Last updated 14 days ago
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
587 patients around the world
Available in Argentina, United States, Brazil
The AG10-501 ACT-EARLY study is a randomized, multicenter, double-blind,
placebo-controlled study of acoramidis for prevention of ATTR (with specific reference to
either its cardiomyopathic or polyneuropathic manifestations). Participants will be
stratified at randomization.
The study population will be asymptomatic carriers of a known pathogenic TTR gene
variant. A participant must be 18 to 75 inclusive years of age, and the age of the
participant must be within 10 years younger than or older than the predicted age of
disease onset (PADO) based either on family history (pedigree analysis) or, if family
history is insufficient, based on a TTR Variant Actuarial table from published
literature. For example, if PADO for a given individual is found to be 50 years, the age
of the participant must be between 40 and 75 years inclusive.
Eidos Therapeutics, a BridgeBio company
1Research sites
587Patients around the world
This study is for people with
Rare diseases
Amyloidosis
Requirements for the patient
To 75 Years
All Gender
Medical requirements
- 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 confirmed by central laboratory prior to randomization.Participant's age is no more than 10 years (≤ 10) younger than the PADO.
- Evidence of ATTR-CM or ATTR-PN.Presence of a TTR variant known to be phenotypically protective.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 as 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.
Sites
Hospital Italiano de Buenos Aires - CABA, Buenos Aires
Recruiting
SponsorEidos Therapeutics, a BridgeBio company
Study typeInterventional
Conditions
Amyloidosis
Requirements
To 75 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06563895
