Pelizaeus-Merzbacher Disease (DBCOND0002551)

Identifiers

Synonyms

Pelizaeus-Merzbacher disease / Pelizaeus-Merzbacher disease (disorder)

Associated Data

Indicated Drugs and Targets

Not Available

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT02699190	LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies	No drug interventions	Not Available	Not Available	active_not_recruiting
NCT03333200	Longitudinal Study of Neurodegenerative Disorders	No drug interventions	Not Available	Not Available	recruiting
NCT05659901	A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease	No drug interventions	Not Available	Not Available	recruiting
NCT03047369	The Myelin Disorders Biorepository Project	No drug interventions	Not Available	Not Available	recruiting
NCT01391637	Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)	No drug interventions	Not Available	Not Available	completed
NCT06150716	Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)	ION356	treatment	1	recruiting
NCT02254863	UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells	No drug interventions	treatment	1	recruiting
NCT01005004	Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects	No drug interventions	Not Available	1	completed