Preventing transthyretin amyloidosis in young carriers of a genetic variant

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study investigates the use of acoramidis to prevent transthyretin amyloidosis (ATTR) in asymptomatic carriers of pathogenic TTR gene variants. It is a randomized, multicenter, double-blind, placebo-controlled trial that aims to assess the efficacy of the treatment in preventing the onset of cardiomyopathic or polyneuropathic manifestations of the disease. Participants aged 18 to 75, who are carriers of a known pathogenic TTR variant, will be enrolled based on their predicted age of disease onset. The study will compare the effects of acoramidis against a placebo over a specified period.

Why it matters

Who can join: the full rules

Key Inclusion Criteria:

* 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.

Key Exclusion Criteria:

* 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.

Where it's running

• University of California, San Diego (UCSD) - Medical Center
  La Jolla, California, United States Recruiting
• University of California, Los Angeles (UCLA) - David Geffen School of Medicine
  Los Angeles, California, United States Recruiting
• University of California, San Francisco (UCSF)
  San Francisco, California, United States Recruiting
• Stanford University
  Stanford, California, United States Recruiting
• University of Colorado Anschutz
  Aurora, Colorado, United States Recruiting
• Yale University School of Medicine - Section of Cardiology
  New Haven, Connecticut, United States Recruiting

+54 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Medical Information
• Email: medinfo@eidostx.com
• Phone: 1-844-550-2246

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Acoramidis for ATTR-CM: Cardiac Imaging Study

  This study tests whether acoramidis can improve heart structure and function in people with transthyretin amyloid cardiomyopathy.

  Recruiting Phase 4 Eidos Therapeutics, a BridgeBio company · St Louis, Missouri · NCT07695701

• Natural history of PKP2-related arrhythmogenic cardiomyopathy

  This project will collect past and ongoing health information to see how PKP2-related arrhythmogenic cardiomyopathy progresses in people managed with standard care.

  Recruiting at 2 sites Rocket Pharmaceuticals Inc. · Boston, Massachusetts and 2 other locations · NCT06644742

• Evaluating a non-invasive method for cardiac resynchronization therapy

  This study tests a new non-invasive device to see how well cardiac resynchronization therapy works for people with heart failure and left bundle branch block during their treatment.

  Recruiting Oslo University Hospital · Oslo · NCT05868616

• Evaluating Iodine-124 Evuzumitide in Patients with Systemic Amyloidosis

  This study is testing how a new imaging agent called iodine-124 evuzumitide works in people with systemic amyloidosis, especially those with heart issues, to see how it affects their organs and overall health.

  Recruiting Phase 2 Oregon Health and Science University · Portland, Oregon · NCT05758493

• Evaluating the prevalence of transthyretin amyloidosis in heart failure patients

  This study is trying to find out how common transthyretin amyloidosis is in older heart failure patients to help catch it early for better treatment.

  Recruiting Massachusetts General Hospital · Boston, Massachusetts · NCT05577819

• Identifying Risk Factors for Heart Toxicity from Immune Checkpoint Inhibitors

  This study is trying to find out what factors might increase the risk of heart problems in people receiving immune checkpoint inhibitors for cancer treatment.

  Recruiting Groupe Hospitalier Pitie-Salpetriere · Paris · NCT04294771

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.