NCT07306949招募中4 期
A Multicenter, Open-Label, Single Arm, Post-Marketing Clinical Study to Evaluate the Therapeutic Effects of Acoramidis Hydrochlorideon Cardiac Biomarkers and Current Clinical Features in Patients With Transthyretin-type Cardiac Amyloidosis: The CARE ATTR Study
干预措施
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 16
- 主要终点
- Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI)
研究概览
简要总结
The purpose of this study is to confirm that the treatment with acoramidis prevents the deterioration of the ATTR-CM disease progression index and that these indexes are surrogate markers of disease progression.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Treatment history of ATTR-CM is one of the following:
- •Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
- •Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
- •Naive participants must meet the following requirements:
- •History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
- •Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)
- •Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods
- •Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
- •Bone scintigraphy showing strong accumulation ** (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis
排除标准
- •Have confirmed diagnosis of AL amyloidosis
- •Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
- •Likelihood of receiving a heart transplant within 1 year from the time screening begins
- •Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
- •Pregnant or lactating women
- •Has a clinically significant medical condition, an abnormal laboratory test result, or a condition that may jeopardize the safety of the study participant, increase the risk of participation in the post-marketing clinical trial, or affect the study
- •Participating in an interventional study other than this study, including a clinical trial
- •In the opinion of the responsible (sub)physician for the post-marketing clinical trial, has a history of drug abuse, alcoholism, or psychiatric disorder that would preclude compliance with this Post-Marketing Clinical Study Protocol
研究组 & 干预措施
Acoramidis
Experimental
Participants will receive 800 mg of acoramidis twice daily for 18 months.
干预措施: Acoramidis (Drug)
结局指标
主要结局
Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI)
时间窗: Baseline through 12 months
次要结局
- Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT)(Baseline through 12 months)
- Change from Baseline in Serum Transthyretin (TTR)(Baseline, 28 days)
- Number of Participants with a 30% Change from Baseline in NT-proBNP(Baseline, 12 months and 18 months)
- Change from Baseline in hs-CTnT(Baseline, 12 months and 18 months)
- Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score(Baseline, 12 months and 18 months)
- Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)(Baseline, 12 months and 18 months)
研究者
研究点 (16)
Loading locations...
相似试验
招募中
3 期
Extension Study of ACT-EARLY2024-513676-18-00Eidos Therapeutics Inc.92
已完成
3 期
A study to learn if lacosamide is safe to take over a long period of time in children with epilepsyEpilepsy2022-502639-21-00UCB Biopharma19
招募中
3 期
Comparison of Cyclophosphamide and doxorubicin as a first line treatment in elderly patients with metastatic sarcoma2024-510653-10-00Unicancer214
招募中
3 期
An open study for participants who have been on darolutamide treatment in any other Bayer studyCancer2022-502084-38-00Bayer Consumer Care AG, Bayer AG258
已完成
3 期
A study to investigate the safety and efficacy of AMX0035 in patients with Amyotrophic Lateral Sclerosis2023-508511-23-00Amylyx Pharmaceuticals Inc.526