Amyloid CardiomyopathyPhase 2NCT03536767
Open-Label Study of AG10 in Patients With Cardiomyopathy
A Phase 2 study testing Acoramidis in people with Amyloid Cardiomyopathy, run by Eidos Therapeutics, a BridgeBio company at 9 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyloid Cardiomyopathy
- What is being tested?
- Acoramidis
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 47 participants
- Where
- 9 sitesUnited States
- Study dates
- 8 Aug 2018to 15 Sep 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Completed the study AG10-201.
- Willing and able to comply with the study medication regimen and all other study requirements based on screening laboratory results from study AG10-201.
- The ability to understand and provide informed consent, which must be obtained prior to initiation of study participation based on screening laboratory results from study AG10-201.
- Male patients and female patients of childbearing potential who engage in heterosexual intercourse must agree to use effective method(s) of contraception.
Who cannot join
- Hemodynamic instability that would pose too great a risk to the subject based on screening laboratory results from study AG10-201.
- Abnormalities in clinical laboratory tests that would pose too great a risk to the subject based on screening laboratory results from study AG10-201.
- Any clinically significant ongoing medical condition or any laboratory abnormality or condition that could adversely affect the safety of the patient.
- Known hypersensitivity to study drug (acoramidis), its metabolites, or formulation excipients.
- Likely to undergo heart transplantation or placement of a mechanical circulatory device within the next year.
- Receiving current treatment with diflunisal, tafamidis, green tea, doxycycline, TUDCA/Ursodiol, patisiran, inotersen or any other investigational ATTR agent within 14 days or 5 half-lives of the prior investigational agent (whichever is longer) prior to dosing with study drug.
- Females who are pregnant or breastfeeding. Lactating females must agree to discontinue nursing before the study drug is administered. Negative urine pregnancy test at each visit is required for female patients of childbearing potential.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Medical University of South Carolina
Charleston, South Carolina, 29425
Northwestern University
Chicago, Illinois, 60611
Yale University
New Haven, Connecticut, 06520
Columbia University
New York, New York, 10032
Oregon Health & Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55905
University of California San Francisco
San Francisco, California, 94143
Stanford University
Stanford, California, 94305
About this study
Official title: An Open-Label Extension and Safety Monitoring Study of Patients With Symptomatic Transthyretin Cardiomyopathy Who Have Completed the Phase II Study AG10-201
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.
Read the detailed description
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.
- Conditions
- Amyloid Cardiomyopathy
- Being tested
- Acoramidis
- Keywords
- Amyloidosis, ATTR-CM, Transthyretin, Amyloid, TTR, Cardiomyopathy
- Medical subject headings
- Amyloid Neuropathies, Familial, Amyloidosis, Alzheimer Disease, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AcoramidisDrug
Also called AG10
Acoramidis 800 mg twice daily, oral administration
| Study group | Role | What they receive |
|---|---|---|
| Open-Label | Experimental | Participants received open-label acoramidis 800 mg orally twice daily. Receives: Drug: Acoramidis |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From the first dose of study drug until 30 days after the last dose of study drug. (Maximum exposure reported 84.1 months).
Secondary outcomes (4)
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
Measured: 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).
Measured: 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.
Measured: 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.
Measured: Baseline, Day 45, and Month 78
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2018
- Study start8 Aug 2018
- Primary completion15 Sep 2025
- Study completion15 Sep 2025
Why it stopped: Study ended by the sponsor due to meeting Study Objectives
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Amyloid CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 47 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eidos Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03536767
- Sponsor's protocol ID
- AG10-202
- Status last verified
- Sep 2026
- First submitted
- 11 May 2018
- Last updated
- 14 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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 Transthyretin Amyloid Cardiomyopathy: Open-Label Extension Study Long-Term Follow-Up. J Card Fail. 2025 Aug;31(8):1354-1359. doi: 10.1016/j.cardfail.2025.03.017. Epub 2025 Apr 9. No abstract available.
