Acoramidis for Amyloid Cardiomyopathy

Recruiting at 146 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

389 Participants Needed

Trial Summary

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.

What data supports the effectiveness of the drug Acoramidis (AG10) for treating amyloid cardiomyopathy?

While there is no direct data on Acoramidis (AG10), similar drugs like Tafamidis have been shown to improve outcomes in patients with transthyretin amyloid cardiomyopathy by slowing disease progression and improving physical performance. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial is testing the safety and effectiveness of acoramidis in people with a heart condition called ATTR-CM. The drug works by preventing harmful protein clumps in the heart. Participants must have completed a previous study and cannot use other specific treatments during this trial.

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Tafamidis treatment delays structural and functional changes of the left ventricle in patients with transthyretin amyloid cardiomyopathy. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Impact of Tafamidis and Optimal Background Treatment on Physical Performance in Patients With Transthyretin Amyloid Cardiomyopathy. [2022]

3. England 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. [2021]

4. New Zealand pubmed.ncbi.nlm.nih.gov

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

5. England pubmed.ncbi.nlm.nih.gov

Monitoring tafamidis treatment with quantitative SPECT/CT in transthyretin amyloid cardiomyopathy. [2023]