Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04902781 | Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia | PHASE2 | COMPLETED | 47 | — | — | Mar 20, 2021 | May 21, 2025 | Oct 29, 2025 | 3 | United States |
| NCT04117711 | Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia | PHASE1 | COMPLETED | 114 | — | — | Jun 21, 2019 | Dec 14, 2021 | May 30, 2024 | 4 | United States |
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.
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.
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.
To evaluate the safety and tolerability of AT-007 after administration to healthy subjects, including clinically-significant changes in clinical laboratory test results, physical examination findings, vital sign evaluations, and electrocardiogram results.
| Arm | Type | Description |
|---|---|---|
| Experimental: AT-007 | EXPERIMENTAL | AT-007 The starting dose in Part A will be 5 mg/kg for all age groups. For each age group, Part B of the study will not start until the optimum dose evaluated in Part A has been identified |
| Placebo | PLACEBO_COMPARATOR | Placebo given orally |
| AT-007 | EXPERIMENTAL | AT-007 is a CNS and retina penetrant aldose reductase inhibitor. |
| Placebo Comparator | PLACEBO_COMPARATOR | Placebo is used as a comparator to the experimental arm. |
| Name | Type | Description |
|---|---|---|
| AT-007 | DRUG | Treatment with AT-007 given orally |
| Placebo | OTHER | Placebo given orally |
Inclusion: * Male * Female non-pregnant * Female non-lactating subjects aged ≥2 to \<18 years. * Diagnosis of Classic Galactosemia, confirmed by \<1% GALT (galactose-1-phosphate uridyltransferase) enzyme activity in erythrocytes, or a historical record of diagnosis of \<1% GALT enzyme activity. Ex...