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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06351592 | First in Human (FIH) Study of ALN-SOD in Adult Participants With Amyotrophic Lateral Sclerosis Associated With Mutation in the SOD1 Gene (SOD1-ALS) | PHASE1 | RECRUITING | 42 | — | — | Aug 28, 2024 | Jun 5, 2031 | Feb 27, 2026 | 16 | Australia, Belgium +4 |
| Arm | Type | Description |
|---|---|---|
| Cohort 1 - Low | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 2 - Mid-Dose | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 3 - High-Dose | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Cohort 4 (Optional) - ≤ High Dose | EXPERIMENTAL | Placebo during 4-Week double-blind treatment period |
| Name | Type | Description |
|---|---|---|
| ALN-SOD | DRUG | Administered per the protocol |
| Diluent | OTHER | Administered per the protocol |
| Placebo (PB) | DRUG | Administered per the protocol |
Key Inclusion Criteria: 1. Weakness attributable to ALS and a SOD1 variant that has been previously described as associated with ALS or is considered likely to cause ALS, as defined in the protocol 2. Slow Vital Capacity (SVC) ≥50% predicted value based on age, gender and height, measured in uprigh...