ION356

DRUG2 trials

Sponsors

Ionis Pharmaceuticals Inc., Ionis Pharmaceuticals, Inc.

Conditions

Pelizaeus-Merzbacher Disease

Phase 1

ION356-CS1: A Phase 1b Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Patients with Pelizaeus-Merzbacher Disease

RecruitingCTIS2022-502432-39-00

Ionis Pharmaceuticals Inc.Pelizaeus-Merzbacher Disease

Start: 2024-04-10Target: 33Updated: 2025-09-02

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

RecruitingNCT06150716

Ionis Pharmaceuticals, Inc.Pelizaeus-Merzbacher Disease

Start: 2024-04-10End: 2028-06-30Target: 24Updated: 2025-12-12