Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers

Официальное название

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

Обзор

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.

Вмешательства

• Препарат Acoramidis
  Participants will receive acoramidis tablets orally.

Первичные конечные точки

• Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI) [Срок оценки: Baseline through 12 months]

Вторичные конечные точки (6)

• Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT) [Срок оценки: Baseline through 12 months]
• Change from Baseline in Serum Transthyretin (TTR) [Срок оценки: Baseline, 28 days]
• Number of Participants with a 30% Change from Baseline in NT-proBNP [Срок оценки: Baseline, 12 months and 18 months]
• Change from Baseline in hs-CTnT [Срок оценки: Baseline, 12 months and 18 months]
• Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score [Срок оценки: Baseline, 12 months and 18 months]
• Change from Baseline in Estimated Glomerular Filtration Rate (eGFR) [Срок оценки: Baseline, 12 months and 18 months]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Япония · 16 центров

• Research Site — Bunkyō City
• Research Site — Bunkyō City
• Research Site — Kumamoto
• Research Site — Kurume-shi
• Research Site — Kyoto
• Research Site — Mitaka-shi
• Research Site — Nagoya
• Research Site — Nankoku-shi
• … и ещё 8 центров

Идентификаторы

NCT: NCT07306949 · D9400L00001 · 2025-RWE-000214

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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