NCT06150716

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

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 24 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: Male
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

This is a Phase 1b, open-label multiple-ascending dose (MAD) study of ION356 in approximately 24 pediatric participants with Pelizaeus-Merzbacher Disease and genetic confirmation of proteolipid protein 1 (PLP1) gene duplication. The study will have 2 parts: a 48-week multiple-ascending dose (MAD) part followed by a long-term extension (LTE) part of 109 weeks. Eligible participants will receive doses of ION356 during the MAD portion of the study and upon completion will seamlessly transition to the open-label LTE to receive doses of ION356.

Conditions

• Pelizaeus-Merzbacher Disease

Interventions

Type	Name	Description
DRUG	ION356	Administered as intrathecal (IT) injection.

Timeline

• Start date: 2024-04-10
• Primary completion: 2028-06-01
• Completion: 2028-06-01
• First posted: 2023-11-29
• Last updated: 2025-12-12

Locations

7 sites across 5 countries: United States, France, Germany, Japan, Netherlands

Regulatory

• FDA-regulated drug study