Biorepository Project for Leukodystrophy
(MDBP Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to gather and study data and samples from individuals with leukodystrophy, a rare disorder affecting the brain's white matter. Researchers hope to identify new genetic causes, develop diagnostic tools, and improve future treatments. Individuals with a confirmed or suspected diagnosis of leukodystrophy, supported by brain imaging, may be suitable for participation. The trial also welcomes healthy individuals, such as caregivers, who do not have the disorder. As an unphased trial, participants can contribute to groundbreaking research that may enhance the understanding and treatment of leukodystrophy.
Why are researchers excited about this trial?
The Myelin Disorders Biorepository Project is unique because it focuses on collecting and analyzing biological samples from patients with myelin disorders to better understand these conditions. Unlike current treatments that primarily aim to manage symptoms, this project seeks to uncover the underlying mechanisms and potential biomarkers of myelin disorders, which could lead to more effective and targeted therapies in the future. Researchers are excited because this comprehensive data collection and analysis could pave the way for breakthroughs in diagnosing and treating myelin disorders, potentially transforming the standard of care for patients.
Who Is on the Research Team?
Adeline Vanderver, MD
Principal Investigator
Children's Hospital of Philadelphia
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Data Collection and Biorepository Development
Collection and analysis of clinical data and biological samples from leukodystrophy patients to support research projects
Next-Generation Sequencing Assessment
Assess the utility and validity of next-generation sequencing in the diagnosis of leukodystrophies
Follow-up
Participants are monitored for changes in care, clinical state, and natural history of leukodystrophy
What Are the Treatments Tested in This Trial?
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital of Philadelphia
Lead Sponsor
Ionis Pharmaceuticals, Inc.
Industry Sponsor
Dr. Brett P. Monia
Ionis Pharmaceuticals, Inc.
Chief Executive Officer since 2020
PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College
Dr. Eric Bastings
Ionis Pharmaceuticals, Inc.
Chief Medical Officer
MD
Sana Biotechnology
Industry Sponsor
United MSD Foundation
Collaborator
Calliope Joy Foundation
Collaborator
Don't Forget Me Foundation
Collaborator
Pennsylvania Department of Health
Collaborator
Foundation to Fight H-ABC
Collaborator
PMD Foundation
Collaborator
Takeda Pharmaceutical Co. Limited
Collaborator