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Purpose

Phase 3 efficacy and safety study to evaluate AG10 800 mg compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Conditions

Eligibility

Eligible Ages
Between 18 Years and 90 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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 prior to randomization - Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL - Have left ventricular wall (interventricular septum or left ventricular posterior wall) thickness ≥12 mm

Exclusion Criteria

  • 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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
AG10 800 mg 		Subjects will receive AG10 800 mg twice daily. 6 Minute Walk Test (6MWT) primary outcome will be assessed at the end of 12 months, followed by all-cause mortality and cardiovascular-related hospitalization assessed at the end of 30 months.
  • Drug: AG10
    TTR stabilizer administered orally twice daily (BID)
    Other names:
    • acoramidis
    • ALXN2060
Placebo Comparator
Placebo 		Subjects will receive placebo to match twice daily. 6 Minute Walk Test (6MWT) primary outcome will be assessed at the end of 12 months, followed by all-cause mortality and cardiovascular-related hospitalization assessed at the end of 30 months.
  • Drug: Placebo Oral Tablet
    Non-active control administered orally twice daily (BID)

More Details

Status
Active, not recruiting
Sponsor
Eidos Therapeutics

Study Contact

Detailed Description

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. There are two forms of ATTR-CM: - Wild Type* This form of the condition primarily develops in older individuals who do not carry gene mutations - Hereditary* This form of the condition comes from gene mutations passed down in families In this study we are researching the investigational drug AG10 800mg administered orally twice a day. Through the study, we want to evaluate the efficacy and safety of AG10 in patients with ATTR-CM versus placebo This is a 30 month, 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 AG10 or placebo The primary outcomes of the study are: 1. The impact of AG10 versus placebo on the 6 minute walk test after 12 months of treatment 2. The impact of AG10 versus placebo on the frequencies of deaths and cardiovascular-related hospitalizations after 30 months of treatment At the end of 30 months, participants may be eligible to receive investigational AG10, and there is no placebo. This is called an "open label extension." This part of the study may help us better understand the safety related to taking AG10 over a longer period of time

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.