Transthyretin amyloidosis (ATTR) is a condition where a protein called transthyretin (TTR) breaks down and forms a sticky substance called amyloid. This amyloid can build up in organs, causing damage. There are two types: wild-type (ATTRwt), which happens with age, and variant (ATTRv), which is inherited. Acoramidis is a new experimental drug designed to prevent TTR from breaking down and forming amyloid. This study aims to see if acoramidis can stop or delay heart and nerve problems in people with the ATTRv type before symptoms appear.
- The study lasts until disease development or 24 months, whichever comes first, with several clinic visits.
- Participants must be 18-75 years old and have a known defective TTR gene.
- Exclusion includes previous TTR treatment, specific health conditions, and recent major surgery.
Participants will be randomly assigned to receive either acoramidis or a placebo, without knowing which they are getting (double-blind). This trial is conducted at multiple centers. It’s important for participants to not have current symptoms or organ issues related to ATTR. If you meet the criteria, participating in this study could help delay or prevent the disease and contribute to vital scientific research.