Acoramidis for Amyloidosis

Recruiting at 69 trial locations

Overseen By Medical Information

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Eidos Therapeutics, a BridgeBio company

Must not be taking: TTR modifying therapies

Disqualifiers: ATTR-CM, ATTR-PN, Major organ dysfunction, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 7 years

582 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests acoramidis, a new drug that might prevent or delay transthyretin amyloidosis (ATTR). ATTR can lead to heart and nerve issues when a protein breaks down and forms harmful plaques. The study focuses on individuals with a specific gene defect who haven't shown symptoms, aiming to determine if early treatment can prevent disease development. Those with a known defective TTR gene but no symptoms of heart or nerve problems might qualify for this trial. Participants will receive either acoramidis or a placebo to compare results. As a Phase 3 trial, this study is the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are currently or have previously been treated with other TTR modifying therapies, you would not be eligible to participate.

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

Research shows that acoramidis is generally well-tolerated by people with transthyretin amyloid cardiomyopathy (ATTR-CM). Earlier studies did not raise any major safety concerns about acoramidis. Participants who took acoramidis experienced effects similar to those who took a placebo, suggesting the drug is safe for most people. While some mild side effects occurred, serious side effects were rare.

Acoramidis stabilizes a protein in the blood, helping to prevent the formation of harmful deposits. This action aims to improve heart function and overall health in people with amyloidosis. So far, the data show positive results in patients, making acoramidis a promising option for those at risk of developing symptoms of amyloidosis. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Acoramidis is unique because it targets transthyretin, a protein involved in the buildup of amyloid deposits in the body, which is the root problem in amyloidosis. Unlike current treatments that might only stabilize or slow disease progression, acoramidis is designed to directly stabilize transthyretin, potentially preventing these harmful deposits from forming in the first place. Researchers are excited about acoramidis because this approach could offer more comprehensive management of amyloidosis, improving patients' quality of life by addressing the disease at its source.

What evidence suggests that acoramidis might be an effective treatment for ATTR?

Research has shown that acoramidis, which participants in this trial may receive, can benefit people with transthyretin amyloidosis, particularly in advanced stages affecting the heart, known as transthyretin amyloid cardiomyopathy (ATTR-CM). Studies have found that acoramidis stabilizes the TTR protein, preventing it from breaking down and forming harmful clumps. In one study, patients taking acoramidis walked farther in a 6-minute test compared to those on a placebo, indicating improved physical function. Another study demonstrated that acoramidis reduced heart-related problems within the first month of treatment, suggesting it might protect the heart. Overall, these findings suggest that acoramidis could help delay or prevent symptoms in people at risk for heart and nerve issues from this condition. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

This trial is for men and women aged 18 to 75 who carry a pathogenic TTR gene variant but have no symptoms of transthyretin amyloidosis, which can lead to heart failure or nerve problems. They must be within 10 years of the age when symptoms typically start.

Inclusion Criteria

I am within 10 years of the PADO's age.

My genetic test shows I have a TTR gene variant linked to disease.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive acoramidis or placebo to prevent or delay the development of ATTR-CM or ATTR-PN

Up to 7 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Acoramidis

Trial Overview The study tests if acoramidis, an experimental drug that stabilizes TTR protein in blood, can prevent or delay heart disease (ATTR-CM) or nerve damage (ATTR-PN) in people with genetic risk but no symptoms yet. Participants will either receive acoramidis or a placebo tablet.

How Is the Trial Designed?

Group I: acoramidis Experimental Treatment 1 Intervention

Participants will receive acoramidis 712 mg orally BID (which is equivalent to 800 mg acoramidis HCl BID)

Group II: Placebo Placebo Group 1 Intervention

Subjects will receive placebo to match twice daily

Acoramidis is already approved in United States, European Union for the following indications:

Approved in United States as Attruby for:

• Cardiomyopathy of transthyretin-mediated amyloidosis (ATTR-CM)

Approved in European Union as Attruby for:

• Transthyretin-related hereditary amyloidosis

Who Is Running the Clinical Trial?

