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

Ionis Pharmaceuticals, Inc.

Summary

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

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.

Eligibility

Age range: 2–12 years
Sex: All
Healthy volunteers: No

Detailed criteria

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 diagnosis of DS according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study Consortium, Inc (ESCI). 4. Has confirmation of a pathogenic or likely pathogenic SCN1A variant. 5. Must be currently receiving ≥ 1 concomitant ASM at a stable dose/regimen for ≥ 4…

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

Summary

Description

Eligibility

Interventions

Locations (6)