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NCT05397665 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study of Hereditary Neuropathy Caused by SORD Deficiency, sponsored by Applied Therapeutics.

Active
Registry status
Phase 2
Development phase
56
Enrollment target
10
Study locations

NCT05397665: Active Phase 2 study of Hereditary Neuropathy Caused by SORD Deficiency, sponsored by Applied Therapeutics.

NCT05397665 is a Phase 2 study of Hereditary Neuropathy Caused by SORD Deficiency that is active but no longer recruiting, run by Applied Therapeutics. The registered enrollment target is 56 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (58% lower). The trial reports 10 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT05397665, a Phase 2 study of Hereditary Neuropathy Caused by SORD Deficiency, is active but no longer recruiting, sponsored by Applied Therapeutics.

ACTIVE NOT RECRUITING
Registry status
Phase 2
Development phase
56 participants
Enrollment target
10
Study locations

Study 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.

Primary Outcome

The 10MWRT is a timed functional test used to measure walking or running speed over 10 meters by the study population

Interventions

  • DRUG Placebo
  • DRUG AT-007

Study Locations (10)

Colorado

  • University of Colorado - Aurora

Florida

  • University of Miami - Coral Gables

Iowa

  • University of Iowa - Iowa City

Massachusetts

  • Massachusetts General Hospital - Boston

New Jersey

  • Hassman Research Institute - Berlin

New York

  • University of Rochester - Rochester

Pennsylvania

  • University of Pennsylvania - Philadelphia

Prague

  • Axon Clinical, s.r.o. - Prague

Trial Details

FieldValue
Enrollment Target 56 participants
Start Date 2022-01-01
Est. Completion 2026-10
Phase Phase 2
Applied Therapeutics

4 total trials

What the registry record for NCT05397665 still lists

NCT05397665 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 56 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (58% lower).

The record links to 1 condition, with Hereditary Neuropathy Caused by SORD Deficiency appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.

NCT05397665 lists 10 locations in 10 states (Colorado, Florida, Iowa).

Frequently Asked Questions

What is clinical trial NCT05397665 about?

NCT05397665 is a clinical study titled "Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency". 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.

What is the current status of trial NCT05397665?

This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 56 participants. The study started on 2022-01-01. Estimated completion is 2026-10.

What conditions does trial NCT05397665 study?

This clinical trial studies the following conditions: Hereditary Neuropathy Caused by SORD Deficiency.

What interventions are being tested in trial NCT05397665?

The interventions under investigation include: Placebo (DRUG), AT-007 (DRUG).

Who is sponsoring clinical trial NCT05397665?

This trial is sponsored by Applied Therapeutics, which has 4 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05397665 being conducted?

This trial has 10 study locations across Colorado, Florida, Iowa, Massachusetts, New Jersey. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

How this trial's enrollment target compares

Where NCT05397665's enrollment target sits among peer trials

56 1146th of 2000 higher than 848 of 2,000 other Phase 2 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT05397665, the US trial registry maintained by the National Library of Medicine. NCT05397665 (small enrollment · multi site footprint · active not recruiting) retrieved and formatted by PlainTrial, see methodology.