Frontlight ResearchEarly access
Taking people in United StatesNo phase
Collects samples and data from people with leukodystrophy worldwide
Gathers clinical data and biological samples from people with leukodystrophy for future research.
Key facts
- Who runs it
- Children's Hospital of Philadelphia
- Where
- Akron, Atlanta, Baltimore, Boston, Charlottesville, Chicago, Columbus, Houston, Los Angeles, Minneapolis, Orange, Palo Alto, Philadelphia, Pittsburgh, Rochester, Sacramento, Salt Lake City, San Diego, San Francisco, Seattle, Washington D.C., and Winston-Salem, United StatesSee the 24 sites
- Trial code
- Registry title
- The Myelin Disorders Biorepository Project
- Status
- Recruiting
- People taking part
- 12,000 people, planned
- Started
- 8 Dec 2016
- Expected to end
- 8 Dec 2030
- Type of study
- Observational
- Record updated
- 12 November 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people of any age with a suspected or confirmed leukodystrophy or another disease of the brain's white matter. Healthy controls can also join.
- Age
- Any age.
- Sex
- Men and women.
- Disease stage
- Suspected or confirmed leukodystrophy or other white matter disorder, based on brain imaging or an existing diagnosis, or a gene variant consistent with leukodystrophy.
- Biomarker or test result
- A gene variant of uncertain significance, or a genotype consistent with leukodystrophy, can also be a way in.
Main reasons for not taking part
- A diagnosis that is not a genetic white matter disorder, such as multiple sclerosis or an infection (except effects of congenital infections such as CMV)
- Cannot give consent
- Not willing to share clinical data, take part in standard assessments or give biological samples
The registry criteria, word for word
Show all 10 criteriaShow less
Who can take part
- Male or female of any age
- Suspected or confirmed diagnosis of leukodystrophy or other disorder affecting the white matter of the brain based primarily on the finding of central nervous system neuroimaging consistent with this diagnosis or on an existing diagnosis of a leukodystrophy or genetic leukoencephalopathy as defined in existing classification systems, or in the presence of variant(s) of uncertain significance or genotype consistent with leukodytrophy
- Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent
- Willingness to provide clinical data, participate in standardized assessments, and/or provide biologic samples
- Male or female of any age
- Individuals with no confirmed or suspected diagnosis of leukodystrophy or other disorder affecting the white matter of the brain (including affected patients' caregivers)
- Documentation of informed consent by the subject, parent, or legal guardian, and, if appropriate, documentation of assent
Who cannot take part
- Established diagnosis at the time of referral that is not consistent with a genetic disorder of the white matter, such as an acquired demyelinating condition (e.g. multiple sclerosis), or an infectious etiology, with the exception of sequelae of congenital infections such as CMV
- Inability to provide consent
- Inability to provide consent
Where in United States
- Akron Children's HospitalAkron, United StatesContact: Matthew Ginsberg, MDTaking people
- Emory University, Children's Healthcare of AtlantaAtlanta, United StatesContact: Stephanie Keller, MDTaking people
- Kennedy Krieger InstituteBaltimore, United StatesContact: Ali Fatemi, MD, MBATaking people
- Massachusetts General Hospital (MGH)Boston, United StatesContact: Florian Eichler, MDTaking people
- University of VirginiaCharlottesville, United StatesContact: Jamie Fraser, MD PhDTaking people
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, United StatesContact: Jennifer P Rubin, MDTaking people
- Nationwide Children's HospitalColumbus, United StatesContact: Margie Ream, MD, PhDTaking people
- Baylor College of Medicine/ Texas Children's HospitalHouston, United StatesContact: Lisa Emrick, MDTaking people
- UT Health HoustonHouston, United StatesContact: Mary Kay Koenig, MDTaking people
- Children's Hospital of Los AngelesLos Angeles, United StatesContact: Jonathan Santoro, MDTaking people
- University of MinnesotaMinneapolis, United StatesContact: Paul Orchard, MDTaking people
- Children's Hospital of Orange CountyOrange, United StatesContact: Changrui Xiao, MDTaking people
- Stanford University (Lucile Packard Children's Hospital)Palo Alto, United StatesContact: Keith Van Haren, MDTaking people
- The Children's Hospital Of PhiladelphiaPhiladelphia, United StatesContact: Omar Sherbini, MPHTaking people
- University of PennsylvaniaPhiladelphia, United StatesContact: Jennifer Orthmann-Murphy, MD, PhDTaking people
- University of Pittsburgh Medical CenterPittsburgh, United StatesContact: Deepa Rajan, MDTaking people
- Mayo ClinicRochester, United StatesContact: Radhika Dhamija, MBBSTaking people
- University of California, Davis (UC Davis Health)Sacramento, United StatesContact: William Benko, MDTaking people
- University of Utah, Primary Children's HospitalSalt Lake City, United StatesContact: Joshua Bonkowsky, MD, PhDTaking people
- University of California, San Diego Rady Children's HospitalSan Diego, United StatesContact: Jennifer Yang, MDTaking people
- UCSF Benioff Children's HospitalSan Francisco, United StatesContact: Alexander Fay, MD, PhDTaking people
- Seattle Children's HospitalSeattle, United StatesContact: Emily Shelkowitz, MDTaking people
- Children's National Medical CenterWashington D.C., United StatesContact: Laura Tochen, MDTaking people
- Atrium Health Wake Forest BaptistWinston-Salem, United StatesContact: Jennifer Harmon, MD, PhDTaking people
About the trial
What the trial wants to find out
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.
Read full original descriptionShow less
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.
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
- Plain-language title
- Collects samples and data from people with leukodystrophy worldwideGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Record updated
- 12 November 2026