Acoramidis for Amyloidosis

Phase 3

Recruiting

Research Sponsored by Eidos Therapeutics, a BridgeBio company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Key

* Participants must have an established genotype (hetero- or homozygosity) of a TTR gene variant that is known to be pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or any other pathogenic TTR variant(s)) confirmed by central laboratory prior to randomization.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up since randomization up to approximately 7 years or until the study is declared over

Awards & highlights

Pivotal Trial

Summary

This trial is testing a new drug called acoramidis in people who have a genetic defect that causes a protein called transthyretin (TTR) to fall apart and form harmful amyloid

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects are not detailed here, but as with any medication, there may be risks involved. Typically these could include allergic reactions, gastrointestinal issues, fatigue among others depending on individual response.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

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I am between 18 and 75 years old.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ since randomization up to approximately 7 years or until the study is declared over

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~since randomization up to approximately 7 years or until the study is declared over

This trial's timeline: 3 weeks for screening, Varies for treatment, and since randomization up to approximately 7 years or until the study is declared over for reporting.