A Phase 2 interventional study of Acoramidis in Amyloid Cardiomyopathy, sponsored by Eidos Therapeutics, a BridgeBio company. Terminated at 9 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Eidos Therapeutics, a BridgeBio company (part of BridgeBio) · Phase 2, Interventional, and Treatment
Study ended by the sponsor due to meeting Study Objectives
Masri A, Aras M, Grogan M, Jacoby D, Maurer MS, Shah SJ, Witteles R, Wong PW, Ji AX, DU J, Siddhanti S, Katz L, Sinha U, Fox JC, Judge DP. Acoramidis for Trans…PubMed 40216220 ↗. 1 publication citing this study linked from PubMed
This prospective, multicenter open-label study will evaluate the long-term safety, tolerability, PK and PD of acoramidis administered on a background of stable heart failure therapy.
An Open-Label Extension and Safety Monitoring Study of Patients with Symptomatic Transthyretin Cardiomyopathy Who Have Completed the Phase II Study AG10-201
The primary objective of this study is to evaluate the long-term safety and tolerability of acoramidis administered to adult patients with symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM) in patients who have completed the study AG10-201.
This study will be an Open-Label Extension and Safety Monitoring Study of up to 55 male and/or female patients with symptomatic ATTR-CM, ≥18 years of age, who have completed the Phase II Study AG10-201. Enrollment into the study will be followed by visits at Day 14, Day 45, 3 Months and every 3 months thereafter until Month 54. After the Month 54 Visit, clinic visits will be every 6 months. There will be one follow-up visit approximately 30 days after last dose.
If all doses are well tolerated, the duration of each patient's participation in the study will continue based on periodic recommendations of the acoramidis Data Monitoring Committee (DMC) and/or registration of the product for the treatment of symptomatic ATTR-CM.
The secondary objectives of this study are to characterize the pharmacokinetics (PK) of acoramidis administered orally twice daily in patients with symptomatic ATTR-CM, and to describe the long-term pharmacodynamic (PD) properties of acoramidis as assessed by established assays of transthyretin (TTR) stabilization, including Fluorescent Probe Exclusion (FPE) assay and Western blot, and to describe the PK-PD relationship of acoramidis in adult patients with symptomatic ATTR-CM.
188 studies on the registry are indexed under Amyloid Neuropathies, Familial; 94 are open to participants now.
This study's enrollment of 47 is below the median of 54 across 90 interventional studies indexed under Amyloid Neuropathies, Familial.
Browse Amyloid Neuropathies, Familial 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.
Counted across the registry records on this site, refreshed daily.
The criteria were revised on the registry on Oct 14, 2026. See what changed
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants received open-label acoramidis 800 mg orally twice daily.
Drug: Acoramidis
Acoramidis 800 mg twice daily, oral administration
Also known as: AG10
Overall Summary of Treatment-Emergent Adverse Events.
Safety and tolerability were assessed by treatment-emergent adverse events (TEAEs), serious TEAEs, adverse events leading to treatment discontinuation.
Time frame: From the first dose of study drug until 30 days after the last dose of study drug. (Maximum exposure reported 84.1 months).
Acoramidis Pharmacodynamic Assessments of TTR Stabilization by Fluorescent Probe Exclusion Assay
Acoramidis binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: Fluorescent Probe Exclusion (FPE) Assay
Time frame: Day 45 and Month 78
Acoramidis Pharmacodynamic Assessments: Prealbumin
Acoramidis binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: quantitation of prealbumin (TTR).
Time frame: Baseline, Day 45, and Month 78
Plasma Concentrations of Acoramidis
Predose plasma concentrations of acoramidis were summarized by study visit in the PK Analysis Set.
Time frame: Baseline, Day 45, and Month 78
Plasma Concentrations of Acoramidis Acylglucuronide
Predose plasma concentrations of acoramidis acylglucuronide were summarized by study visit in the PK Analysis Set.
Time frame: Baseline, Day 45, and Month 78
| Milestone | Acoramidis HCl |
|---|---|
| Started | 47 |
| Completed | 0 |
| Not completed | 47 |
| Withdrew: Adverse event | 7 |
| Withdrew: Death | 12 |
| Withdrew: Physician decision | 3 |
| Withdrew: Withdrawal by subject | 1 |
| Withdrew: Discontinued from study due to sponsor decision to end the study | 17 |
| Withdrew: Other study discontinuation reasons | 6 |
| Withdrew: Discordant entry noted after early site closure | 1 |
Safety and tolerability were assessed by treatment-emergent adverse events (TEAEs), serious TEAEs, adverse events leading to treatment discontinuation.
