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Transthyretin Amyloid CardiomyopathyPhase 4NCT05233163

SGLT2 Inhibitors in Transthyretin Amyloid Cardiomyopathy

A Phase 4 study testing Empagliflozin in people with Transthyretin Amyloid Cardiomyopathy, run by Columbia University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Transthyretin Amyloid Cardiomyopathy
What is being tested?
Empagliflozin
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteUnited States
Study dates
14 Mar 2022to 1 May 2023
Phase
Phase 4
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Every participant must meet all of the following inclusion criteria to be eligible for enrollment in this study:
  • 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

Who cannot join

  • 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
Show all 29Show fewer
  • 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
  • Chronic kidney disease with estimated glomerular filtration rate (eGFR) <15 mL/min/1.73 m2 or end-stage renal disease
  • Current or prior SGLT2i use
  • Type 1 diabetes mellitus or insulin-dependent diabetes mellitus
  • N-terminal (NT)-pro hormone BNP (NT-proBNP) < 300 pg/mL or < 900 pg/mL if concomitant diagnosis of atrial fibrillation
  • History of ketoacidosis
  • History of complex urinary tract or genital infections
  • History of kidney stone
  • Systolic blood pressure < 90 mmHg and symptomatic hypotension
  • Systolic blood pressure ≥ 180 mmg Hg
  • Chronic obstructive pulmonary disease
  • Major surgery in the 90 days before or after screening
  • Chronic alcohol or drug abuse
  • Nursing home resident
  • Other reason that would make the subject inappropriate for entry into this study

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Columbia University Irving Medical Center / NewYork-Presbyterian Hospital

    New York, New York, 10032

Other trials in United States

About this study

Official title: Sodium-Glucose Cotransporter-2 (SGLT2) Inhibitors in Transthyretin Amyloid (ATTR) Cardiomyopathy

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.

Read the detailed description

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.

Being tested
Empagliflozin
Keywords
Sodium-glucose cotransporter 2 inhibitor (SGLT2i), Empagliflozin, Cardiomyopathy
Medical subject headings
Cardiomyopathies

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EmpagliflozinDrug

    Also called Jardiance

    Empagliflozin 10 mg oral daily for 12 weeks

Study groups in NCT05233163
Study groupRoleWhat they receive
Study Drug ArmExperimentalSubjects will take empagliflozin 10 mg oral daily for 12 weeks.
Receives: Drug: Empagliflozin

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: 12 weeks

Secondary outcomes (5)

  1. 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.

    Measured: Baseline, 12 weeks

  2. Mean Change in Body Weight

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

    Measured: Baseline, 6 weeks and 12 weeks

  3. 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

    Measured: Baseline, 6 weeks and 12 weeks

  4. 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.

    Measured: Baseline, 12 weeks

  5. 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.

    Measured: Baseline, 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Feb 2022
  2. Study start14 Mar 2022
  3. Primary completion1 May 2023
  4. Study completion1 May 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05233163
Sponsor's protocol ID
AAAT9709
Status last verified
Jul 2024
First submitted
31 Jan 2022
Last updated
6 Aug 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Empagliflozin in people with Transthyretin Amyloid Cardiomyopathy, run by Columbia University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 6 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants Who Experienced a Serious Adverse Event (SAE) (time frame: 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05233163.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Aug 2024.

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