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

Recruiting now · Phase 4

Conditions studied: Transthyretin-type Cardiac Amyloidosis

In brief

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.

Key facts

Study ID

NCT07306949

Run by

Alexion Pharmaceuticals, Inc.

People needed

200

Starts

2025-10-01

Expected to finish

2028-03-31

Last updated by the study team

2026-01-07

Who can join

Age: 18 and older, up to 100. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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 it is running

• Research Site — Kurume-shi, Japan (enrolling)
• Research Site — Yufu-shi, Japan (enrolling)
• Research Site — Kumamoto, Japan
• Research Site — Kyoto, Japan
• Research Site — Mitaka-shi, Japan
• Research Site — Nagoya, Japan
• Research Site — Nankoku-shi, Japan
• Research Site — Bunkyō City, Japan
• Research Site — Ōtsu, Japan
• Research Site — Sagamihara-shi, Japan
• Research Site — Sapporo, Japan
• Research Site — Shinjuku-ku, Japan
• Research Site — Suita-shi, Japan
• Research Site — Tsu, Japan
• Research Site — Okayama, Japan
• Research Site — Bunkyō City, Japan

Full record on ClinicalTrials.gov

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