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Acoramidis to slow progression of transthyretin cardiac amyloidosis

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

Phase 4 Interventional Alexion Pharmaceuticals, Inc. · NCT07306949

This trial will test whether acoramidis slows disease progression in people with transthyretin cardiac amyloidosis who are either newly starting treatment or switching from tafamidis.

Quick facts

PhasePhase 4
Study typeInterventional
Enrollment200 (estimated)
Ages18 Years to 100 Years
SexAll
SponsorAlexion Pharmaceuticals, Inc. Industry-sponsored
Locations16 sites (Bunkyō City and 15 other locations)
Trial IDNCT07306949 on ClinicalTrials.gov

What this trial studies

This phase 4 interventional study gives acoramidis to adults with ATTR-CM who are treatment‑naive or currently treated with tafamidis and considered for switching. Participants will be followed over time using a composite disease progression index and serial cardiac biomarkers including NT-proBNP and high-sensitivity troponin T (hs-cTnT). The primary aim is to confirm that acoramidis prevents deterioration of the progression index and to determine whether these indexes function as surrogate markers of clinical worsening. The trial is being conducted at multiple research sites in Japan and is sponsored by Alexion Pharmaceuticals.

Who should consider this trial

Good fit: Ideal participants are adults with confirmed ATTR-CM who are either newly diagnosed and untreated (with prior heart‑failure symptoms or hospitalization and septal thickness >12 mm) or current tafamidis users whom investigators judge may benefit from switching to acoramidis.

Not a fit: Patients with cardiomyopathy from non‑ATTR causes, those with very advanced irreversible cardiac damage, or those who do not meet the diagnostic criteria are unlikely to receive benefit from this intervention.

Why it matters

Potential benefit: If successful, acoramidis could slow or prevent worsening of cardiac function and reduce biomarkers associated with heart failure in people with ATTR-CM.

How similar studies have performed: Randomized trials of TTR stabilizers such as tafamidis have demonstrated benefit in ATTR-CM, but direct real-world, post‑marketing evidence for acoramidis is still limited and is being collected in this study.

Eligibility criteria

Show full inclusion / exclusion 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:

  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

Where this trial is running

Bunkyō City and 15 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Transthyretin-type Cardiac AmyloidosisATTR-CMNT-proBNPODIhs-cTnTAcoramidis
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.