Trial · NCT04902781
Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia
NCT04902781, Phase 2, Phase 3, Completed, Applied Therapeutics, Inc.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia. Trials, record NCT04902781. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04902781
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia
- NCT number
- NCT04902781
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 47 participants, actual
- Lead sponsor
- Applied Therapeutics, Inc.
- Conditions
- Classic Galactosemia
- Interventions
- AT-007, Placebo
- First posted
- 2021-05-26
- Last updated
- 2025-10-29
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Global Statistical Test (GST) | Month 6, Month 12 and Month 18 | 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 standardized z-scores is calculated for active versus placebo. |
| Primary | Sensitivity Analysis of the Primary Endpoint (GST) with Cognition | Month 6, Month 12 and Month 18 | Sensitivity Analysis adding the National Institutes of Health Toolbox Cognition Battery (NIH-CB) test which measures cognition. NIH-CB is described below in the secondary endpoints. For the GST with cognition, change from baseline using standardized z-scores is calculated for active versus placebo. |
| Primary | Sensitivity Analysis of the Primary Endpoint (GST) with Fine Motor Skills | Month 6, Month 12 and Month 18 | Sensitivity Analysis adding the National Institutes of Health Toolbox Motor Battery (NIH-MB) 9-Hole Pegboard test which measures fine motor skills. NIH-MB is described below in the secondary endpoints. For the GST with fine motor skills, change from baseline using standardized z-scores is calculated for active versus placebo. |
| Secondary | Galactitol Level | Month 1, Month 2, Month 3, Month 6, Month 12, and Month 18 | The change from baseline in plasma galactitol (Pharmacodynamic measure) |
| Secondary | Behavioral Assessment System for Children-3 [Individual Components] | Month 6, Month 12 and Month 18 | Behavioral Assessment System for Children (BASC-3) has a normal range of 40-60 with the higher score showing more difficulty. There are multiple outcome scores included such as: Behavioral Symptoms Index, Activities of Daily Living, Withdrawal, Adapatability, Adaptive Skills, Socialization, and Functional Impairment Index. |
| Secondary | National Institute of Health Toolbox Cognition Battery | Month 6, Month 12 and Month 18 | Change from baseline in the NIH Toolbox Cognition Battery (NIH-CB). The NIH-CB uses multiple tests to evaluate Cognition and consists of tests of multiple constructs to yield individual ranking and demonstrate Cognitive Function Composite, Fluid Cognition Composite Sequence Memory, List Sorting, Pattern Comparison, and Crystallized Cognition Composite, Picture Vocabulary and Reading Recognition. This is standardized normal or 100 with a standard deviation of 10. Higher scores are better. |
| Secondary | Archimedes Spiral Drawing Test | Month 6, Month 12 and Month 18 | The Archimedes Spiral Drawing Test evaluates tremor on a scale of 0 (no tremor) to 4 (severe tremor). |
| Secondary | National Institute of Health Toolbox Motor Battery | Month 6, Month 12 and Month 18 | NIH Toolbox Motor Battery (NIH-MB) is a multiple of tests to determine fine motor skills using the 9-Hole Pegboard Test and gross motor skills using the Standing Balance Test. This is standardized normal or 100 with a standard deviation of 10. Higher scores are better. |
| Secondary | Scale for Assessment & Rating of Ataxia | Month 6, Month 12 and Month 18 | Scale for Assessment \& Rating of Ataxia (SARA) is 0-40, the higher the score the worse value |
| Secondary | Oral and Written Language Scales-II | Month 6, Month 12 and Month 18 | Oral and Written Language Scales-II (OWLS-II) has both an Oral Expression and Listening Comprehension score. Scores are 0-100 with a standard deviation of 15. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 39569553 | DERIVED |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests1
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
| Test | Named as |
|---|---|
| Spiral Drawing Test | Secondary outcome |
Source ConductCognition
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04902781
GET https://conductscience.org/api/trials/v1/NCT04902781/registered