Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

Launched by EIDOS THERAPEUTICS, A BRIDGEBIO COMPANY · Aug 18, 2024

Keywords

ClinConnect Summary

The ACT-EARLY Study is a clinical trial investigating a new experimental drug called acoramidis, aimed at preventing or delaying the onset of a serious condition known as transthyretin amyloidosis (ATTR) in individuals who carry a faulty gene responsible for this disease but have not yet shown any symptoms. ATTR occurs when a protein called transthyretin (TTR) breaks down and forms sticky plaques that can build up in various organs, particularly the heart and nerves, leading to serious health issues like heart failure and nerve problems. This trial is specifically looking at people aged 18 to 75 who have a known pathogenic TTR gene variant but do not currently exhibit any signs of the disease.

Eligible participants will receive treatment with acoramidis, which is designed to stabilize the TTR protein in the blood to help prevent the harmful buildup of amyloid. Throughout the trial, participants will be monitored closely to see if the treatment helps delay or prevent the development of heart or nerve problems associated with ATTR. It's important to note that individuals with existing symptoms of ATTR or certain other health conditions will not be able to join the study. If you meet the eligibility criteria, participating in this study could be an important step in understanding how to protect against the onset of ATTR in those at risk.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • Male or female ≥ 18 to ≤ 75 years of age inclusive.
• • 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.
• • Participant's age is no more than 10 years (≤ 10) younger than the PADO.
• Key Exclusion Criteria:
• • Evidence of ATTR-CM or ATTR-PN.
• • Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H).
• • Current or past treatment with other TTR modifying therapies.
• • Contraindication to or inability to undergo Cardiac magnetic resonance testing.
• • Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy
• • Other diseases or conditions such has cancer within 3 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV.
• • Major surgery within the past 3 months or planned during the next 12 months.
• • Known hypersensitivity to acoramidis.