Active not recruitingPHASE3INTERVENTIONAL

Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy

This research study is investigating a new medication called AT-001 for individuals living with 'diabetic cardiomyopathy'. This is a heart condition that can develop in people with diabetes. The main goal is to find out if AT-001 is safe to use and if it can help prevent the heart condition from getting worse and leading to more serious heart failure. Around 675 adult patients will take part. Some will receive AT-001, and others will receive a 'placebo' (a dummy drug) to compare the effects. The study will look at whether the drug helps maintain people's physical ability and will also monitor for any serious heart-related events like hospitalisation or death over an extended period.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Applied Therapeutics, Inc.

Enrolment target

675

Start

20 Sep 2019

Estimated completion

01 Dec 2025

Diabetic Cardiomyopathies

What is this study about?

This study is looking into a new treatment, AT-001, for a heart condition called 'diabetic cardiomyopathy'. This condition affects some people who have diabetes, where the heart muscle changes and struggles to pump blood effectively. Doctors are particularly interested in finding ways to help people whose diabetic cardiomyopathy puts them at a higher risk of developing more serious heart failure in the future.

The main aim of this study is to test if AT-001 is both safe and effective at preventing this progression to more severe heart failure. It brings together researchers from different centres to carefully compare AT-001 with a 'placebo' – which looks exactly like the study drug but doesn't contain any active medicine. This comparison helps researchers understand the true effects of AT-001.

The study is divided into two parts. In the first part, researchers will look closely at how well AT-001 helps people maintain their physical capacity and how safe it is at different doses. If promising, the study will continue into a second, longer part to see if taking AT-001 over an extended period continues to be safe and if it can reduce the chances of serious heart-related events like needing to go to hospital or even death.

Key takeaways

Tests a new drug (AT-001) for diabetic cardiomyopathy.
Aims to prevent worsening heart problems in people with diabetes.
Compares AT-001 against a dummy drug (placebo).
Involves regular health checks and physical fitness tests over a long period.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you would need to be an adult, at least 40 years old, and have a diagnosis of type 2 diabetes. Importantly, you would also need to have been diagnosed with diabetic cardiomyopathy and show some signs that your physical fitness, specifically how well your body uses oxygen during exercise, is less than what doctors would typically expect for someone of your age and gender.

However, there are several reasons why you might not be able to join. For example, if you've already been diagnosed with more severe heart failure, or if you've had certain heart procedures or conditions in the past like a heart attack, bypass surgery, or severe valve disease, you wouldn't be eligible. Conditions like uncontrolled high blood pressure or very high blood sugar levels (HbA1c over 8.5%) would also rule you out. The study is also not suitable if you have other severe health problems that might make it difficult to perform exercise tests.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you at least 40 years old?
Do you have type 2 diabetes?
Have you been diagnosed with diabetic cardiomyopathy?
Do you have an existing diagnosis of severe heart failure or specific significant heart conditions?
Is your blood pressure generally well-controlled (systolic below 140 mmHg and diastolic below 90 mmHg)?
Is your long-term blood sugar (HbA1c) below 8.5%?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be randomly assigned to either receive the study drug, AT-001, or a placebo (a dummy drug). This means neither you nor your study doctor would know which you are receiving. The study involves regular visits to the clinic for various assessments, which will include checks on your heart health, blood tests, and exercise tests to measure your physical fitness. Initially, these assessments will be more frequent, and then, if you continue into the second part of the study, they will be spread out over at least 12 months. The total duration of your participation would depend on how long you remain in the study, but it is expected to be over a year.

Potential risks and benefits

Taking part in a clinical trial offers the potential benefit of accessing a new treatment before it's widely available, which could potentially slow down the progression of your heart condition. However, there are also potential risks, including side effects from the study drug, which could range from mild to more serious. The placebo group won't receive the active drug, so they won't experience its effects, positive or negative. You would be closely monitored throughout the study for your safety and well-being. It's important to remember that participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (79)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Westside Medical Associates of Los Angeles
Verified postcode
Beverly Hills, United States
University of California, San Diego (UCSD)
Verified postcode
La Jolla, United States
Clinical Trials Research
Verified postcode
Lincoln, United States
University of California - Irvine Medical Center
Verified postcode
Orange, United States
Metabolic Institute of America
Verified postcode
Tarzana, United States
Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center
Verified postcode
Torrance, United States
ALL Medical Research, LLC
Verified postcode
Cooper City, United States
New Generation of Medical Research
Verified postcode
Hialeah, United States
Broward Research Center
Verified postcode
Pembroke Pines, United States
Progressive Medical Research
Verified postcode
Port Orange, United States
UnityPoint Health - Methodist Hospital
Verified postcode
Peoria, United States
Brigham and Women's Hospital
Verified postcode
Boston, United States

Common questions

What is diabetic cardiomyopathy?

It's a type of heart problem that can affect some people with diabetes, where the heart muscle changes and doesn't pump as strongly as it should.

What is a placebo?

A placebo is a substance that looks exactly like the study drug but contains no active medicine. It's used for comparison to see if the study drug has a real effect.

What does 'Phase 3' mean for a clinical trial?

Phase 3 means the drug has already shown promise in earlier stages, and this is a larger trial to confirm its safety and effectiveness before it might be considered for wider use.

What is 'heart failure'?

Heart failure is a condition where the heart can't pump enough blood to meet the body's needs. It doesn't mean the heart has stopped working, but it needs to work harder.

Will I know if I'm getting the actual drug or the placebo?

No, this is a 'blinded' study, meaning neither you nor your study doctor will know whether you are receiving AT-001 or the placebo until the study is over.