SGLT2 Inhibitors in Transthyretin Amyloid Cardiomyopathy

NCT05233163 | Completed Phase 4 Results DMC FDA Drug

• 1 other identifier: AAAT9709
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Transthyretin Amyloid Cardiomyopathy

Keywords

Sodium-glucose cotransporter 2 inhibitor (SGLT2i); Empagliflozin; Cardiomyopathy

Outcome Measures

Primary Outcomes (1)

• Number of Participants Who Experienced a Serious Adverse Event (SAE)

  12 weeks

Secondary Outcomes (5)

• Mean Change in Daily Diuretic Dose

  Baseline, 12 weeks

• Mean Change in Body Weight

  Baseline, 6 weeks and 12 weeks

• Mean Change in Total Water Content

  Baseline, 6 weeks and 12 weeks

• Mean Change in Kansas City Cardiomyopathy Questionnaire (KCCQ) Score

  Baseline, 12 weeks

• Mean Change in Short Physical Performance Battery Score

  Baseline, 12 weeks

Study Arms (1)

Study Drug Arm

EXPERIMENTAL

Subjects will take empagliflozin 10 mg oral daily for 12 weeks.

Drug: Empagliflozin

Interventions

Empagliflozin DRUG

Empagliflozin 10 mg oral daily for 12 weeks

Also known as: Jardiance

Study Drug Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years old
• Diagnosis of TTR cardiac amyloidosis (wild type or variant), confirmed by the presence of amyloid deposits on analysis of biopsy specimens obtained from cardiac and noncardiac sites (e.g. fat aspirate, gastrointestinal sites, salivary glands, or bone marrow), technetium-99m pyrophosphate cardiac scintigraphy, or mass spectrometry
• Normal serum free light chain ratio and the absence of abnormal monoclonal band on serum and urine immunofixation
• Subjects will have at least 1 of the indications below for an SGLT2i, and meet package-insert criteria for drug initiation: non-insulin dependent diabetes mellitus with hemoglobin A1c ranging from 6.5-9.9 OR chronic kidney disease (defined as an estimated glomerular filtration rate of 25-75 ml/minute/1.73 m2 of body-surface area)
• On stable oral diuretics (defined as no more than a 50% increase from baseline diuretic dose established during a sustained 2 week period) within 2 weeks before enrollment
• Able to understand and sign the informed consent document after the nature of the study has been fully explained

You may not qualify if:

• The presence of any of the following excludes eligibility for enrollment in this study:
• Prior liver or heart transplantation
• Active malignancy or non-amyloid disease with expected survival of less than 1 year
• Heart failure, in the opinion of the investigator, primarily caused by severe left-sided valve disease. Note: if valve was repaired, subject may be considered as no longer with severe valve disease
• Heart failure, in the opinion of the investigator, primarily caused by ischemic heart disease
• Ventricular assist device or anticipated within the next 6 months
• Pacemaker or implantable cardioverter defibrillator incompatible with magnetic resonance technology
• Absolute contraindication for quantitative magnetic resonance (e.g. aneurysmal clips, metal objects)
• Impairment from stroke, injury or other medical disorder that precludes participation in the study
• Myocardial infarction, cardiovascular surgery, stroke or transient ischemic attack within the prior 90 days
• Disabling dementia or other mental or behavioral disease
• Enrollment in a clinical trial not approved for co-enrollment
• Expected use of continuous intravenous inotropic therapy in the next 6 months
• High risk for non-adherence as determined by screening evaluation
• Inability or unwillingness to comply with the study requirements

Sponsors & Collaborators

• Columbia University: lead

Study Sites (1)

Columbia University Irving Medical Center / NewYork-Presbyterian Hospital

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Cardiomyopathies

Interventions

empagliflozin

Condition Hierarchy (Ancestors)

Heart Diseases; Cardiovascular Diseases

Results Point of Contact

• Title: Ani Nalbandian, MD
• Organization: Columbia University Irving Medical Center

an2558@cumc.columbia.edu

646-906-2255

Study Officials

• Mathew Maurer, MD

  Columbia University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Arnold and Arlene Goldstein Professor of Cardiology

Study Record Dates

• First Submitted: January 31, 2022
• First Posted: February 10, 2022
• Study Start: March 14, 2022
• Primary Completion: May 1, 2023
• Study Completion: May 1, 2023
• Last Updated: August 6, 2024
• Results First Posted: August 6, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)