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Recruiting NCT07306949

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

Phase IV Interventional Transthyretin-type Cardiac Amyloidosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Acoramidis.
Who it may be relevant to
Registry conditions: Transthyretin-type Cardiac Amyloidosis. Basic parameters: 18 years — 100 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Japan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug Acoramidis
    Participants will receive acoramidis tablets orally.

Primary outcome measures

  • Disease Progression Rate as Measured by N-terminal pro-brain-type Natriuretic Peptide (NT-proBNP) and/or Outpatient Diuretic Intensification (ODI) [Time frame: Baseline through 12 months]
Secondary outcome measures (6)
  • Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT) [Time frame: Baseline through 12 months]
  • Change from Baseline in Serum Transthyretin (TTR) [Time frame: Baseline, 28 days]
  • Number of Participants with a 30% Change from Baseline in NT-proBNP [Time frame: Baseline, 12 months and 18 months]
  • Change from Baseline in hs-CTnT [Time frame: Baseline, 12 months and 18 months]
  • Change from Baseline in Kansas City Cardiomyopathy Questionnaire (KCCQ-12) Overall Summary Score [Time frame: Baseline, 12 months and 18 months]
  • Change from Baseline in Estimated Glomerular Filtration Rate (eGFR) [Time frame: Baseline, 12 months and 18 months]

Eligibility criteria

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Japan · 16 centers
  • 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
  • … and 8 more centers

Identifiers

NCT: NCT07306949 · D9400L00001 · 2025-RWE-000214

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