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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06181136 | Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) | PHASE1 | ACTIVE NOT_RECRUITING | 20 | — | — | Dec 7, 2023 | Aug 1, 2028 | Dec 5, 2025 | 4 | United States |
| Arm | Type | Description |
|---|---|---|
| Cohort A1 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort A2 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort A3 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort B1 | EXPERIMENTAL | Participants with MPS IIIA |
| Cohort B2 | EXPERIMENTAL | Participants with MPS IIIA |
| Name | Type | Description |
|---|---|---|
| DNL126 | DRUG | intravenous repeating dose |
Key Inclusion Criteria: * Confirmed diagnosis of MPS IIIA * For Cohort A2: No more than 1 participant may have predictors of a slow-progressing phenotype * For Cohort A3: Approximately 2 participants will have predictors of the slow-progressing phenotype * For Cohort B1: Have a severe phenotype bas...