NIPH Clinical Trials Search

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

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Transthyretin amyloidosis
Date of first enrollment	20/12/2025
Target sample size	25
Countries of recruitment	Argentina,Japan,Australia,Japan,Belgium,Japan,Brazil,Japan,Canada,Japan,Denmark,Japan,France,Japan,Germany,Japan,Greece,Japan,Ireland,Japan,Italy,Japan,Malaysia,Japan,Mexico,Japan,Netherlands,Japan,Portugal,Japan,Singapore,Japan,South Korea,Japan,Spain,Japan,Sweden,Japan,Taiwan,Japan,United Kingdom,Japan,United States,Japan
Study type	Interventional
Intervention(s)	Two tablets of acoramadis 356 mg (equivalent to acoramidis HCl 400 mg) will be administered orally twice daily.

Outcome(s)

Primary Outcome	1. Time to development of ATTR (ATTR-CM or ATTR-PN, whichever occurs first; centrally adjudicated) [Time Frame: Since randomization up to approximately 7 years or until the study is declared over] a. ATTR-CM defined by biopsy or imaging-based diagnosis b. ATTR-PN defined by new signs or symptoms and biopsy-based diagnosis
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	<= 75age old
Gender	Both
Include criteria	1. Male or female >= 18 to <= 75 years of age inclusive. 2. 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. 3. Participant's age is no more than 10 years (<=10) younger than the PADO.
Exclude criteria	1. Evidence of ATTR-CM or ATTR-PN. 2. Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H). 3. Current or past treatment with other TTR modifying therapies. 4. Contraindication to or inability to undergo Cardiac magnetic resonance testing. 5. Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy. 6. Other diseases or conditions such has cancer within 3 years, untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV. 7. Major surgery within the past 3 months or planned during the next 12 months. 8. Known hypersensitivity to acoramidis.