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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04849741 | A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) | PHASE3 | ACTIVE NOT_RECRUITING | 54 | — | — | Jun 1, 2021 | Sep 1, 2029 | Apr 3, 2026 | 13 | United States, Australia +6 |
10-Meter Walk Test (10MWT) is an assessment of gait speed.
| Arm | Type | Description |
|---|---|---|
| zilganersen | EXPERIMENTAL | Zilganersen will be administered by intrathecal bolus (ITB) injection once every 12 weeks through Week 49. The 60-week double-blind treatment period will be followed by the open-label and long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 229. |
| Placebo | PLACEBO_COMPARATOR | Matching placebo will be administered by ITB injection once every 12 weeks through Week 49. It will be followed by the open-label and long-term extension periods, where participants will receive zilganersen by ITB injection from Week 61 to Week 229. |
| Name | Type | Description |
|---|---|---|
| zilganersen | DRUG | zilganersen will be administered by ITB injection. |
| Placebo | DRUG | zilganersen-matching placebo will be administered by ITB injection. |
Key Inclusion Criteria: 1. Clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease 2. Documented genetic mutation in the GFAP gene 3. Aged ≥ 2 to 65 years old at the time of informed consent 4. Able and willing to meet all study requirements, including travel to Study ...