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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04798235 | First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis | PHASE1 | ACTIVE NOT_RECRUITING | 3 | — | — | Mar 12, 2021 | Mar 12, 2027 | May 9, 2023 | 1 | Canada |
Incidence, severity, and relatedness of TEAEs
Number of participants with Changes from Baseline in laboratory assessments
Changes from Baseline in 12-lead ECG findings in QT interval
| Arm | Type | Description |
|---|---|---|
| TSHA-101 | EXPERIMENTAL | Subjects who will receive one-time intrathecal TSHA-101, brain volume based sliding scale for dosage |
| Name | Type | Description |
|---|---|---|
| TSHA-101 | BIOLOGICAL | AAV9 viral vector containing HEXA and HEXB genes to be administered via Intrathecal injection |
Key Inclusion Criteria: * male or female with age less than or equal to 15 months * diagnosis of GM2 gangliosidosis with genetic and enzymatic documentation of infantile disease Key Exclusion Criteria: * a second neurodevelopmental disorder independent of the HEXA or HEXB * inability to tolerate ...