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

January 5, 2026 updated by: Alexion Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

200

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Japan
      • Bunkyō City, Japan, 113-8603
        • Not yet recruiting
        • Research Site
      • Bunkyō City, Japan, 113-8431
        • Not yet recruiting
        • Research Site
      • Kumamoto, Japan, 860-8556
        • Not yet recruiting
        • Research Site
      • Kurume-shi, Japan, 830-0011
        • Recruiting
        • Research Site
      • Kyoto, Japan, 606-8507
        • Not yet recruiting
        • Research Site
      • Mitaka-shi, Japan, 181-8611
        • Not yet recruiting
        • Research Site
      • Nagoya, Japan, 466-8560
        • Not yet recruiting
        • Research Site
      • Nankoku-shi, Japan, 783-8505
        • Not yet recruiting
        • Research Site
      • Okayama, Japan, 700-8558
        • Not yet recruiting
        • Research Site
      • Sagamihara-shi, Japan, 252-0375
        • Not yet recruiting
        • Research Site
      • Sapporo, Japan, 060-8543
        • Not yet recruiting
        • Research Site
      • Shinjuku-ku, Japan, 160-8582
        • Not yet recruiting
        • Research Site
      • Suita-shi, Japan, 564-8565
        • Not yet recruiting
        • Research Site
      • Tsu, Japan, 514-8507
        • Not yet recruiting
        • Research Site
      • Yufu-shi, Japan, 879-5593
        • Recruiting
        • Research Site
      • Ōtsu, Japan, 520-2192
        • Not yet recruiting
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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:

    1. History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
    2. Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)

    3. Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods

      1. Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
      2. 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Acoramidis
Participants will receive 800 mg of acoramidis twice daily for 18 months.
Drug: Acoramidis
Participants will receive acoramidis tablets orally.
Other Names:
  • acoramidis hydrochloride Byontra

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Baseline through 12 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Disease Progression Rate as Measured by NT-proBNP and/or High-sensitivity Cardiac Troponin T (hs-CTnT)
Time Frame: Baseline through 12 months
Baseline through 12 months
Change from Baseline in Serum Transthyretin (TTR)
Time Frame: Baseline, 28 days
Baseline, 28 days
Number of Participants with a 30% Change from Baseline in NT-proBNP
Time Frame: Baseline, 12 months and 18 months
Baseline, 12 months and 18 months
Change from Baseline in hs-CTnT
Time Frame: Baseline, 12 months and 18 months
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
Baseline, 12 months and 18 months
Change from Baseline in Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Baseline, 12 months and 18 months
Baseline, 12 months and 18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 1, 2025

Primary Completion (Estimated)

March 31, 2028

Study Completion (Estimated)

March 31, 2028

Study Registration Dates

First Submitted

December 15, 2025

First Submitted That Met QC Criteria

December 15, 2025

First Posted (Estimated)

December 29, 2025

Study Record Updates

Last Update Posted (Actual)

January 7, 2026

Last Update Submitted That Met QC Criteria

January 5, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • D9400L00001
  • 2025-RWE-000214 (Other Identifier: Alexion)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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