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NCT04902781 · ClinicalTrials.gov registry record · Phase 2
Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia
A Phase 2 study, sponsored by Applied Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 47
- Enrollment target
NCT04902781: Completed Phase 2 study, sponsored by Applied Therapeutics.
NCT04902781 is a Phase 2 study that has completed, run by Applied Therapeutics. The registered enrollment target is 47 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04902781, a Phase 2 study, has completed, sponsored by Applied Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 47 participants
- Enrollment target
Study Summary
This study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (PK), and pharmacodynamics (PD) (reduction of galactitol levels) of AT-007 in pediatric subjects with Classic Galactosemia (CG).
Primary Outcome
The primary endpoint is a GST of four components: 1) Behavioral Symptoms Index of the Behavioral Assessment Scale for Children 3 (BASC-3); 2) Activities of Daily Living from the BASC-3; 3) Oral Expression from the Oral and Written Language Skills-II (OWLS-II); 4) Listening Comprehension from the OWLS-II. Each individual component is also a secondary endpoint on their own with the test and scoring described below in the secondary endpoint section. For the GST, change from baseline using standardi
Interventions
- OTHER Placebo
- DRUG AT-007
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 47 participants |
| Start Date | 2021-03-20 |
| Est. Completion | 2025-05-21 |
| Phase | Phase 2 |
What the finished NCT04902781 record still lists
NCT04902781 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 47 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (65% lower).
The record links to 0 conditions, and to 2 interventions - of which Placebo is the first listed.
NCT04902781 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04902781 about?
NCT04902781 is a clinical study titled "Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia". This study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (PK), and pharmacodynamics (PD) (reduction of galactitol levels) of AT-007 in pediatric subjects with Classic Galactosemia (CG).
What is the current status of trial NCT04902781?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 47 participants. The study started on 2021-03-20. Estimated completion is 2025-05-21.
What interventions are being tested in trial NCT04902781?
The interventions under investigation include: Placebo (OTHER), AT-007 (DRUG).
Who is sponsoring clinical trial NCT04902781?
This trial is sponsored by Applied Therapeutics, which has 4 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04902781's enrollment target sits among peer trials
47 1283rd of 2000 higher than 715 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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