Phase 3Recruiting
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
- Sponsor
- Eidos Therapeutics, a BridgeBio company (Industry)
- Enrollment
- 587 planned
- Conditions
- Amyloidosis; Amyloid Cardiomyopathy; Transthyretin Amyloidosis; Cardiomyopathies; Heart Diseases; Polyneuropathies
- Interventions
- Acoramidis; Placebo oral tablet
- Ages
- adults, older adults
- Registry
- NCT06563895, jRCT2071250116
Full record on ClinicalTrials.gov
From the registry
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like substance that accumulates in different organs in the body and can cause damage to the organ. There are two ways that the TTR protein can fall apart. One way occurs as a person ages, where the normal TTR protein can fall apart and form amyloid that may no longer be sufficiently cleared by the body. This type of ATTR is known as wild-type ATTR (ATTRwt). The other way occurs when a person inherits a defective TTR gene that causes the TTR protein to spontaneously fall apart. This form of the disease is known as variant ATTR (ATTRv) and can be detected in adults by a genetic test of their TTR gene before they age. Amyloid build-up in the heart causes the heart wall to become thick and stiff and can result in heart failure and even death. Accumulation of TTR amyloid in the heart is known as transthyretin amyloid cardiomyopathy or ATTR-CM. Amyloid can also deposit in the nerve tissues leading to nerve problems. Accumulation of TTR in…
Primary outcomes
- Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated)
History
Study completion expected
Primary completion expected
Last registry update
Started
Registered