A new Phase 4 clinical trial, published on ClinicalTrials.gov, will investigate whether long-term treatment with acoramidis (Attruby) can not only stabilize but improve heart structure and function in people with transthyretin amyloid cardiomyopathy (ATTR-CM).
Acoramidis, which was approved by the U.S. Food & Drug Association (FDA) in 2024 to treat ATTR-CM, is an oral transthyretin (TTR) stabilizer. In ATTR-CM, misfolded TTR forms clumps called called amyloid fibrils that build up in the heart muscle; acoramidis is designed to stabilize TTR to help prevent the formation of these amyloid deposits.
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The ASCEND-ATTR study, sponsored by Eidos Therapeutics, a BridgeBio company, is designed to track changes in heart structure and function as well as the amount of amyloid deposits in the heart. The trial will use two types of imaging: cardiac magnetic resonance imaging (MRI) and echocardiography, which uses ultrasound to create images of the heart.
A recent press release from BridgeBio noted that the new study will build on findings from the Phase 3 ATTRibute-CM trial and its open-label extension, where researchers observed evidence of disease regression alongside sustained clinical benefits. In the open-label extension, acoramidis was associated with a 44% reduction in the risk of cardiovascular death through 42 months.
Learn more about ATTR-CM treatment and care
Up to about 150 adults ages 18 to 80 are expected to participate in the trial. The study is open-label, meaning all participants will receive acoramidis. The treatment (712 mg equivalent to 800 mg of acoramidis hydrochloride) will be taken twice daily for 36 months, with participants undergoing imaging and other assessments throughout the study.
The duration for participation is expected to be approximately 38 months, including 45 days for screening, 36 months for treatment and one additional month for follow up.
ASCEND-ATTR is currently recruiting patients and is expected to be completed by November 2030.
