AT-001 for Diabetic Cardiomyopathy

Recruiting at 79 trial locations

Overseen By Alessia Urbinati

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Applied Therapeutics, Inc.

Disqualifiers: Heart failure, Coronary disease, Arrhythmia, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

675 Participants Needed

Trial Summary

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What evidence supports the effectiveness of the drug AT-001 for treating diabetic cardiomyopathy?

The research highlights that diabetic cardiomyopathy is linked to metabolic disturbances in the heart, and while specific treatments like AT-001 are not directly mentioned, there is ongoing exploration of therapies targeting these metabolic issues. Some preclinical studies have shown promising results with antifibrotic and anti-inflammatory agents, which could be relevant to the development of AT-001. ^{1 2 3 4 5}

How is the drug AT-001 different from other treatments for diabetic cardiomyopathy?

AT-001 is unique because it is specifically being tested for diabetic cardiomyopathy, a condition with no standard treatment options. Unlike other treatments that may focus on general heart failure, AT-001 is designed to target the specific heart issues caused by diabetes. ^{1 6 7 8 9}

What is the purpose of this trial?

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.

Research Team

James L Januzzi, MD

Principal Investigator

Harvard Medical School (HMS and HSDM)

Eligibility Criteria

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.

Inclusion Criteria

I have Type 2 Diabetes.

My exercise capacity is less than 75% of what's expected for my age and gender.

I have heart problems related to diabetes.

Exclusion Criteria

I need treatment for a serious heart valve problem.

I have a serious irregular heartbeat.

Your blood pressure is higher than 140 over 90 at the initial check-up.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Part A - Treatment

Evaluation of the safety and efficacy of two doses of AT-001 vs placebo to improve or prevent the decline of functional capacity in patients with Diabetic Cardiomyopathy

15-18 months

Part B - Extension

Extension phase to evaluate the safety and efficacy of chronic administration of AT-001 vs placebo, including safety endpoints and exploratory clinical efficacy endpoints

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• AT-001
• Placebo

Trial Overview 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.

Participant Groups

Group I: AT-001 Low Dose Experimental Treatment 1 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 dose Experimental Treatment 1 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 Comparator Placebo Group 1 Intervention

Placebo capsules will be used as comparator

Who Is Running the Clinical Trial?

Applied Therapeutics, Inc.

Lead Sponsor

Trials

5

Recruited

900+

References

1. France pubmed.ncbi.nlm.nih.gov

The current state of preclinical modeling of human diabetic cardiomyopathy using rodents. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Diabetic cardiomyopathy: pathophysiology and clinical features. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Diabetic cardiomyopathy: insights into pathogenesis, diagnostic challenges, and therapeutic options. [2022]

4. England pubmed.ncbi.nlm.nih.gov

Mechanisms of diabetic cardiomyopathy and potential therapeutic strategies: preclinical and clinical evidence. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Targeting metabolic disturbance in the diabetic heart. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

Diabetic cardiomyopathy - A comprehensive updated review. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Ameliorative effect of flavonoid-rich extracts from Gongronema latifolium against diabetic cardiomyopathy via serpin A3 and socs3-a in streptozocin treated rats. [2022]

8. China pubmed.ncbi.nlm.nih.gov

[Protective effect of terpenes from fructus corni on the cardiomyopathy in alloxan-induced diabetic mice]. [2012]

9. United States pubmed.ncbi.nlm.nih.gov

The critical role of Astragalus polysaccharides for the improvement of PPARα [ correction of PPRAα]-mediated lipotoxicity in diabetic cardiomyopathy. [2022]