Phase 3
Completed N=632
Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy
Amyloidosis · Amyloid Cardiomyopathy · Transthyretin Amyloidosis · Cardiomyopathy
Source: ClinicalTrials.gov NCT03860935 ↗
Enrolled (actual)
632
Serious AEs
58.1%
Results posted
Jun 2024
Primary outcomePrimary: A Hierarchical Combination of All-Cause Mortality, Cumulative Frequency of CV-related Hospitalization, Change From Baseline in NT-proBNP and Change From Baseline in 6MWT at the Last Available Visit Where Both Subjects Had Non-missing Assessments. — 63.7; 35.9 Percent of Wins from Win Ratio — p=<0.0001
◆ Published Evidence
Emerging
4citations · ~4 / year
Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.
Summary
Phase 3 efficacy and safety study to evaluate acoramidis (AG10) HCl 800 mg administered orally twice a day compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
Linked Publications (5)
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Efficacy of Acoramidis in Wild-Type and Variant Transthyretin Amyloid Cardiomyopathy: Results From ATTRibute-CM and Its Open-Label Extension.
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A plain language review of the ATTRibute-CM study: efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy.
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Outpatient worsening heart failure in transthyretin amyloid cardiomyopathy: Findings from ATTRibute-CM.
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Efficacy of Acoramidis in Adults With Transthyretin Amyloid Cardiomyopathy: Propensity Score Comparison Between a Single-Arm Open-Label Phase 3 Trial in Japan and a Global Double-Blind Phase 3 Trial.
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Contemporary Oral Medication Use and Frequency in Patients with Transthyretin Amyloid Cardiomyopathy.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY A Hierarchical Combination of All-Cause Mortality, Cumulative Frequency of CV-related Hospitalization, Change From Baseline in NT-proBNP and Change From Baseline in 6MWT at the Last Available Visit Where Both Subjects Had Non-missing Assessments. |
63.7; 35.9 | <0.0001 sig |
| SECONDARY Change From Baseline to Month 30 in the Distance Walked During the 6 Minute Walk Test (6MWT) |
-64.65; -104.29 | <0.0001 sig |
| SECONDARY Change From Baseline to Month 30 of the Kansas City Cardiomyopathy Questionnaire Overall Score (KCCQ-OS) |
-11.48; -21.42 | <0.0001 sig |
| SECONDARY Change From Baseline to Month 30 in Serum TTR (Prealbumin) Level |
5.78; -1.32 | <0.0001 sig |
| SECONDARY All-cause Mortality by Month 30, Including Death Due to Any Cause, Heart Transplant or Cardiac Mechanical Assist Device (CMAD) |
79; 52 | 0.0569 |
Eligibility Criteria
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 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 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
Data sourced from ClinicalTrials.gov (NCT03860935) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.