Recent Updates
Recently added Catalysts

AT-001

Phase 3

Diabetic Cardiomyopathies | Small molecule | Other |Applied Therapeutics, Inc.|Last Updated: Dec 8, 2022

Target and mechanism

Molecular targetAldose Reductase
Target classEnzyme
ModalitySmall molecule

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment675

FDA Designations

No designations recorded

Clinical trial landscape

AT-001 · 1 trial · 1 indication

Phase 3 1
NCT04083339Safety and Efficacy of AT-001 in Patients With Diabetic CardiomyopathyDiabetic Cardiomyopathies
ACTIVE NOT_RECRUITING675 Analytics
PHASE3ACTIVE NOT_RECRUITING
Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy
Diabetic CardiomyopathiesUnlock trial analytics

Study Endpoints

Primary Endpoints

Peak VO2 during cardio-pulmonary exercise test (CPET);
15 months after randomization]

Changes in Peak VO2 during cardio-pulmonary exercise test (CPET) from baseline to approximately Month 15 (15-18 months). A CPET may be repeated at approximately Month 27 (27-30 months).

Secondary Endpoints

Progression to overt heart failure (Stage C Heart Failure)
27 months after randomization
Changes in NT-proBNP
27 months after randomization
Changes in the modified Kansas City Cardiomyopathy Questionnaire (KCCQ) score
27 months after randomization
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AT-001 High doseEXPERIMENTALThe 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).
AT-001 Low DoseEXPERIMENTALThe 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).
Placebo ComparatorPLACEBO_COMPARATORPlacebo capsules will be used as comparator

Interventions

NameTypeDescription
AT-001DRUGAT-001 will be administered as 3 capsules twice daily, before breakfast and before dinner. At present AT001 is the sole name for the active substance. No INN/genetic name is available to date
PlaceboDRUGMatching placebo will be administered as 3 capsules twice daily, before breakfast and before dinner
Unlock Study Design Details

Eligibility Criteria

Age Range40 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites79

Inclusion Criteria: * Type 2 Diabetes Mellitus * Diabetic cardiomyopathy * Peak VO2 \< 75% of predicted normal value based on age and gender Exclusion Criteria: * Prior diagnosis or signs/symptoms of overt/symptomatic heart failure / stage C heart failure * Prior echocardiogrphic measurement of e...

Countries:United StatesAustraliaCanadaCzechiaFranceGermanyHong KongPolandSpainUnited Kingdom
Unlock Eligibility Criteria

Frequently asked questions about AT-001

What is AT-001 used for?

AT-001 is an investigational small molecule being developed for diabetic cardiomyopathy, a heart muscle disease that occurs in people with diabetes. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

What does AT-001 target?

AT-001 targets the enzyme aldose reductase. By inhibiting this enzyme, the drug is intended to address a pathway implicated in diabetic cardiomyopathy. The drug is being studied for its potential to affect the progression of this condition.

Who is developing AT-001?

AT-001 is being developed by Applied Therapeutics, Inc., a biopharmaceutical company listed on the stock exchange under the ticker symbol APLT. The company is conducting clinical trials to evaluate the safety and efficacy of AT-001 in patients with diabetic cardiomyopathy.

What phase is AT-001 in?

AT-001 is in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities. The ongoing Phase 3 trial is designed to assess its safety and efficacy in patients with diabetic cardiomyopathy.

What clinical trials is AT-001 in?

AT-001 is being studied in a Phase 3 clinical trial registered as NCT04083339, titled "Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy." The trial is active but not recruiting, with an enrollment of 675 participants across multiple countries including the United States, Australia, Canada, and several European nations.

Is AT-001 the same as any other drug?

AT-001 is the primary name used for this investigational drug in clinical development. No alternative names have been reported for this compound in the context of its ongoing trials for diabetic cardiomyopathy.