Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ISIS 333611 · 1 trial · 1 indication
| 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...
ISIS 333611 is an investigational small molecule being developed for the treatment of familial amyotrophic lateral sclerosis (ALS), a form of ALS caused by SOD1 gene mutations. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.
ISIS 333611 is being developed by Ionis Pharmaceuticals, Inc. (NASDAQ: IONS). The company is conducting clinical research on this investigational drug for familial amyotrophic lateral sclerosis.
ISIS 333611 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA or any other regulatory agency. One Phase 1 trial has been completed, and the drug remains under clinical investigation.
ISIS 333611 has been studied in one completed Phase 1 clinical trial, NCT01041222, which evaluated its safety, tolerability, and activity in patients with familial amyotrophic lateral sclerosis caused by SOD1 gene mutations. The trial enrolled 33 participants in the United States.
ISIS 333611 is also known as ISIS SOD1Rx, as referenced in the clinical trial NCT01041222. The trial title refers to the drug as ISIS SOD1Rx, indicating that both names are used for the same investigational compound.