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 consecutive tests
  • Biomarkers of myocardial wall stress, NT-proBNP level ≥300 pg/mL
  • Have left ventricular wall (interventricular septum or left ventricular posterior wall) thickness ≥13 mm

Exclusion Criteria

  • Had acute myocardial infarction, acute coronary syndrome or coronary revascularization, or experienced stroke within 90 days
  • 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 ≥7000 pg/mL pg/mL
  • Measure of kidney function, eGFR by MDRD formula < 15 mL/min/1.73 m2
  • Current treatment with other investigational agents for the treatment of ATTR-CM
  • Current treatment with calcium channel blockers or digitalis

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)
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)

Recruiting Locations

Montefiore Medical Center
Bronx, New York 10461
Contact:
Veronica Day
vday@montefiore.org

More Details

NCT ID
NCT03860935
Status
Recruiting
Sponsor
Eidos Therapeutics

Study Contact

Vincent Knobel
4158871471
clinicaltrials@eidostx.com

Detailed Description

This Phase 3 prospective, randomized, multicenter, parallel-group study will evaluate the efficacy and safety of AG10 800 mg in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM) for a total of 30 months of blinded, placebo-controlled treatment.

At the end of 12 months of treatment (Part A) efficacy of AG10 will be assessed through analyses of the functional endpoint, 6 Minute Walk Test, and the health-related Quality of Life endpoint, heart failure-specific instrument Kansas City Cardiomyopathy Questionnaire.

At the end of 30 months of treatment (Part B) efficacy of AG10 will be further assessed through analysis of all-cause mortality and frequency of cardiovascular-related hospitalization.

All subjects who complete 30 months of blinded, placebo-controlled treatment may be eligible to participate in an open label extension study of long-term AG10 treatment.

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.