NCT06563895 · Phase 3 · RECRUITING
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
This trial tests whether a daily pill called acoramidis can delay or prevent heart or nerve damage in people who carry an inherited TTR gene variant but have no symptoms yet. It is compared with a placebo. It is a Phase 3 trial: a large, late-stage test of whether it works and is safe, not yet proven or approved for this use.
You may qualify if
- Male or female ≥ 18 to ≤ 75 years of age inclusive.
- Participants must have an established genotype (hetero- or homozygosity) through a medically-indicated genetic test of a TTR gene variant that is known to be pathogenic or likely pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/p.T80A, or all other pathogenic TTR variants).
- Participant's age is within 10 years younger than or older than PADO.
You're excluded if
- Evidence of ATTR-CM or ATTR-PN.
- Current or past (within last 1 to 12 months, depending on specific agent) 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 5 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.
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2026-08-04