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Confirmatory Study of Govorestat in CMT-SORD

NCT07191912 · Status: WITHDRAWN · Phase: PHASE3 · Type: INTERVENTIONAL

Last updated 2026-05-29

No results posted yet for this study

Summary

This study is designed to assess the long-term efficacy and clinical benefit of AT-007 in patients with CMT-SORD. This randomized, double-blind study will assess the effect of govorestat compared to placebo in patients with CMT-SORD for up to 36 months.

Conditions

  • Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD)

Interventions

DRUG

Govorestat

Govorestat will be provided as a liquid suspension (200mg/mL) for weight-based administration and administered orally at 20 mg/kg QD (every 24 hours)

DRUG

Placebo Control

Placebo will also be provided as a matching liquid suspension to be taken orally QD

Sponsors & Collaborators

  • Applied Therapeutics, Inc.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
16 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-31
Primary Completion
2028-10-31
Completion
2029-01-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • France
  • Germany
  • Italy
  • Spain
  • Turkey (Türkiye)

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07191912 on ClinicalTrials.gov