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ION337 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Part 1: Single Ascending Dose (SAD): Dose Level 1 | EXPERIMENTAL | Participants aged 2 to ≤ 12 years will receive a single intrathecal bolus (ITB) injection of ION337. |
| Part 1: SAD: Dose Level 2 | EXPERIMENTAL | Participants aged 2 to ≤ 12 will receive a single dose of ION337. |
| Part 1: SAD: Dose Level 3 | EXPERIMENTAL | Participants aged 2 to ≤ 12 will receive a single dose of ION337. |
| Part 1: SAD: Dose Level 4 | EXPERIMENTAL | Participants aged 2 to ≤ 12 will receive a single dose of ION337. |
| Part 2: Multiple Ascending Dose (MAD): Dose Level 1-4 | EXPERIMENTAL | Only participants who complete Part 1 will be eligible to participate in Part 2. Participants will receive multiple doses of ION337. Participants will begin treatment at the same dose level assigned in Part 1. |
| Name | Type | Description |
|---|---|---|
| ION337 | DRUG | ION337 will be administered by ITB injection. |
Key Inclusion Criteria: 1. Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consent. 2. Participant has at least 1 parent or caregiver ≥ 18 years old who is willing and able to provide informed consent (signed and dated) and attend all scheduled study visits. 3. Has a documented di...
ION337 is an investigational small molecule being developed for the treatment of Dravet Syndrome, a severe genetic epilepsy. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities. The drug is being studied to evaluate its safety and tolerability in patients with this condition.
ION337 targets the SCN1A gene, which encodes the sodium channel Nav1.1. Mutations in SCN1A are the primary cause of Dravet Syndrome. By targeting SCN1A, ION337 aims to address the underlying genetic basis of the disorder, though its precise mechanism of action is still under investigation.
ION337 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. Ionis is responsible for the clinical development and manufacturing of this investigational drug candidate for Dravet Syndrome.
ION337 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies. The ongoing Phase 1 trial is designed to assess the safety, tolerability, and preliminary effects of ION337 in patients with Dravet Syndrome.
ION337 is being evaluated in a Phase 1 clinical trial with the identifier NCT07531745, titled "ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome." This trial is currently recruiting participants in the United States and Canada, with an enrollment target of 32 patients aged 2 years and older.