Phase 1 Pelizaeus-Merzbacher Disease Clinical Trials

2 recruitingPhase 1

What is a Phase 1 trial?

Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.

Showing 1–2 of 2 trials

Recruiting

Phase 1

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

Pelizaeus-Merzbacher Disease

Ionis Pharmaceuticals, Inc.24 enrolled7 locationsNCT06150716

Recruiting

Phase 1

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

Batten DiseaseAdrenoleukodystrophyPelizaeus-Merzbacher Disease+9 more

Joanne Kurtzberg, MD40 enrolled1 locationNCT02254863