NCT07531745

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome

Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 32 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 2 Years – 12 Years
• Healthy volunteers: Not accepted

Summary

The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).

Detailed description

This is an open-label study of ION337 in people with DS between the ages of 2 and 12 years old (inclusive). The study consists of 2 parts: Part 1) 6-month single ascending dose (SAD) and Part 2) 24-month multiple ascending dose (MAD), followed by a 7-month safety follow up.

Conditions

• Dravet Syndrome

Interventions

Type	Name	Description
DRUG	ION337	ION337 will be administered by ITB injection.

Timeline

• Start date: 2026-04-01
• Primary completion: 2030-12-01
• Completion: 2030-12-01
• First posted: 2026-04-15
• Last updated: 2026-04-15

Regulatory

• FDA-regulated drug study