AG10 for Cardiomyopathy

Not currently recruiting at 8 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Eidos Therapeutics, a BridgeBio company

Must not be taking: Diflunisal, Tafamidis, others

Disqualifiers: Hemodynamic instability, Heart transplant, others

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

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

Compensation: Varies

Number of Visits: 21

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines a treatment called AG10 for individuals with cardiomyopathy, a condition where the heart struggles to pump blood effectively. Researchers aim to determine if AG10 is safe and effective alongside other heart failure treatments. The trial is open-label, so all participants will receive AG10 and know they are taking it. Those who participated in the previous AG10 study and maintain a stable heart condition may be suitable candidates. Participants must also adhere to the study rules and use effective contraception if applicable. As a Phase 2 trial, this research measures AG10's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant medical advancements.

Will I have to stop taking my current medications?

You may need to stop taking certain medications like diflunisal, tafamidis, green tea, doxycycline, TUDCA/Ursodiol, patisiran, inotersen, or any other investigational ATTR agent at least 14 days before starting the study drug. The trial does not specify stopping other medications, but it's best to discuss with the study team.

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

Research has shown that AG10, also known as acoramidis, is generally safe for individuals with heart conditions. In earlier studies, patients with transthyretin amyloid cardiomyopathy who took AG10 experienced positive outcomes over time. More than half of these patients lived for an extended period, suggesting its safety for long-term use.

Some studies have monitored AG10's safety over time and found that patients continued to respond well to the treatment. While any medication can have side effects, current evidence suggests that AG10's safety is promising. This supports its use in treating heart disease for those already stable on heart failure therapy. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for cardiomyopathy, which often focus on managing symptoms with medications like beta-blockers or ACE inhibitors, AG10 is unique because it targets the root cause of the disease. AG10 works by stabilizing the transthyretin protein, preventing it from misfolding and forming amyloid deposits that can damage heart tissue. Researchers are excited about AG10 because it offers a novel approach that could potentially slow or halt the progression of cardiomyopathy, rather than just alleviating symptoms. This could lead to improved outcomes and quality of life for patients with this challenging condition.

What evidence suggests that AG10 might be an effective treatment for cardiomyopathy?

Research has shown that AG10, also known as acoramidis, holds promise for treating transthyretin amyloid cardiomyopathy (ATTR-CM). Studies have found that acoramidis improves patients' performance in physical activities, such as walking longer distances in a 6-minute test. It also reduces the likelihood of heart-related hospital visits and lowers the risk of death compared to a placebo. Overall, these findings suggest that AG10 can help manage symptoms and enhance the quality of life for individuals with this condition. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

This trial is for patients with cardiomyopathy who have completed a prior AG10 study. Participants must be stable enough to follow the treatment plan and agree to use contraception if necessary. Those at risk of heart transplant, on certain other treatments, or with significant health risks are excluded.

Inclusion Criteria

I understand the study and have given my consent to participate.

Willing and able to comply with the study medication regimen and all study requirements

Completed participation in study AG10-201

Exclusion Criteria

Your blood tests show dangerous abnormalities that could be risky for you.

You are allergic to the study drug AG10 or any of its ingredients.

You might need a heart transplant or a mechanical heart device in the next year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-Label Extension

Participants receive AG10 on a background of stable heart failure therapy, with visits at Day 14, Day 45, 3 Months, and every 3 months thereafter until Month 54

54 months

Visits at Day 14, Day 45, 3 Months, and every 3 months until Month 54

Long-term Follow-up

Participants are monitored for safety and effectiveness after treatment, with visits every 6 months after Month 54

6 months

Visits every 6 months

Final Follow-up

One follow-up visit approximately 30 days after the last dose

1 month

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• AG10

Trial Overview The study tests the long-term safety and effects of AG10 in individuals with cardiomyopathy while they continue their usual heart failure therapy. It's an open-label trial, meaning both researchers and participants know what treatment is being given.

How Is the Trial Designed?

Group I: Open-Label Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Eidos Therapeutics, a BridgeBio company

Lead Sponsor

Trials

12

Recruited

2,400+

Citations

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

Long-Term Efficacy and Safety of Acoramidis in ATTR-CM Transthyretin stabilization by AG10 in ... Acoramidis improves clinical outcomes in transthyretin amyloid cardiomyopathy patients.

2. 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.

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

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

4. 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.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03860935

Study Details | NCT03860935 | Efficacy and Safety of AG10 ... The primary outcomes of the study are: The impact of acoramidis versus placebo on the change in distance walked on the 6 minute walk test (6MWT) after 12 months ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S1071916423006280

Long-term Safety And Tolerability Of Acoramidis (AG10) In ... In this ongoing open-label study, at least 53% of patients with ATTR-CM and NYHA Class II or III at entry to the phase 2 trial have survived for a median follow ...