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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 form of childhood epilepsy. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The drug is being studied to evaluate its safety and tolerability in patients with this condition.
ION337 is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol IONS. The company is conducting clinical research on this investigational drug for Dravet Syndrome, with the drug currently in Phase 1 development.
ION337 is currently in Phase 1 clinical development. It is an investigational drug and has not received regulatory approval. The ongoing Phase 1 trial is recruiting participants to evaluate the safety and tolerability of ION337 in patients with Dravet Syndrome.
ION337 is being studied in the ASCEND trial, registered as NCT07531745, a Phase 1 study titled "ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome." This recruiting trial has an estimated enrollment of 32 participants and is being conducted in the United States and Canada.
ION337 is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Dravet Syndrome. The ongoing clinical trial is designed to assess the safety and tolerability of the drug, and it has not yet completed the clinical development process required for regulatory approval.