Clinical Trials, Galactosemia, DbCM

Applied Therapeutics is committed to developing transformative treatments for high unmet medical need indications - fatal or debilitating diseases with no drugs approved.

COVID-19 Statement

Our clinical trials are designed to incorporate home visits to limit travel and risk of exposure to COVID-19.

Interested in participating in one of our clinical trials?

Galactosemia Trials:

Diabetic Cardiomyopathy (ARISE-HF) Trial:

Sorbitol Dehydrogenase (SORD) Deficiency (INSPIRE) Trial:

ACTION-Galactosemia

Applied Therapeutics has completed a Phase 1/2 study of AT-007 in adults (age 18 and older) with Galactosemia. ACTION-Galactosemia evaluated the safety and pharmacokinetics (PK) of AT-007, a central nervous system (CNS) penetrant Aldose Reductase Inhibitor, in healthy volunteers, and explored safety, PK and biomarker effects in adult subjects with Galactosemia. Applied Therapeutics is now conducting a long-term extension with adult study participants.

ACTION-Galactosemia Kids

Applied Therapeutics has completed the registrational ACTION-Galactosemia Kids study of AT-007 in children (aged 2-17) with Galactosemia. Results from the study demonstrated clinical benefit of AT-007 on activities of daily living, behavioral symptoms, cognition and tremor. Consistent with prior findings in adult and pediatric populations, AT-007 continued to be safe and well-tolerated throughout the trial period in all age groups (2-17).

INSPIRE

Applied Therapeutics is currently enrolling a registrational clinical trial of AT-007 in people (age 16 and older) with SORD Deficiency. INSPIRE (INhibiting Sorbitol Production through Inhibition of the aldose Reductase Enzyme) is evaluating biomarker efficacy, clinical outcomes and safety in people living with SORD Deficiency treated with AT-007 vs. placebo

ARISE-HF

Applied Therapeutics is currently enrolling a Phase 3 clinical trial of AT-001 in adults with Type 2 Diabetes for Diabetic Cardiomyopathy (DbCM). ARISE-HF is evaluating whether AT-001 can improve or prevent the decline of functional capacity in patients with DbCM.