Trial purpose
Cancer treatment
Tumor type
Haematological
Age
18 - 90
Trial acronym
ATTRibute-CM
Clinical summary
Summary
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed condition believed to affect more than 400,000 people worldwide. In ATTR-CM, the accumulation of transthyretin (TTR) amyloid results in thickening and stiffening of the heart, which often leads to heart failure or even death. In this study we are researching the investigational drug acoramidis 800 mg administered orally twice a day. Through the study, we want to evaluate the efficacy and safety of acoramidis in patients with ATTR-CM versus placebo. This is a 30 month, randomized, double-blind, placebo-controlled study. This means that, during the 30 month study, investigators conducting the research and study participants will not know whether the study participant is receiving acoramidis or placebo. In the Experimental Arm, participants will receive acoramidis 800 mg twice daily (orally). 6MWT primary outcome will be assessed at the end of 12 months. The frequencies of deaths, cardiovascular-related hospitalizations, and change in distance walked on the 6MWT will be assessed after 30 months of treatment. In the Placebo Arm, subjects will receive a placebo twice daily (orally). Subjects will receive placebo to match twice daily. 6MWT primary outcome will be assessed at the end of 12 months. The frequencies of deaths, cardiovascular-related hospitalizations, and change in distance walked on the 6MWT will be assessed after 30 months of treatment.
Conditions
This trial is treating people with Transthyretin Amyloid Cardiomyopathy
Eligibility
Inclusion
- Have an established diagnosis of ATTR-CM with either wild-type TTR or variant TTR genotype
- Have a history of heart failure evidenced by at least one prior hospitalization for heart failure or clinical evidence of heart failure without prior heart failure hospitalization manifested by signs or symptoms of volume overload or elevated intracardiac pressures or heart failure symptoms that required or require ongoing treatment with a diuretic.
- New York Heart Association (NYHA) Class I-III symptoms due to ATTR cardiomyopathy.
- On stable doses of cardiovascular medical therapy
- Completed ≥150 m on the 6MWT on 2 tests that are within 15% of total distance walked prior to randomization
- Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL at screening
- Have left ventricular wall (interventricular septum or left ventricular posterior wall) thickness ≥12 mm
Exclusion
- Had acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke or transient ischemic attack within 90 days prior to screening
- Has hemodynamic instability
- Likely to undergo heart transplantation within a year of screening
- Confirmed diagnosis of primary (light chain) amyloidosis
- Biomarkers of myocardial wall stress, NT-proBNP level ≥8500 pg/mL at screening
- Measure of kidney function, eGFR by MDRD formula <15 mL/min/1.73 m2
- Current treatment with marketed drug products and other investigational agents for the treatment of ATTR-CM
- Current treatment with calcium channel blockers with conduction system effects (e.g. verapamil, diltiazem). The use of dihydropyridine calcium channel blockers is allowed. The use of digitalis will only be allowed if required for management of atrial fibrillation with rapid ventricular response
Exclusion
- You have certain types of non-cancer medical conditions.
- You have had certain treatments, surgical procedures or drugs.
Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.
More information
Trial Identifiers
Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:
Trial sponsor
Eidos Therapeutics, a BridgeBio company
Scientific Title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of the Efficacy and Safety of AG10 in Subjects With Symptomatic Transthyretin Amyloid Cardiomyopathy (ATTRibute-CM Trial)
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