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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04165486 | Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON) | PHASE1 | RECRUITING | 40 | — | — | Jul 21, 2022 | Sep 1, 2027 | Apr 27, 2026 | 15 | Austria, France +3 |
| Arm | Type | Description |
|---|---|---|
| Part 1: ION464 | EXPERIMENTAL | ION464 will be administered at multiple-ascending doses by IT injection at regular intervals over 12 weeks. |
| Part 1: Placebo | PLACEBO_COMPARATOR | ION464-matching placebo will be administered by IT injection at regular intervals over 12 weeks. |
| Part 2: ION464 | EXPERIMENTAL | ION464 will be administered at the same doses as Part 1 by IT injection, at regular intervals, for 72 weeks. |
| Part 2: Placebo | PLACEBO_COMPARATOR | ION464-matching placebo will be administered by IT injection, at regular intervals, for 72 weeks. |
| Name | Type | Description |
|---|---|---|
| ION464 | DRUG | ION464 will be administered by IT injection. |
| Placebo | DRUG | ION464-matching placebo will be administered by IT injection. |
Key Inclusion Criteria: * Screening single-photon emission computed tomography (SPECT) with DaTscan™ (ioflupane I123 injection) results demonstrating loss (whether symmetric or asymmetric) of dopamine nerve terminals in the striatum consistent with neurodegenerative parkinsonism, as assessed with q...