A Phase 3 interventional study of Acoramidis and Placebo oral tablet in Amyloidosis, Amyloid Cardiomyopathy and Transthyretin Amyloidosis, sponsored by Eidos Therapeutics, a BridgeBio company. Recruiting at 104 sites in 24 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-08-04.
Sponsored by Eidos Therapeutics, a BridgeBio company (part of BridgeBio) · Phase 3, Interventional, and Prevention
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.
Acoramidis is an experimental drug designed to bind tightly to TTR in the blood and stabilize its structure, so it does not form the harmful amyloid plaques that can cause damage to organs.
This study is intended to determine if treatment with acoramidis in participants with ATTRv who have not yet developed any symptoms of disease can prevent or delay the development of ATTR-CM or ATTR-PN disease. If adults with an inherited defective TTR gene are treated early before any of the symptoms of disease have developed, it may be possible to delay the onset or prevent the disease entirely.
The AG10-501 ACT-EARLY study is a randomized, multicenter, double-blind, placebo-controlled study of acoramidis for prevention of ATTR (with specific reference to either its cardiomyopathic or polyneuropathic manifestations). Participants will be stratified at randomization.
The study population will be asymptomatic carriers of a known pathogenic TTR gene variant. A participant must be 18 to 75 inclusive years of age, and the age of the participant must be within 10 years younger than or older than the predicted age of disease onset (PADO) based either on family history (pedigree analysis) or, if family history is insufficient, based on a TTR Variant Actuarial table from published literature. For example, if PADO for a given individual is found to be 50 years, the age of the participant must be between 40 and 75 years inclusive.
82 studies on the registry are indexed under Amyloidosis, Hereditary, Transthyretin-Related; 28 are open to participants now.
This study's planned enrollment of 587 is above the median of 80 across 36 interventional studies indexed under Amyloidosis, Hereditary, Transthyretin-Related.
Browse Amyloidosis, Hereditary, Transthyretin-Related studies →Eidos Therapeutics, a BridgeBio company is the lead sponsor of 11 studies on the registry; 3 are open to participants now.
First submitted after Eidos Therapeutics became part of BridgeBio.
Compared within Amyloidosis, Hereditary, Transthyretin-Related, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Amyloidosis. The counts are of registry records indexed under each term, refreshed daily. How we classify →
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)
Drug: Acoramidis
Subjects will receive placebo to match twice daily
Drug: Placebo oral tablet
TTR stabilizer administered orally twice daily (BID)
Also known as: AG10, ALXN2060
Non-active control administered orally twice daily (BID)
Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated)
* ATTR-CM defined by biopsy or imaging-based diagnosis * ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
Time frame: Since randomization up to approximately 7 years or until the study is declared over
Time to development of ATTR-CM (centrally adjudicated)
ATTR-CM defined by biopsy or imaging-based diagnosis
Time frame: Since randomization up to approximately 7 years or until the study is declared over
Time to development of ATTR-PN (centrally adjudicated)
ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
Time frame: Since randomization up to approximately 7 years or until the study is declared over
Showing the first 100 of 104 sites across 24 countries. Choose a country and city above to see the rest.
Started May 2025 (actual). Primary completion is estimated for Oct 2031, 77 months after the start.
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Studies of the drug tested here: Acoramidis
Amyloidosis, Hereditary, Transthyretin-Related →
Eidos Therapeutics, a BridgeBio company →