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Unknown

AT-001 for Diabetic Cardiomyopathy

Phase 3
Waitlist Available
Research Sponsored by Applied Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Type 2 Diabetes Mellitus
Peak VO2 < 75% of predicted normal value based on age and gender
Must not have
Severe or moderate cardiac valve disease requiring intervention
Clinically significant arrhythmia
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 15 months after randomization]
Awards & highlights
Pivotal Trial

Summary

This trial is testing a new drug called AT-001 to see if it can help adults with a heart condition related to diabetes. The goal is to improve their heart function or prevent it from getting worse, especially for those at high risk of severe heart failure.

Who is the study for?
This trial is for adults with Diabetic Cardiomyopathy who are at high risk of developing heart failure. Participants must have Type 2 Diabetes and reduced exercise capacity (Peak VO2 < 75% of normal). They can't join if they've had severe heart issues, uncontrolled blood pressure, or a history of significant cardiac events.
What is being tested?
The study tests the safety and effectiveness of AT-001 against a placebo in managing Diabetic Cardiomyopathy to prevent worsening into overt heart failure. It's conducted across multiple centers where participants are randomly assigned to either the drug or placebo group.
What are the potential side effects?
While specific side effects for AT-001 aren't listed here, common ones may include potential risks related to heart function, reactions at injection sites, gastrointestinal symptoms, and possible allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have Type 2 Diabetes.
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My exercise capacity is less than 75% of what's expected for my age and gender.
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I have heart problems related to diabetes.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I need treatment for a serious heart valve problem.
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I have a serious irregular heartbeat.
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I have been diagnosed with a specific heart muscle condition before.
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I have had heart issues like a heart attack, bypass surgery, stent placement, or stroke.
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I have a severe condition that affects my ability to do a cardio-pulmonary exercise test.
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I have been diagnosed with or show signs of advanced heart failure.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~15 months after randomization]
This trial's timeline: 3 weeks for screening, Varies for treatment, and 15 months after randomization] for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Peak VO2 during cardio-pulmonary exercise test (CPET);
Secondary study objectives
Changes in NT-proBNP
Changes in the modified Kansas City Cardiomyopathy Questionnaire (KCCQ) score
Progression to overt heart failure (Stage C Heart Failure)
Other study objectives
Changes in echocardiographic parameters
Worsening of diabetic cardiomyopathy

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: AT-001 Low DoseExperimental Treatment1 Intervention
The total daily doses will be of 2g. The dose selection and the frequency of dosing was based on the results of the phase 1/2 study demonstrating that 2g/day of AT-001 is capable of producing a sufficient inhibition of the production of sorbitol (a pharmacodynamic biomarker of biological activity).
Group II: AT-001 High doseExperimental Treatment1 Intervention
The total daily doses will be of 3g. The dose selection and the frequency of dosing was based on the results of the phase 1/2 study demonstrating that 3g/day of AT-001 is capable of producing the maximum inhibition of the production of sorbitol (a pharmacodynamic biomarker of biological activity).
Group III: Placebo ComparatorPlacebo Group1 Intervention
Placebo capsules will be used as comparator
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
AT-001
2016
Completed Phase 2
~200

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for cardiomyopathy include beta-blockers, ACE inhibitors, aldosterone antagonists, and aldose reductase inhibitors like AT-001. Beta-blockers reduce heart rate and blood pressure, decreasing the heart's workload. ACE inhibitors and aldosterone antagonists help relax blood vessels and reduce fluid buildup, improving heart function. Aldose reductase inhibitors, such as AT-001, target the enzyme aldose reductase, which is involved in glucose metabolism and can contribute to cardiac damage in diabetic cardiomyopathy. These treatments are crucial for managing symptoms, preventing disease progression, and improving the quality of life for cardiomyopathy patients.

Find a Location

Who is running the clinical trial?

Applied Therapeutics, Inc.Lead Sponsor
4 Previous Clinical Trials
224 Total Patients Enrolled
James L Januzzi, MDStudy ChairHarvard Medical School (HMS and HSDM)
4 Previous Clinical Trials
1,448 Total Patients Enrolled

Media Library

AT-001 (Unknown) Clinical Trial Eligibility Overview. Trial Name: NCT04083339 — Phase 3
Cardiomyopathy Research Study Groups: Placebo Comparator, AT-001 High dose, AT-001 Low Dose
Cardiomyopathy Clinical Trial 2023: AT-001 Highlights & Side Effects. Trial Name: NCT04083339 — Phase 3
AT-001 (Unknown) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04083339 — Phase 3
Cardiomyopathy Patient Testimony for trial: Trial Name: NCT04083339 — Phase 3
~112 spots leftby Oct 2025