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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07579910 | Intracerebroventricular Tralesinidase Alfa in Children With Mucopolysaccharidosis Type IIIB | PHASE3 | NOT YET_RECRUITING | 14 | — | — | Dec 1, 2026 | Aug 1, 2033 | May 12, 2026 | - | — |
The BSID-III-C is a standardized tool that evaluates cognitive development in young children, capturing skills such as processing speed, problem solving, and play. Raw scores are used to sensitively measure individual change over time, independent of age. Assessments are administered by a trained, blinded rater and reviewed by a central blinded rater. The endpoint is the difference in mean change from baseline in BSID-III-C raw score between the tralesinidase alfa and control arms at Week 260
| Arm | Type | Description |
|---|---|---|
| Tralesinidase alfa (TA) | EXPERIMENTAL | - |
| Control | NO_INTERVENTION | - |
| Name | Type | Description |
|---|---|---|
| Tralesinidase alfa (TA) | DRUG | TA study drug is a sterile solution for ICV infusion. |
Inclusion Criteria: Has a diagnosis of MPS IIIB confirmed by deficient NAGLU enzyme activity during screening. Has nonattenuated severe MPS IIIB by the Genotype and Clinical Assessment Committee. Is ≥1 and ≤5 years of age with BSID-III-C raw score of \<70. Is male or female as identified at birth...