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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06662188 | JAG201 Gene Therapy Study in Children & Adults With SHANK3 Haploinsufficiency | PHASE1 | RECRUITING | 6 | — | — | Jan 7, 2024 | Jun 1, 2031 | Feb 9, 2026 | 3 | United States |
Incidence, type, severity, and frequency of AEs
Incidence, type, severity, and frequency of SAEs
Changes in clinically significant abnormalities in laboratory values
Incidence of anti-AAV9 antibodies, anti-transgene antibodies, and T-cell reactivity to transgene over time
| Arm | Type | Description |
|---|---|---|
| Pediatric Cohort 1 | EXPERIMENTAL | Starting Dose |
| Pediatric Cohort 2 | EXPERIMENTAL | Escalated Dose |
| Name | Type | Description |
|---|---|---|
| JAG201 | GENETIC | Adeno-associated virus 2/9 expressing a miniature version of the human SHANK3 gene (AAV2/9-miniSHANK3) |
Key Inclusion Criteria: 1. Is male or female, and 2 to 9 years of age at the time of JAG201 administration 2. Has a molecular confirmation of a loss of function mutation in SHANK3 or a 22q13.3 deletion classified as a Class I deletion 3. Has evidence of developmental/cognitive delay of at least 2 s...