Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis

Purpose

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

Conditions

  • Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
  • Amyloidosis
  • Amyloid Cardiomyopathy
  • Transthyretin Amyloidosis

Eligibility

Eligible Ages
Between 18 Years and 80 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 4
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Acoramidis (AG10) 		Open-label study where all participants will receive acoramidis (AG10) during the study
  • 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.

Recruiting Locations

Washington University in St. Louis and nearby locations

Washington University in St. Louis
St Louis, Missouri 63110

More Details

NCT ID
NCT07695701
Status
Recruiting
Sponsor
Eidos Therapeutics, a BridgeBio company

Study Contact

Medical Information
1-844-550-2246
medinfo@bridgebio.com

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.