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Recruiting NCT07695701

Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis

Phase IV Interventional Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM) Amyloidosis Amyloid Cardiomyopathy Transthyretin Amyloidosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Acoramidis (AG10) oral tablet.
Who it may be relevant to
Registry conditions: Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis. Basic parameters: 18 years — 80 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-Label Study to Investigate the Long-Term Effects of Acoramidis on Cardiac Function, Structure and Cardiac Amyloid Burden in Participants With Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Overview

The study aims to evaluate the effect of acoramidis on cardiac functional and structural improvement in participants with ATTR-CM.

Detailed description

Study AG10-404 is an open-label study to investigate the long-term effects of acoramidis on cardiac functional and structural improvement, and the effects on cardiac amyloid burden in participants with ATTR-CM using two types of imaging, cardiac MRI and echocardiograms to track changes in heart structure and amyloid buildup over time.

Up to approximately 150 participants (ages 18-80) will be enrolled. Participants will receive study drug (712 mg equivalent to 800 mg of acoramidis hydrochloride) orally twice daily from Day 1 through Month 36. The study duration for an individual participant is approximately 38 months, including up to 45 days for screening, 36 months for open-label treatment and 1 month follow-up after the last dose of study drug. In-clinic visits will occur at Day 1, Day 28, Month 6, and every 6 months up to Month 36. Phone visits will occur at Month 3 and every 6 months thereafter up to Month 33. Participants will be followed throughout their participation in this study and for 30 days (+7 days) after the last dose of study drug.

Interventions

  • Drug Acoramidis (AG10) oral tablet
    Study drug is defined as acoramidis 712 mg (equivalent to 800 mg acoramidis hydrochloride) taken orally twice daily for 36 months of open label treatment.

Primary outcome measures

  • To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM. [Time frame: Month 36]
Secondary outcome measures (5)
  • To assess the effect of acoramidis on cardiac function improvement or stabilization by CMR in participants with ATTR-CM. [Time frame: Month 36]
  • To assess the effect of acoramidis on cardiac function stabilization by CMR in participants with ATTR-CM. [Time frame: Month 36]
  • To evaluate the effect of treatment with acoramidis on individual parameters of left ventricular systolic function by CMR in participants with ATTR-CM [Time frame: Month 36]
  • To evaluate the effect of acoramidis on cardiac structure by CMR in participants with ATTR-CM [Time frame: Month 36]
  • To evaluate the effect of treatment with acoramidis on cardiac amyloid burden by CMR in participants with ATTR-CM [Time frame: Month 36]

Eligibility criteria

Inclusion criteria

  • Participants aged 18 to 80
  • Confirmed diagnosis of ATTR-CM requiring targeted therapy
  • At screening: New York Heart Association (NYHA) Class I/II; estimated glomerular filtration rate (eGFR) ≥40 mL/min/1.73m2; left ventricular ejection fraction (LVEF) >35%; left ventricular (LV) wall thickness ≥12 mm.
  • Heart failure condition has been clinically stable for at least 6 weeks prior to screening.

Exclusion criteria

  • Current or planned implantation of a cardiac implantable electronic device (eg, pacemaker, implantable cardiac defibrillator).
  • Acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening.
  • Hemodynamic instability.
  • Likely to undergo heart transplantation within 12 months from screening.
  • Confirmed diagnosis of AL amyloidosis at screening.
  • At screening: NYHA Class III/IV; National Amyloidosis Center (NAC) Stage 3.
  • Prior or current treatment with acoramidis.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Washington University in St. Louis — St Louis

Identifiers

NCT: NCT07695701 · AG10-404 · 2026-526068-19-00 · U1111-1339-5700

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