Eidos Therapeutics, a BridgeBio company

Lead Sponsor

Trials

12

Recruited

2,400+

Published Research Related to This Trial

In patients with transthyretin amyloidosis with polyneuropathy (ATTR-PN), long-term treatment with tafamidis showed a significant survival benefit, with 85.9% of Val30Met patients alive after 9 years when treated continuously with tafamidis.

For non-Val30Met patients, 75.9% were alive after 8 years, indicating that tafamidis may also be beneficial for this group, although survival rates were lower compared to Val30Met patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Evaluation of Mortality During Long-Term Treatment with Tafamidis for Transthyretin Amyloidosis with Polyneuropathy: Clinical Trial Results up to 8.5 Years. Merlini, G., Coelho, T., Waddington Cruz, M., et al.[2020]

Tafamidis is an effective treatment for transthyretin amyloidosis (ATTR), approved for use in over 40 countries, and has shown to stabilize the disease's progression in adults with early-stage symptomatic ATTR polyneuropathy and cardiomyopathy.

A phase 1 study involving 30 healthy volunteers demonstrated that the new tafamidis 61-mg free acid capsules are bioequivalent to the standard 80-mg tafamidis meglumine capsules, with both formulations showing a good safety and tolerability profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The Bioequivalence of Tafamidis 61-mg Free Acid Capsules and Tafamidis Meglumine 4 × 20-mg Capsules in Healthy Volunteers. Lockwood, PA., Le, VH., O'Gorman, MT., et al.[2021]

In a study of 82 Japanese patients with wild-type transthyretin cardiac amyloidosis (ATTRwt CA), tafamidis was safely administered to 38 patients, showing no discontinuation due to adverse events and a high 1-year survival rate of 92%.

Patients treated with tafamidis maintained stable disease markers over 12-18 months, indicating that tafamidis may effectively preserve heart function and disease status in the short term for selected patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Early Experience of Tafamidis Treatment in Japanese Patients With Wild-Type Transthyretin Cardiac Amyloidosis From the Kochi Amyloidosis Cohort. Ochi, Y., Kubo, T., Baba, Y., et al.[2022]

In a phase II study involving 21 patients with non-Val30Met transthyretin amyloidosis, tafamidis 20 mg once daily effectively stabilized TTR levels in 94.7% of patients by Week 6, and 100% at Months 6 and 12.

While some patients experienced worsening neurological function, tafamidis was well tolerated and did not lead to significant declines in quality of life or other health markers over the 12-month treatment period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. Merlini, G., Planté-Bordeneuve, V., Judge, DP., et al.[2021]

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]

Citations

1. 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.

2. 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 ...

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

Early Increase in Serum Transthyretin by Acoramidis ... A significantly better outcome was observed in ATTRibute-CM favoring acoramidis compared with placebo in the hierarchical endpoint that included ...

4. attrubyhcp.com attrubyhcp.com/clinical-data

Attruby Clinical Data & Study Findings - Latest Information Acoramidis improves clinical outcomes in patients with transthyretin amyloid cardiomyopathy: post hoc recurrent event analyses of ATTRibute-CM. Poster presented ...

5. investor.bridgebio.com investor.bridgebio.com/news/news-details/2025/Acoramidis-Begins-to-Reduce-Cumulative-Cardiovascular-Outcomes-Within-the-First-Month-of-Treatment-in-Patients-with-ATTR-CM/default.aspx

Acoramidis Begins to Reduce Cumulative Cardiovascular ... By Month 1, numerically fewer cumulative events were observed with acoramidis compared to placebo - Acoramidis significantly reduced the ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/216540s000lbl.pdf

Attruby - accessdata.fda.gov Risk Summary. Available data with acoramidis use in pregnant women are insufficient to establish a drug associated risk of major birth defects, miscarriage ...