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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05898620 | A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome | PHASE3 | RECRUITING | 33 | — | — | Jun 13, 2023 | Dec 1, 2029 | Mar 13, 2026 | 16 | United States, Australia +1 |
Responders will be defined as participants who: * Attain a CGI-I score of ≤ 3 ("minimally improved"); * and gain any one developmental milestone/skill from a list of 28, as captured through standardized video recordings and independently verified by blinded central raters.
| Arm | Type | Description |
|---|---|---|
| Pediatric 1e15 vg dose (fully enrolled) | EXPERIMENTAL | Dose Level 1 for ages 4-10 years |
| Adolescent/Adult 1e15 vg Dose (fully enrolled) | EXPERIMENTAL | Dose Level 1 for ages 11 years \& above |
| Pediatric 3e15 vg dose (discontinued) | EXPERIMENTAL | Dose Level 2 for ages 4-10 years (discontinued) |
| Pivotal Cohort | EXPERIMENTAL | Dose Level 1 for ages 3 and above |
| Name | Type | Description |
|---|---|---|
| NGN-401 | GENETIC | NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene. |
Inclusion Criteria: * Females who are between the ages of ≥4 and ≤10 years for Arms 1 and 2 (Arms closed). Females who are ≥11 years of age or older for Arm 3 (Arm closed). Females who are ≥3 for Arm 4, the pivotal cohort. * Diagnosis of typical Rett syndrome with a documented disease-causing mutat...