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Pelizaeus-Merzbacher Disease clinical trials

View clinical trials related to Pelizaeus-Merzbacher Disease.

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NCT ID: NCT06150716 Recruiting - Clinical trials for Pelizaeus-Merzbacher Disease

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

Start date: April 10, 2024
Phase: Phase 1
Study type: Interventional

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

NCT ID: NCT05659901 Recruiting - Clinical trials for Pelizaeus-Merzbacher Disease

A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

Start date: October 3, 2022
Phase:
Study type: Observational

The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to Pelizaeus-Merzbacher disease (PMD) and longitudinal changes in performance on clinical, participant, and caregiver-reported outcome assessments to inform the development of therapies for PMD.

NCT ID: NCT03333200 Recruiting - Gaucher Disease Clinical Trials

Longitudinal Study of Neurodegenerative Disorders

Start date: January 11, 2012
Phase:
Study type: Observational

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

NCT ID: NCT03047369 Recruiting - Clinical trials for Adrenoleukodystrophy

The Myelin Disorders Biorepository Project

MDBP
Start date: December 8, 2016
Phase:
Study type: Observational [Patient Registry]

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

NCT ID: NCT02699190 Active, not recruiting - Clinical trials for Adrenoleukodystrophy

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

Start date: January 6, 2017
Phase:
Study type: Observational

Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extreme genetic heterogeneity of molecular causes. While recent work has demonstrated that whole genome sequencing (WGS), has the potential to dramatically increase diagnostic efficiency, significant questions remain around the impact on downstream clinical management approaches versus standard diagnostic approaches.

NCT ID: NCT02254863 Recruiting - Clinical trials for Mucopolysaccharidosis II

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

DUOC-01
Start date: September 2014
Phase: Phase 1
Study type: Interventional

The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 as an adjunctive therapy in patients with inborn errors of metabolism who have evidence of early demyelinating disease in the central nervous system (CNS) who are undergoing standard treatment with unrelated umbilical cord blood transplantation (UCBT). The secondary objective of the study is to describe the efficacy of UCBT with intrathecal administration of DUOC-01 in these patients.

NCT ID: NCT01391637 Completed - Clinical trials for Pelizaeus-Merzbacher Disease

Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

Start date: June 2011
Phase: Phase 1
Study type: Observational

The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).

NCT ID: NCT01005004 Completed - Clinical trials for Pelizaeus-Merzbacher Disease

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

Start date: November 2009
Phase: Phase 1
Study type: Interventional

The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).