NCT05397665

Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency

A RandomIzed, Double-Blind, Placebo-CoNtrolled, Two-Part Study to Evaluate the Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With SoRbitol Dehydrogenase (SORD) DEficiency

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 56 (actual)
• Sponsor: Applied Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to assess the efficacy and safety of AT-007 treatment in patients with SORD Deficiency. This randomized, double-blind study will assess the effect of AT-007 compared to Placebo in SORD Deficiency in patients for up to 24 months.

Detailed description

This international, multi-center, randomized, double-blinded, placebo-controlled, phase 2-3 study is designed to assess the pharmacodynamic (PD) efficacy of AT-007 treatment, as well as the clinical benefit of long term administration to patients with SORD Deficiency utilizing a series of functional, patient-reported, and clinical outcomes measures.Safety and pharmacokinetics (PK) of AT-007 will also be evaluated. The study consists of Biomarker and Efficacy Assessments and an open-label extension (OLE) of active treatment for qualified patients. Patients (18-55 years old) with SORD deficiency will be stratified according to their 10MWRT score (a single component of the CMT-FOM) and sex (male vs female). They will then be randomized in a 2:1 ratio to AT-007 20 mg/kg once daily (QD) or placebo. The study will be conducted at up to 13 sites worldwide. A total of up to 72 subjects will be enrolled.

Conditions

• Hereditary Neuropathy Caused by SORD Deficiency

Interventions

Type	Name	Description
DRUG	AT-007	AT-007, aldose reductase inhibitor
DRUG	Placebo	Liquid oral suspension

Timeline

• Start date: 2022-01-01
• Primary completion: 2024-10-12
• Completion: 2026-10-01
• First posted: 2022-05-31
• Last updated: 2025-09-15

Locations

10 sites across 4 countries: United States, Czechia, Italy, United Kingdom

Regulatory

• FDA-regulated drug study