ClinicalTrials.gov record NCT06563895
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)
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AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 3, randomized, double-blind, placebo-controlled trial tests whether acoramidis can prevent or delay transthyretin amyloidosis in people who carry a pathogenic TTR variant but have no symptoms. Participants are assigned to oral acoramidis twice daily or matching placebo and followed for up to about 7 years. The main measure is time until clinical ATTR appears in the heart (ATTR-CM) or nerves (ATTR-PN).
What it is studying. The study is testing whether taking acoramidis twice daily, compared with a matching placebo, changes the time it takes for asymptomatic carriers of a pathogenic TTR gene variant to develop transthyretin amyloid cardiomyopathy (ATTR-CM) or transthyretin amyloid polyneuropathy (ATTR-PN).
What the researchers hope to learn. To determine whether acoramidis delays or prevents the onset of transthyretin amyloid cardiomyopathy (ATTR-CM) or polyneuropathy (ATTR-PN) in asymptomatic carriers of pathogenic TTR variants, measuring time to clinical disease as the primary outcome.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Acoramidis
- Placebo oral tablet
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Male or female ≥ 18 to ≤ 75 years of age inclusive.
- Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or all other pathogenic TTR variants).
- Participant's age is within 10 years younger than or older than PADO.
- Key
Exclusion criteria
- Evidence of ATTR-CM or ATTR-PN.
- Current or past (within last 1 to 12 months, depending on specific agent) treatment with other TTR modifying therapies.
- Contraindication to or inability to undergo cardiac magnetic resonance testing.
- Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy
- Other diseases or conditions such has cancer within 5 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV.
- Major surgery within the past 3 months or planned during the next 12 months.
- Known hypersensitivity to acoramidis.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
If enrolled, participants are randomly assigned to take oral acoramidis or a matching placebo twice daily. They will be followed for up to about 7 years with regular clinic visits that include cardiac imaging (cardiac MRI) and other assessments to monitor for heart or nerve disease; tissue biopsy may be used if needed to confirm ATTR. Some visits and tests required for diagnosis or safety (for example MRI or biopsies) are mentioned in the registry; the exact visit schedule and other routine tests are not specified in the record.
How the study is structured. Participants are randomly assigned (stratified at randomization) to receive either acoramidis 712 mg orally twice daily (BID) or matching placebo in a parallel-group, double-blind design. The study is Phase 3 and placebo-controlled, with central adjudication of ATTR diagnoses. Participants are followed for outcomes for up to approximately 7 years or until the study ends.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Amyloidosis; Amyloid Cardiomyopathy; Transthyretin Amyloidosis; Cardiomyopathies; Heart Diseases; Polyneuropathies. Does it apply to my diagnosis?
- Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
- The listed sites are in Argentina, Australia, Belgium, Brazil, Canada, Denmark, France, Germany, Greece, Ireland, Italy, Japan, Malaysia, Martinique, Mexico, Netherlands, Portugal, Singapore, South Korea, Spain, Sweden, Taiwan, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on wild-type ATTR amyloidosis
This trial is also listed on this site under ATTRv amyloidosis.
Important dates
- Study start
- May 12, 2025
- Primary completion
- October 2031 (estimated)
- Study completion
- December 2032 (estimated)
- Target enrollment
- 587 participants (estimated)
- Last updated on registry
- Aug 4, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Medical Information 📞 1-844-550-2246 ✉ medinfo@eidostx.com
Participating sites (104)
- University of California, San Diego (UCSD) - Medical CenterRecruiting
La Jolla, California, 92037 · United States
- University of California, Los Angeles (UCLA) - David Geffen School of MedicineRecruiting
Los Angeles, California, 90095 · United States
- University of California, San Francisco (UCSF)Recruiting
San Francisco, California, 94143 · United States
- Stanford UniversityRecruiting
Stanford, California, 94305 · United States
- University of Colorado AnschutzRecruiting
Aurora, Colorado, 80045 · United States
- Yale University School of Medicine - Section of CardiologyRecruiting
New Haven, Connecticut, 06519 · United States
- MedStar Washington Hospital Center - MedStar Heart and Vascular InstituteRecruiting
Washington D.C., District of Columbia, 20010 · United States
- Mayo Clinic - JacksonvilleRecruiting
Jacksonville, Florida, 32224 · United States
- University of MiamiRecruiting
Miami, Florida, 33136 · United States
- Cleveland Clinic FloridaRecruiting
Weston, Florida, 33331 · United States
Terms used on this page · explanations by MedicalUpdates.ai
- Contraindication
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- Key Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Key Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- double-blind
- Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
- placebo-controlled
- A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
- stratified
- Randomisation done separately within subgroups, such as age bands or disease severity, so that each group contains a similar mix.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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