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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 therapy being studied for the treatment of familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene. It is not approved for any indication and remains in clinical development.
ISIS 333611 is being developed by Ionis Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker symbol IONS. The company is the sponsor of the clinical program for this investigational drug.
ISIS 333611 is in Phase 1 clinical development. One Phase 1 trial has been completed, and no active trials are currently listed. The drug is investigational and has not been approved by the FDA for any use.
ISIS 333611 has been evaluated in a single Phase 1 trial, NCT01041222, titled "Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations." The trial was completed and enrolled 33 participants in the United States.
ISIS 333611 targets SOD1, the superoxide dismutase 1 gene. It is designed to reduce production of the SOD1 protein, addressing the underlying genetic cause of familial ALS associated with SOD1 mutations.