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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01041222 | Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations | PHASE1 | COMPLETED | 33 | — | — | Jan 1, 2010 | Jan 1, 2012 | Apr 13, 2012 | 4 | United States |
| Arm | Type | Description |
|---|---|---|
| Arm 1 | EXPERIMENTAL | 0.15 mg ISIS 333611 continuous intrathecal infusion over 12 hours |
| Arm 2 | EXPERIMENTAL | 0.5 mg ISIS 333611 continuous intrathecal infusion over 12 hours |
| Arm 3 | EXPERIMENTAL | 1.5 mg ISIS 333611 continuous intrathecal infusion over 12 hours |
| Arm 4 | EXPERIMENTAL | 3.0 mg ISIS 333611 continuous intrathecal infusion over 12 hours |
| Placebo (phosphate buffered saline) | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| ISIS 333611 | DRUG | 5 arms of 12 hour infusion: Arm 1 0.15 mg, Arm 2 0.5 mg, Arm 3 1.5 mg, Arm 4 3.0 mg, matching volume of placebo |
Inclusion Criteria: * Clinical signs of weakness attributed to ALS. * Familial ALS with a documented SOD1 gene mutation. * Age 18 years or older. * Capable of providing informed consent and willing to comply with trial procedures and time commitments. * Vital capacity (VC) at least 50% predicted va...