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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04304482 | ANAVEX2-73 Study in Pediatric Patients With Rett Syndrome | PHASE2 | COMPLETED | 92 | — | — | Jul 1, 2020 | Jun 30, 2023 | Aug 21, 2023 | 12 | Australia, Canada +1 |
Change from baseline to End of Treatment (EOT) in the Rett Syndrome Behaviour Questionnaire (RSBQ) Total score
Change from baseline to End of Treatment (EOT)
| Arm | Type | Description |
|---|---|---|
| ANAVEX2-73 Active | EXPERIMENTAL | ANAVEX2-73 liquid oral solution |
| ANAVEX2-73 Placebo | PLACEBO_COMPARATOR | Placebo liquid oral solution |
| Name | Type | Description |
|---|---|---|
| ANAVEX2-73 oral liquid | DRUG | Liquid oral solution |
| Placebo oral liquid | DRUG | Liquid oral solution |
Inclusion Criteria: * Aged ≥ 5 years to 17 (inclusive). * Diagnosis of classic RTT, according to 2010 criteria, and a MECP2 mutation. * Post-regression stage, defined as ≥ 6 months since last loss of spoken language or motor (fine or gross) skills. * Clinical Global Impression - Severity (CGI-S) sc...