| Participants | Acoramidis HCl |
|---|---|
| Any TEAEs | 47 |
| Any Treatment-Related TEAEs | 7 |
| Any TEAEs with an Outcome of Death | 17 |
| Any Serious TEAEs | 40 |
| Any Treatment-Related Serious TEAEs | 1 |
| Any Non-Serious TEAEs | 46 |
| Any TEAEs Leading to Discontinuation of IMP | 6 |
| Any TEAEs Leading to Study Discontinuation | 17 |
Acoramidis binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: Fluorescent Probe Exclusion (FPE) Assay
| Percent Target Engagement | Acoramidis HCl |
|---|---|
| Day 45 | 118.69 ± 90.707 |
| Month 78 | 103.54 ± 16.712 |
Acoramidis binding to and/or stabilization of TTR will be evaluated by established ex vivo assays: quantitation of prealbumin (TTR).
| mg/dL | Acoramidis HCl |
|---|---|
| Baseline (Day 1) | 21.55 ± 5.667 |
| Day 45 | 30.35 ± 5.028 |
| Month 78 | 30.68 ± 5.358 |
Predose plasma concentrations of acoramidis were summarized by study visit in the PK Analysis Set.
| ng/mL | Acoramidis HCl |
|---|---|
| Baseline (Predose) | 0.00 ± 0.000 |
| Day 45 (Predose) | 2166.13 ± 609.524 |
| Month 78 (Predose) | 2739.23 ± 870.981 |
Predose plasma concentrations of acoramidis acylglucuronide were summarized by study visit in the PK Analysis Set.
| ng/mL | Acoramidis HCl |
|---|---|
| Baseline (Predose) | 0.000 ± 0.0000 |
| Day 45 (Predose) | 2445.844 ± 2128.5501 |
| Month 78 (Predose) | 2911.385 ± 2275.2234 |
Collected over From the first dose of study drug until 30 days after the last dose of study drug. (Maximum exposure reported 84.1 months).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Acoramidis HCl | 17/47 (36.2%) | 40/47 (85.1%) | 46/47 (97.9%) |
| Event | Acoramidis HCl |
|---|---|
| Cardiac failure acuteCardiac disorders | 14/47 |
| Acute kidney injuryRenal and urinary disorders | 12/47 |
| FallInjury, poisoning and procedural complications | 8/47 |
| Atrial fibrillationCardiac disorders | 6/47 |
| Cardiac failure congestiveCardiac disorders | 6/47 |
| Cardiorenal syndromeCardiac disorders | 6/47 |
| Atrial flutterCardiac disorders | 5/47 |
| Cardiac failureCardiac disorders | 5/47 |
| Cardiac amyloidosisCardiac disorders | 4/47 |
| Cardiogenic shockCardiac disorders | 4/47 |
| Event | Acoramidis HCl |
|---|---|
| FallInjury, poisoning and procedural complications | 25/47 |
| ConstipationGastrointestinal disorders | 16/47 |
| Back painMusculoskeletal and connective tissue disorders | 15/47 |
| COVID-19Infections and infestations | 13/47 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 12/47 |
| FatigueGeneral disorders | 12/47 |
| CoughRespiratory, thoracic and mediastinal disorders | 12/47 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 11/47 |
| EpistaxisRespiratory, thoracic and mediastinal disorders | 11/47 |
| HypervolaemiaMetabolism and nutrition disorders | 11/47 |
| Age, Categorical(Participants) | Acoramidis HCl |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 2 |
| >=65 years | 45 |
| Age, Continuous(Years) | Acoramidis HCl |
|---|---|
| Mean | 74.3 ± 6.76 |
| Sex: Female, Male(Participants) | Acoramidis HCl |
|---|---|
| Female | 4 |
| Male | 43 |
| Race (NIH/OMB)(Participants) | Acoramidis HCl |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 1 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 9 |
| White | 35 |
| More than one race | 0 |
| Unknown or Not Reported | 2 |
| Ethnicity (NIH/OMB)(Participants) | Acoramidis HCl |
|---|---|
| Hispanic or Latino | 4 |
| Not Hispanic or Latino | 42 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(Participants) | Acoramidis HCl |
|---|---|
| United States | 47 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Study ended by the sponsor due to meeting Study Objectives
Masri A, Aras M, Grogan M, Jacoby D, Maurer MS, Shah SJ, Witteles R, Wong PW, Ji AX, DU J, Siddhanti S, Katz L, Sinha U, Fox JC, Judge DP. Acoramidis for Trans…PubMed 40216220 ↗. 1 publication citing this study linked from PubMed
Study ended by the sponsor due to meeting Study Objectives
Masri A, Aras M, Grogan M, Jacoby D, Maurer MS, Shah SJ, Witteles R, Wong PW, Ji AX, DU J, Siddhanti S, Katz L, Sinha U, Fox JC, Judge DP. Acoramidis for Trans…PubMed 40216220 ↗. 1 publication citing this study linked from PubMed
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is terminated, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Acoramidis
Amyloid Neuropathies, Familial →
Eidos Therapeutics, a BridgeBio company →