Applied Therapeutics ((APLT)) announced an update on their ongoing clinical study.
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Applied Therapeutics is conducting a study titled ‘A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Long-Term Clinical Benefit of Govorestat in Patients With Charcot-Marie-Tooth-Sorbitol Dehydrogenase Deficiency (CMT-SORD).’ The study aims to assess the long-term efficacy of Govorestat, an aldose reductase inhibitor, in treating CMT-SORD, a rare genetic disorder.
The intervention being tested is Govorestat, administered as a liquid suspension at 20 mg/kg daily. It is designed to inhibit aldose reductase, potentially reducing symptoms of CMT-SORD.
This Phase 3 study is interventional, employing a randomized, double-blind, placebo-controlled design. It uses a parallel assignment model with quadruple masking to ensure unbiased results. The primary purpose is treatment-focused.
The study is set to begin recruiting on September 10, 2025, with primary completion expected 36 months later. The latest update was submitted on September 22, 2025, indicating the study’s current status.
The market implications of this study could be significant for Applied Therapeutics, as positive results may enhance investor confidence and boost stock performance. The study’s progress is crucial in the competitive landscape of rare disease treatments.
The study is ongoing, with further details available on the ClinicalTrials portal.