Acoramidis for Amyloid Cardiomyopathy

(AG10 Trial)

Not currently recruiting at 159 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Eidos Therapeutics, a BridgeBio company

Must not be taking: Prohibited medications

Disqualifiers: Heart transplant, CMAD, AL amyloidosis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial focuses on the safety and effects of acoramidis, a treatment for transthyretin amyloid cardiomyopathy, a heart condition caused by abnormal protein deposits. Participants will receive acoramidis, continuing from their involvement in an earlier phase of research. Ideal candidates have completed a prior 30-month study with acoramidis and continue to experience symptoms of this heart condition. As a Phase 3 trial, this study is the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications, but it mentions that participants cannot be on prohibited medications at the start of the study. It's best to discuss your current medications with the study team to see if any are prohibited.

Is there any evidence suggesting that Acoramidis (AG10) is likely to be safe for humans?

Research has shown that acoramidis (AG10) is generally safe for individuals with transthyretin amyloid cardiomyopathy. Studies have found that acoramidis is linked to fewer heart failure incidents. In previous research involving similar participants, acoramidis was well-tolerated over time, indicating its safety. Although specific side effects were not detailed, the consistent emphasis on safety in these studies is reassuring. Overall, the evidence suggests that acoramidis is a safe option for those considering joining this clinical trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Acoramidis (AG10) is unique because it targets the underlying cause of amyloid cardiomyopathy by stabilizing transthyretin, a protein that can misfold and deposit as amyloid in the heart. Unlike current treatments that primarily focus on managing symptoms or slowing disease progression, such as tafamidis, AG10 directly addresses the stability of the transthyretin protein, potentially halting the disease process more effectively. Researchers are excited because this mechanism could lead to improved heart function and quality of life for patients with this challenging condition.

What evidence suggests that Acoramidis might be an effective treatment for amyloid cardiomyopathy?

Research has shown that Acoramidis (AG10) effectively treats transthyretin amyloid cardiomyopathy (ATTR-CM). Studies found that patients taking Acoramidis experienced significantly better health outcomes compared to those on a placebo, which contains no active medicine. Specifically, Acoramidis lowered the risk of heart failure by 60% and reduced hospital visits for heart issues. Over 30 months, patients on Acoramidis experienced slower disease progression. This treatment appears to work by stabilizing a protein involved in the disease, improving heart function and increasing patient survival. ^{1 3 5 6 7}

Are You a Good Fit for This Trial?

This trial is for people who finished the AG10-301 study, understand and can sign consent forms, and agree to use effective birth control. It's not for those with certain medical conditions or sensitivities, recent heart issues or strokes, kidney problems, other clinical trials within 30 days, substance abuse history, psychiatric conditions that affect compliance, mechanical heart devices or transplants.

Inclusion Criteria

Completed 30 months of the blinded study treatment in Study AG10-301 and the Study AG10-301 Month 30 visit including assessments and procedures

I agree to use effective birth control during and 30 days after the trial.

Have the ability to understand and sign a written informed consent form, which must be obtained prior to initiation of study procedures

Exclusion Criteria

Has hemodynamic instability, that in the judgment of the Investigator, would pose too great a risk for participation in the study

Participation in another interventional clinical trial (with the exception of Study AG10-301) within 30 days prior to dosing. Participation in observational and/or registry studies should be discussed with the Medical Monitor

I am not taking any medications that are not allowed in the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants receive open-label acoramidis for long-term safety and tolerability assessment

60 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Acoramidis (AG10)

Trial Overview The trial tests Acoramidis (AG10) in patients with Transthyretin Amyloid Cardiomyopathy who completed a previous Phase 3 trial. It's an open-label extension meaning everyone knows they're getting AG10 and it focuses on long-term safety monitoring of the drug.

How Is the Trial Designed?

Group I: AG10 Experimental Treatment 1 Intervention

Open-label study all participants will receive AG10 during this study.

Who Is Running the Clinical Trial?

Eidos Therapeutics, a BridgeBio company

Lead Sponsor

Trials

12

Recruited

2,400+

Published Research Related to This Trial

Tafamidis significantly reduces all-cause mortality and cardiovascular-related hospitalizations in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), with a median follow-up of 51 months from the ATTR-ACT trial and its long-term extension study.

The 80 mg dose of tafamidis showed a greater survival benefit compared to the 20 mg dose, while both doses had a safety profile comparable to placebo, suggesting that 80 mg is the optimal dose for treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of tafamidis doses in the Tafamidis in Transthyretin Cardiomyopathy Clinical Trial (ATTR-ACT) and long-term extension study. Damy, T., Garcia-Pavia, P., Hanna, M., et al.[2021]

Tafamidis is an effective treatment for transthyretin amyloid cardiomyopathy (ATTR-CM), significantly reducing all-cause mortality and cardiovascular-related hospitalizations compared to placebo in the phase III ATTR-ACT trial, which involved patients over a 30-month period.

The treatment was well tolerated with a safety profile similar to placebo, making it a suitable long-term option for patients, and it provides a disease-modifying therapy rather than just symptom management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tafamidis: A Review in Transthyretin Amyloid Cardiomyopathy. Lamb, YN.[2021]

Citations

1. nejm.org nejm.org/doi/full/10.1056/NEJMoa2305434

Efficacy and Safety of Acoramidis in Transthyretin Amyloid ... In patients with transthyretin amyloid cardiomyopathy, the receipt of acoramidis resulted in a significantly better four-step primary hierarchical outcome.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40074465/

Efficacy of Acoramidis on All-Cause Mortality and ... In participants with ATTR-CM, acoramidis reduced the composite of ACM or first CVH vs placebo, with an early effect driven by a reduction in CVH.

3. ahajournals.org ahajournals.org/doi/10.1161/CIRCULATIONAHA.124.072771

Long-Term Efficacy and Safety of Acoramidis in ATTR-CM In patients with ATTR-CM, 30 months of treatment with acoramidis reduced the rate of disease progression as assessed by NT-proBNP and better ...

4. jacc.org jacc.org/doi/10.1016/j.jacc.2025.03.542

Early Increase in Serum Transthyretin by Acoramidis ... Median survival in untreated patients typically ranges between 25 to 41 months, with longer survival in patients with wild-type transthyretin ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S1071916424006389

Acoramidis For The Treatment Of Amyloid Cardiomyopathy It revealed that Acoramidis reduced the risk of heart failure by 60% in patients taking the drug compared to the placebo. It is important to note that the ...

6. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39556242/

Long-Term Efficacy and Safety of Acoramidis in ATTR-CM We report the efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.

7. clinicaltrials.gov clinicaltrials.gov/study/NCT04988386

NCT04988386 | Open-Label Safety Study of Acoramidis ... The primary objective of this prospective, multi-center, open-label study is to evaluate the long-term safety and tolerability of acoramidis in patients ...