drugset

drugset / Trial / NCT06150716

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

NCT06150716 ↗

Phase 1 Recruiting 24 enrolled Ionis Pharmaceuticals, Inc.

RandomizedSequentialOpen-labelTreatment

Summary

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

Timeline

Start: 2024-04-10
Primary completion: 2028-06
Completion: 2028-06

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ION356	Unknown	—	Intrathecal

Indications