main goal of the study is to understand how patients' quality of life, daily functioning, and overall well-being change during the first 12 months of treatment with acoramidis, using questionnaires completed by the patients themselves. The study will also collect information about the characteristics of patients receiving acoramidis, how the medicine is used in routine clinical practice, and its safety and tolerability. The information collected will help improve the understanding of ATTR-CM and may help improve the care of people living with this condition.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 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
- •Greater than or equal to 18 years of age
- •Established diagnosis of ATTR-CM with either wild-type TTR or a variant TTR genotype
- •Initiating treatment with acoramidis with the decision to treat having been made by the treating physician prior to study enrolment in accordance with the current French Summary of Product Characteristics (SmPC)
排除标准
- •Any contraindications as listed in the local approved product information
- •Prior treatment with disease-modifying treatment (e.g., tafamidis, TTR silencers and/or depleters)
- •Participation in an investigational trial evaluating new Investigational Medicinal Products or new Investigational Medical Device outside of routine clinical practice
- •Under legal protection (guardianship, curatorship, or other legal protection measures) or deprived of liberty by administrative or judicial decision at the time of inclusion.
研究组 & 干预措施
Single Arm
Patients will receive acoramidis 712 mg orally BID (twice daily)
干预措施: Acoramidis (Drug)
结局指标
主要结局
Patient Reported Outcome Measure: Amylo-AFFECT-QOL score
时间窗: At 3, 6, 9, and 12 months after initiation of acoramidis
Change from baseline in Amylo-AFFECT-QOL total score
Patient Reported Outcome Measure: KCCQ-23 score
时间窗: At 3, 6, 9, and 12 months after initiation of acoramidis
Change from baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-23) total score
Patient Reported Outcome Measure: EQ-5D-3L index score
时间窗: At 3, 6, 9, and 12 months after initiation of acoramidis
3L index score of EQ-5D Health-Related Quality of Life Questionnaire
Patient Reported Outcome Measure: EQ VAS score
时间窗: At 3, 6, 9, and 12 months after initiation of acoramidis
Visual Analog scale score of EQ-5D Health-Related Quality of Life Questionnaire
次要结局
- Demographic characteristics: Age(Before treatment with acoramidis)
- Demographic characteristics: Sex(Before treatment with acoramidis)
- Demographic characteristics: Body Mass Index(Before treatment with acoramidis)
- Clinical characteristics: Time between diagnosis and treatment initiation(Before treatment with acoramidis)
- Clinical characteristics: ATTR-CM diagnosis(Before treatment with acoramidis)
- Clinical characteristics: ATTR-CM manifestations(Before treatment with acoramidis)
- Clinical characteristics: Comorbidities(Up to 12 months before initiation of acoramidis, and during treatment with acoramidis)
- Clinical characteristics: Prior medication and procedures(Up to 12 months before initiation of acoramidis)
- Treatment patterns: Treatment duration(During treatment with acoramidis)
- Treatment patterns: Treatment interruptions(During treatment with acoramidis)
- Treatment patterns: Treatment discontinuation(During treatment with acoramidis)
- Treatment patterns: Time to treatment discontinuation(During treatment with acoramidis)
- Treatment patterns: Concomitant medications and procedures(During treatment with acoramidis)
- Safety and tolerability: Adverse events(During treatment with acoramidis and up to 6 days after the last intake of acoramidis in case of premature permanent treatment discontinuation)