ACT-EARLY OLE
Research type
Research Study
Full title
A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients with Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)
IRAS ID
1012521
Contact name
Adam Castano
Contact email
Sponsor organisation
Eidos Therapeutics, Inc.
Clinicaltrials.gov Identifier
Research summary
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 the nerves is known as transthyretin amyloid polyneuropathy or ATTR-PN.
This study is an extension of the ACT-EARLY (AG10-501) study. ACT-EARLY is a study under investigation for the prevention of ATTR-CM and ATTR-PN in individuals that carry a defect in the TTR gene known to cause ATTRv disease but who have not yet developed any symptoms of the disease. This study, called the ACT-EARLY Open-Label Extension study (ACT-EARLY OLE), is designed so that participants in ACT-EARLY who transitioned to a diagnosis of ATTR-CM (ie they were diagnosed with ATTR-CM disease) can receive treatment with acoramidis and be followed for their ATTR-CM disease progression.
The primary objective of this study is to evaluate over a 5-year period how well acoramidis is tolerated and the side effects in participants with newly diagnosed ATTR-CM.
REC name
London - City & East Research Ethics Committee
REC reference
25/LO/0713
Date of REC Opinion
20 Oct 2025
REC opinion
Further Information Favourable Opinion