ClinicalTrials.gov record NCT07116473

To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

A Phase 3, Open-Label, Multicenter, Extension Study of Acoramidis in Patients With Newly Diagnosed Variant Transthyretin Amyloid Cardiomyopathy (ACT-EARLY OLE)

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age18 years old and older
SexAll
AI summaryWhat this study is about, 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.

This is an open-label extension study testing acoramidis for long-term safety and tolerability in people with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM). Only participants who completed the earlier AG10-501 (ACT-EARLY) study with a diagnosis of ATTR-CM may enroll. Participants receive oral acoramidis and are followed for up to 5 years.

What it is studying. The study is testing the long-term safety and tolerability of acoramidis in people with newly diagnosed ATTR-CM. Safety will be tracked by recording treatment-emergent adverse events (AEs) and serious adverse events (SAEs), AEs that cause stopping the drug, abnormal physical exam findings, clinically relevant changes in vital signs, ECG changes, and changes in clinical safety laboratory tests over the study period.

What the researchers hope to learn. To build a longer-term safety and tolerability profile of acoramidis in people with newly diagnosed ATTR-CM by collecting data on adverse events, vital signs, ECGs, and relevant laboratory safety measures over up to 5 years.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

The AG10-504 study is an open-label extension study of acoramidis in participants with newly diagnosed transthyretin amyloid cardiomyopathy (ATTR-CM) or both ATTR-CM and transthyretin amyloid polyneuropathy (ATTR-PN).

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Acoramidis (AG10)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Participants must have completed the AG10-501 study (ACT-EARLY study) within the past 60 calendar days with a diagnosis of ATTR-CM (based on the AG10-501 protocol definition of ATTR-CM).
  • Key

Exclusion criteria

  • Participants who completed the AG10-501 study with a diagnosis of ATTR-PN only or who permanently discontinued study drug prior to diagnosis of ATTR-CM in AG10-501.
  • History of light-chain amyloidosis (AL) or another non-TTR amyloid subtype (eg, ApoA-1, gelsolin).
  • History of a monoclonal paraprotein or abnormal light chains in serum or urine (ie, monoclonal gammopathy of undetermined significance, MGUS) in which AL has not been ruled out.
  • Stage IV or V chronic kidney disease.
  • Active malignancy, except for basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix that has been successfully treated.
  • History of any organ transplant (with the exception of corneal transplant).
  • Known hypersensitivity to acoramidis or any of its excipients.
  • Major surgery planned during the next 6 months (some exceptions).
  • Treatment of ATTR-CM with any ATTR-oriented on- or off-label or OTC product.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

The AG10-504 study is the extension of the Phase 3 AG10-501 study (ACT-EARLY) which was designed to investigate if the use of acoramidis as a preventative intervention in individuals who are carriers of a known pathogenic transthyretin (TTR) variant but with no clinical evidence of ATTR could prevent or delay the onset of clinically detectable ATTR and the considerable morbidity and mortality that result from this devastating, progressive, and ultimately fatal disease. Only participants who have completed the AG10-501 study (ACT-EARLY) with a diagnosis of ATTR-CM may enroll in this AG10-504 Open Label Extension (OLE) study. The primary objective of the AG10-504 study is to evaluate the long-term safety and tolerability of acoramidis in participants with newly diagnosed ATTR-CM. The duration of this study will be up to 5 years. Currently, acoramidis is approved for the treatment of ATTR-CM in some regions including the United States, all member states of the European Union (EU), Albania, Bosnia Herzegovina, Brazil, Iceland, Israel, Japan, Liechtenstein, Montenegro, North Macedonia, Norway, Switzerland, and the United Kingdom (UK).

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.

To join, participants must have completed the AG10-501 (ACT-EARLY) study within the past 60 days with a diagnosis of ATTR-CM. Enrolled participants will take acoramidis 712 mg orally twice daily and be followed for up to 5 years in this open‑label extension. The study involves regular safety monitoring — reporting side effects, physical exams, vital signs, ECGs, and clinical safety laboratory tests — and the registry record does not list invasive procedures such as biopsies or lumbar punctures. People who completed AG10-501 with ATTR-PN only, have a history of non‑TTR amyloidosis (including light‑chain/AL), certain monoclonal protein or abnormal light chain findings where AL has not been ruled out, stage IV or V chronic kidney disease, active malignancy (with specified exceptions), prior organ transplant (except corneal), known hypersensitivity to acoramidis or its excipients, planned major surgery within 6 months (some exceptions), or who are being treated for ATTR-CM with any ATTR‑oriented product are not eligible according to the registry.

How the study is structured. Phase 3, open-label, single-group extension trial. Participants receive acoramidis orally twice daily (712 mg BID, equivalent to 800 mg acoramidis HCl BID) with no masking. The study continues for up to 5 years and follows participants who completed the AG10-501 (ACT-EARLY) study and have a diagnosis of ATTR-CM.

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 in Transthyretin (TTR); Amyloidosis, Familial; Amyloid Cardiomyopathy; Amyloid Cardiomyopathy, Transthyretin-Related; Amyloidogenic Transthyretin (ATTR) Amyloidosis; Heart Disease; Cardiomyopathies; Amyloidosis, Hereditary, Transthyretin-Related; Polyneuropathies; Amyloidosis. Does it apply to my diagnosis?
  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • No sites are listed on the registry yet. Where will the study take place?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on ATTRv amyloidosis

This trial is also listed on this site under wild-type ATTR amyloidosis.

Clinical trials106 trials listed for ATTRv amyloidosisEvery trial the registry lists for ATTRv amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in ATTRv amyloidosis researchLatest monthly update: September 2026. Includes: Gene-silencing drugs. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
November 2026 (estimated)
Primary completion
November 2037 (estimated)
Study completion
December 2037 (estimated)
Target enrollment
250 participants (estimated)
Last updated on registry
Aug 11, 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.

Participating sites

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

open-label extension
A continuation study in which participants who completed an earlier study can keep receiving the treatment, usually with everyone knowing what they are given.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
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.
Open Label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
primary objective
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.

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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