General Information

Age Group

Adults

Status

Recruiting

Protocol Number

NCT06563895

Background Information

To determine the efficacy of acoramidis to prevent ATTR (ATTRCM or ATTR-PN, whichever occurs first) in asymptomatic carriers with a pathogenic TTR variant who are at risk of developing ATTR but have no clinical evidence of disease at study entry.

Offered At

3300 Gallows Road                           
Falls Church, VA 22042
Principal Investigator

Sumeet Mitter, MD

Eligibility Information

  • Male or female ≥ 18 to ≤ 75 years of age inclusive.
  • Participants must have an established genotype (hetero- or homozygosity) through a medically-genetically indicated test of a TTR gene variant that is known to be pathogenic (eg, V30M/p.V50M, V122I/p.V142I, T60A/pT80Ap.T80A, or all other pathogenic TTR variants) confirmed by central laboratory prior to randomization.
  • Participant's age is no more than 10 years (≤ 10) younger than the PADO.

Ineligibility Information

  • Evidence of ATTR-CM or ATTR-PN.
  • Presence of a TTR variant known to be phenotypically protective (eg, T119M, R104H).
  • Current or past (within 3 months) treatment with patisiran, inotersen, or tafamidis.
  • Contraindication to or inability to undergo CMR testing.
  • Major organ dysfunction, including: kidney disease, liver disease, heart disease (including cardiomyopathy), neuropathy.
  • Other diseases or conditions such has untreated hyperthyroidism or hypothyroidism, type 1 diabetes, active hepatitis B or C, HIV, cancer.
  • Major surgery within the past 3 months or planned during the next 12 months