This is a single center, single arm, prospective, 12 week open label pilot trial of the sodium-glucose cotransporter 2 inhibitor (SGLT2i), empagliflozin 10 mg oral daily, in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The target population for enrollment will be subjects with ATTR-CM and either non-insulin dependent diabetes mellitus or chronic kidney disease. The primary aim will be to assess the safety and tolerability of empagliflozin 10 mg oral daily in subjects with heart failure secondary to ATTR, which remain unexplored. The accrual target is 15 subjects. Consented subjects will be evaluated for safety and tolerability of study drug, empagliflozin 10 mg oral daily, over a period of 12 weeks. Subjects will undergo a total of 6 study visits: 3 in-person and 3 telephone follow-ups.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underrecognized cause of heart failure among older adults, in which misfolded transthyretin (TTR) proteins build up in the muscle tissue of the heart. In 2018, a drug called tafamidis was proven to stabilize the protein and protect people from further damage, and in 2019, it was approved for use in ATTR-CM by the U.S. Food and Drug Administration (FDA). But while people with ATTR-CM are living longer, they continue to suffer from symptoms of heart failure and cardiorenal syndrome as the disease progresses. Sodium glucose co-transporter inhibitors (SGLT2i) are drugs used to treat diabetes mellitus, heart failure, and chronic kidney disease. They may also reduce systemic inflammation, and affect body composition (fat, lean mass, and total water contents) in patients with heart failure, a condition which also has significant overlap with obesity. No one has explored the safety, tolerability, and clinical effects of SGLT2i in patients with heart failure due to ATTR-CM.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
15
Empagliflozin 10 mg oral daily for 12 weeks
Columbia University Irving Medical Center / NewYork-Presbyterian Hospital
New York, New York, United States
Number of Participants Who Experienced a Serious Adverse Event (SAE)
Time frame: 12 weeks
Mean Change in Daily Diuretic Dose
Calculated in mg/kg of furosemide equivalence. This outcome was assessed at Baseline, 6 weeks, and 12 weeks; the change from baseline to 12 weeks is reported.
Time frame: Baseline, 12 weeks
Mean Change in Body Weight
Change in body weight (kg) at trial end compared to trial start
Time frame: Baseline, 6 weeks and 12 weeks
Mean Change in Total Water Content
Change in total water content (kg) as assessed by bioimpedance analysis and quantitative magnetic resonance techniques at trial end compared to trial start
Time frame: Baseline, 6 weeks and 12 weeks
Mean Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a 23-item self-administered questionnaire developed to independently measure the patient's perception of their health status, which includes heart failure symptoms, impact on physical and social function, and how their heart failure impacts their quality of life (QOL) within a 2-week recall period. KCCQ scores are scaled from 0 to 100 and frequently summarized in 25-point ranges, where scores represent health status as follows: 0 to 24: very poor to poor; 25 to 49: poor to fair; 50 to 74: fair to good; and 75 to 100: good to excellent (better outcome). This outcome was assessed at Baseline, 6 weeks, and 12 weeks; the change from baseline to 12 weeks is reported.
Time frame: Baseline, 12 weeks
Mean Change in Short Physical Performance Battery Score
The Short Physical Performance Battery (SPPB) is an objective assessment tool for evaluating lower extremity functioning in older persons. Each test is scored on a 0 to 4 scale using previously validated norms and summed for an overall score range of 0 to 12, with 0 indicating the lowest physical performance, and scores of 12 indicating the highest performance (better outcome). This outcome was assessed at Baseline, 6 weeks, and 12 weeks; the change from baseline to 12 weeks is reported.
Time frame: Baseline, 12 weeks
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