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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT07480564 | Safety and Preliminary Efficacy of TSHA-102 Gene Therapy in Pediatric Females Aged >2 to <4 Years With Rett Syndrome | PHASE3 | RECRUITING | 3 | — | — | May 8, 2026 | Jun 1, 2031 | May 22, 2026 | 1 | United States |
| NCT05606614 | A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study) | PHASE3 | RECRUITING | 15 | — | — | Mar 6, 2023 | Jun 1, 2031 | Dec 30, 2025 | 6 | United States, Canada |
| NCT06152237 | Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) | PHASE1 | ACTIVE NOT_RECRUITING | 6 | — | — | Dec 12, 2023 | Nov 2, 2031 | Oct 15, 2025 | 5 | United States, Canada +1 |
Safety and Tolerability of TSHA-102 Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Proportions of participants experiencing any treatment-emergent adverse events (AEs) and serious adverse events (SAEs)
Change from baseline in percentage of participants who gain or regain any one or more of the 28 items from the Developmental Milestones Assessment (DMA), which are video recorded and scored by independent, blinded central raters.
| Arm | Type | Description |
|---|---|---|
| Treatment | EXPERIMENTAL | Participants receive a single intrathecal (IT) administration of TSHA-102 at 1.0 × 10¹⁵ total vector genomes (vg) adjusted for the participant's brain volume. |
| Part A Cohort 1 | EXPERIMENTAL | TSHA-102 Dose Level 1: 5.7×10¹⁴ total vector genomes (vg). Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 1 (fully enrolled, 2 participants). |
| Part A Cohort 2 | EXPERIMENTAL | TSHA-102 Dose Level 2: 1.0×10¹⁵ total vector genomes (vg) Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (fully enrolled, 4 participants). |
| Part B Pivotal Cohort | EXPERIMENTAL | TSHA-102 at Selected Dose (Dose Level 2): 1.0 × 10¹⁵ total vector genomes (vg) Participants receive a single intrathecal (IT) administration of TSHA-102 at Dose Level 2 (1.0 × 10¹⁵). |
| Cohort 1 | EXPERIMENTAL | Dose Level 1 |
| Cohort 2 | EXPERIMENTAL | Dose Level 2 |
| Name | Type | Description |
|---|---|---|
| TSHA-102 | GENETIC | TSHA-102 is a recombinant, non-replicating, self-complementary adeno-associated virus serotype 9 (scAAV9) vector encoding for the miniMECP2 gene. TSHA-102 is a one-time intrathecal (IT) administration. |
Inclusion Criteria: * Pediatric females between the ages of 2 and less than 4 years old. * Participant has a clinical diagnosis of classic/typical Rett syndrome with a documented pathogenic mutation of the methyl-CpG-binding protein 2 (MECP2) gene that results in loss of gene function. * Participan...