Amyloid Cardiomyopathy, Amyloidosis, Transthyretin Amyloidosis, Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
Conditions
Keywords
amyloidosis, cardiomyopathy, ATTR-CM, Amyloid, TTR
Brief summary
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
DRUGAcoramidis (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.
Sponsors
Eidos Therapeutics, a BridgeBio company
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Key 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. Key
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To assess the effect of acoramidis on cardiac function improvement by CMR in participants with ATTR-CM. | Month 36 | Change from baseline in left ventricular ejection fraction (LVEF) or left ventricular stroke volume index (LVSVi) at Month 36. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To assess the effect of acoramidis on cardiac function improvement or stabilization by CMR in participants with ATTR-CM. | Month 36 | Change from baseline in LVEF and LVSVi at Month 36 |
| To assess the effect of acoramidis on cardiac function stabilization by CMR in participants with ATTR-CM. | Month 36 | Change from baseline in LVEF and LVSVi at 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 | Month 36 | Mean change from baseline in LVEF, LVSVi, left ventricular global longitudinal strain (LVGLS) at Month 36 |
| To evaluate the effect of acoramidis on cardiac structure by CMR in participants with ATTR-CM | Month 36 | Mean change from baseline in left ventricular mass index (LVMi) at Month 36 |
| To evaluate the effect of treatment with acoramidis on cardiac amyloid burden by CMR in participants with ATTR-CM | Month 36 | Mean change from baseline in extracellular volume (ECV) at Month 36 |
Countries
United States
Contacts
CONTACTMedical Information
Outcome results
None listed