PubMed 40216220Testani JM, Judge DP, Borlaug BA, Cherney D, Cox ZL, Gillmore JD, Lewis JB, Adler SH, Cao X, Castano A, Fox JC, Katz L, Mathur V, Xiong K, Butler J, Masri A. Effects of Acoramidis on Kidney Function in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2026 Jul 2:e014656. doi: 10.1161/CIRCHEARTFAILURE.126.014656. Online ahead of print.
PubMed 42389794
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension and Safety Monitoring Study of Patients With Symptomatic Transthyretin Cardiomyopathy Who Have Completed the Phase II Study AG10-201
- ClinicalTrials.gov ID
- NCT03536767
- Acronym / sponsor ID
- AG10-202
- Conditions
- Amyloid Cardiomyopathy
- Keywords
- Amyloidosis, ATTR-CM, Transthyretin, Amyloid, TTR, Cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study ended by the sponsor due to meeting Study Objectives
- Enrolment
- 47 participants (actual)
- Lead sponsor
- Eidos Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 8 Aug 2018 (actual)
- Primary completion
- 15 Sep 2025 (actual)
- Study completion
- 15 Sep 2025 (actual)
- First posted
- 25 May 2018 (actual)
- Results
- Posted 14 Oct 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-Label | Experimental | Drug: AcoramidisParticipants received open-label acoramidis 800 mg orally twice daily. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Completed the study AG10-201.
- Willing and able to comply with the study medication regimen and all other study requirements based on screening laboratory results from study AG10-201.
- The ability to understand and provide informed consent, which must be obtained prior to initiation of study participation based on screening laboratory results from study AG10-201.
- Male patients and female patients of childbearing potential who engage in heterosexual intercourse must agree to use effective method(s) of contraception.
Key exclusion criteria (7 listed)
- Hemodynamic instability that would pose too great a risk to the subject based on screening laboratory results from study AG10-201.
- Abnormalities in clinical laboratory tests that would pose too great a risk to the subject based on screening laboratory results from study AG10-201.
- Any clinically significant ongoing medical condition or any laboratory abnormality or condition that could adversely affect the safety of the patient.
- Known hypersensitivity to study drug (acoramidis), its metabolites, or formulation excipients.
- Likely to undergo heart transplantation or placement of a mechanical circulatory device within the next year.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AcoramidisAlso: AG10Acoramidis 800 mg twice daily, oral administration | Drug | Open-Label |
Primary outcome
Overall Summary of Treatment-Emergent Adverse Events.
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).
Safety and tolerability were assessed by treatment-emergent adverse events (TEAEs), serious TEAEs, adverse events leading to treatment discontinuation.
4 secondary outcomes
Acoramidis Pharmacodynamic Assessments of TTR Stabilization by Fluorescent Probe Exclusion Assay
Time frame: Day 45 and Month 78
Acoramidis Pharmacodynamic Assessments: Prealbumin
Time frame: Baseline, Day 45, and Month 78
Plasma Concentrations of Acoramidis
Time frame: Baseline, Day 45, and Month 78
Plasma Concentrations of Acoramidis Acylglucuronide
Time frame: Baseline, Day 45, and Month 78
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 40216220Masri 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 Transthyretin Amyloid Cardiomyopathy: Open-Label Extension Study Long-Term Follow-Up. J Card Fail. 2025 Aug;31(8):1354-1359. doi: 10.1016/j.cardfail.2025.03.017. Epub 2025 Apr 9. No abstract available.
- Derived from this study: PubMed 42389794Testani JM, Judge DP, Borlaug BA, Cherney D, Cox ZL, Gillmore JD, Lewis JB, Adler SH, Cao X, Castano A, Fox JC, Katz L, Mathur V, Xiong K, Butler J, Masri A. Effects of Acoramidis on Kidney Function in Transthyretin Amyloid Cardiomyopathy. Circ Heart Fail. 2026 Jul 2:e014656. doi: 10.1161/CIRCHEARTFAILURE.126.014656. Online ahead of print.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Acoramidis in people with Amyloid Cardiomyopathy, run by Eidos Therapeutics, a BridgeBio company at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 14 Oct 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Summary of Treatment-Emergent Adverse Events. (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).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03536767, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026; AzTrials last refreshed its copy on 7 Nov 2026.