Clinical Trials Directory

Trials / Conditions / Pelizaeus-Merzbacher Disease

Pelizaeus-Merzbacher Disease

8 registered clinical trials studyying Pelizaeus-Merzbacher Disease — 5 currently recruiting.

StatusTrialSponsorPhase
RecruitingOrbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administe
NCT06150716
Ionis Pharmaceuticals, Inc.Phase 1
RecruitingRocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzba
NCT05659901
Ionis Pharmaceuticals, Inc.—
CompletedLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
NCT02699190
Children's Hospital of Philadelphia—
RecruitingThe Myelin Disorders Biorepository Project
NCT03047369
Children's Hospital of Philadelphia—
RecruitingUCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-
NCT02254863
Joanne Kurtzberg, MDPhase 1
RecruitingLongitudinal Study of Neurodegenerative Disorders
NCT03333200
University of Pittsburgh—
CompletedLong-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Dise
NCT01391637
StemCells, Inc.—
CompletedStudy of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) S
NCT01005004
StemCells, Inc.Phase 1