Trial Outcomes & Findings for SGLT2 Inhibitors in Transthyretin Amyloid Cardiomyopathy (NCT NCT05233163)

NCT ID: NCT05233163

Last Updated: 2024-08-06

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 15 participants
• Primary outcome timeframe: 12 weeks
• Results posted on: 2024-08-06

Participant Flow

Participant milestones

Measure	Empagliflozin Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Overall Study STARTED	15
Overall Study COMPLETED	15
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

SGLT2 Inhibitors in Transthyretin Amyloid Cardiomyopathy

Baseline characteristics by cohort

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Age, Continuous	81 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 weeks

Outcome measures

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Number of Participants Who Experienced a Serious Adverse Event (SAE)	0 Participants

SECONDARY outcome

Timeframe: Baseline, 12 weeks

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.

Outcome measures

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Mean Change in Daily Diuretic Dose	-12 mg/kg Interval -21.0 to -3.0

SECONDARY outcome

Timeframe: Baseline, 6 weeks and 12 weeks

Change in body weight (kg) at trial end compared to trial start

Outcome measures

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Mean Change in Body Weight	-0.97 kg Interval -1.73 to -0.21

SECONDARY outcome

Timeframe: Baseline, 6 weeks and 12 weeks

Change in total water content (kg) as assessed by bioimpedance analysis and quantitative magnetic resonance techniques at trial end compared to trial start

Outcome measures

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Mean Change in Total Water Content	-0.63 kg Interval -1.22 to -0.04

SECONDARY outcome

Timeframe: Baseline, 12 weeks

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.

Outcome measures

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Mean Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score	4 score on a scale Interval -2.0 to 10.0

SECONDARY outcome

Timeframe: Baseline, 12 weeks

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.

Outcome measures

Measure	Empagliflozin n=15 Participants Participants will take empagliflozin 10 mg oral daily for 12 weeks.
Mean Change in Short Physical Performance Battery Score	-0.7 score on a scale Interval -2.0 to 1.0

Adverse Events

Empagliflozin

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Empagliflozin n=15 participants at risk Participants will take empagliflozin 10 mg oral daily for 12 weeks.
General disorders Transient episode of symptomatic hypotension	13.3% 2/15 • Up to 12 weeks
Gastrointestinal disorders Diarrhea	13.3% 2/15 • Up to 12 weeks
Renal and urinary disorders Increased urination	6.7% 1/15 • Up to 12 weeks
Gastrointestinal disorders Constipation	6.7% 1/15 • Up to 12 weeks
Eye disorders Eye stye	6.7% 1/15 • Up to 12 weeks
Infections and infestations Symptomatic COVID-19	20.0% 3/15 • Up to 12 weeks
General disorders Headache	6.7% 1/15 • Up to 12 weeks
General disorders Leg cramping	6.7% 1/15 • Up to 12 weeks
General disorders Lower extremity pruritus	6.7% 1/15 • Up to 12 weeks
General disorders Sinusitis	6.7% 1/15 • Up to 12 weeks
Infections and infestations Lower extremity wound infection	6.7% 1/15 • Up to 12 weeks
Eye disorders Floaters	6.7% 1/15 • Up to 12 weeks
Injury, poisoning and procedural complications Mechanical fall	13.3% 2/15 • Up to 12 weeks
General disorders Tingling sensation in arm	6.7% 1/15 • Up to 12 weeks
General disorders Foot pain, acute onset	6.7% 1/15 • Up to 12 weeks
General disorders Head congestion	13.3% 2/15 • Up to 12 weeks
Musculoskeletal and connective tissue disorders Muscle pain	20.0% 3/15 • Up to 12 weeks
Cardiac disorders Irregular heart rate	6.7% 1/15 • Up to 12 weeks
General disorders Disruption in taste and appetite	6.7% 1/15 • Up to 12 weeks
Skin and subcutaneous tissue disorders Bilateral groin intertrigo	6.7% 1/15 • Up to 12 weeks

Additional Information

Ani Nalbandian, MD

Columbia University Irving Medical Center

Phone: 646-906-2255

Email: an2558@cumc.columbia.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place