The Myelin Disorders Biorepository Project

Part of paid clinical trials in Los Angeles, California.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT03047369
Status
Recruiting
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Conditions

  • 4H Syndrome
  • ADLD
  • AGS
  • ALD
  • ALD (Adrenoleukodystrophy)
  • ALD Gene Mutation
  • ALSP
  • AMN
  • Adrenoleukodystrophy
  • Adrenomyeloneuropathy
  • Aicardi Goutieres Syndrome
  • Alexander Disease
  • Alexanders Leukodystrophy
  • Allan-Herndon-Dudley Syndrome
  • AxD
  • BPAN
  • CMT
  • CSF1R Gene Mutation
  • CTX
  • Cadasil
  • Canavan Disease
  • Cerebrotendinous Xanthomatoses
  • Charcot-Marie-Tooth
  • Cockayne Syndrome
  • GALC Deficiency
  • GM2 Gangliosidosis
  • Gangliosidoses
  • Globoid Leukodystrophy
  • H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum
  • HBSL
  • HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity
  • HCC - Hypomyelination and Congenital Cataract
  • Krabbe Disease
  • LBSL
  • LCC
  • Labrune Syndrome
  • Leukodystrophy
  • Leukoencephalopathies
  • Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)
  • Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation
  • MLC1
  • MLD
  • Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency
  • Megalencephalic Leukoencephalopathy With Subcortical Cysts
  • Metachromatic Leukodystrophy
  • Mucopolysaccharidoses
  • Multiple Sulfatase Deficiency
  • PLP1 Gene Duplication | Blood or Tissue | Mutations
  • PLP1 Null Syndrome
  • PMD
  • Pelizaeus Merzbacher Like Disease
  • Pelizaeus-Merzbacher Disease
  • Peroxisomal Biogenesis Disorder
  • Refsum Disease
  • Salla Disease
  • Sialic Storage Disease
  • Sjogren-Larsson Syndrome
  • Sjögren
  • TBCK-Related Intellectual Disability Syndrome
  • TUBB4A-Related Leukodystrophy
  • Van Der Knapp Disease
  • Vanishing White Matter Disease
  • White Matter Disease
  • X-ALD
  • X-linked Adrenoleukodystrophy
  • Zellweger Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Dec 8, 2016
Status verified
Oct 2025
Primary completion
Dec 8, 2030
Completion
Dec 8, 2030

Study Design

Enrollment
12,000 participants (estimated)

Primary Outcome Measure

Define Novel Homogeneous Groups of Patients with Unclassified Leukodystrophy [ Time Frame: 10 years from enrollment ]

Central Contacts

Locations (23)

FacilityCityStateZIPSite coordinators
Children's Hospital of Los AngelesLos AngelesCalifornia90027
Jonathan Santoro, MD
Children's Hospital of Orange CountyOrangeCalifornia92868
Changrui Xiao, MD
Stanford University (Lucile Packard Children's Hospital)Palo AltoCalifornia94304
Keith Van Haren, MD
University of California, Davis (UC Davis Health)SacramentoCalifornia95817
William Benko, MD
University of California, San Diego (Rady Children's Hospital)San DiegoCalifornia92123
Jennifer Yang, MD
UCSF Benioff Children's HospitalSan FranciscoCalifornia94158
Alexander Fay, MD, PhD
Children's National Medical CenterWashington D.C.District of Columbia20010
Jamie Fraser, MD, PhD
Emory University (Children's Healthcare of Atlanta)AtlantaGeorgia30342
Stephanie Keller, MD
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Jennifer P Rubin, MD
Kennedy Krieger InstituteBaltimoreMaryland21205
Ali Fatemi, MD, MBA
Massachusetts General Hospital (MGH)BostonMassachusetts02114
Florian Eichler, MD
University of MinnesotaMinneapolisMinnesota55454
Paul Orchard, MD
Mayo ClinicRochesterMinnesota55905
Radhika Dhamija, MBBS
Atrium Health Wake Forest BaptistWinston-SalemNorth Carolina27157
Jennifer Harmon, MD, PhD
Akron Children's HospitalAkronOhio44308
Matthew Ginsberg, MD
Nationwide Children's HospitalColumbusOhio43205
Margie Ream, MD, PhD
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Omar Sherbini, MPH
[email protected]
215-590-3068
Adeline Vanderver, MD (PRINCIPAL_INVESTIGATOR)
Laura Adang, MD, PhD, MSTR (SUB_INVESTIGATOR)
Amy Waldman, MD, MSCE (SUB_INVESTIGATOR)
University of PennsylvaniaPhiladelphiaPennsylvania19104
Jennifer Orthmann-Murphy, MD, PhD
University of Pittsburgh Medical CenterPittsburghPennsylvania15219
Deepa Rajan, MD
Baylor College of Medicine (Texas Children's Hospital)HoustonTexas77030
Lisa Emrick, MD
UT Health HoustonHoustonTexas-
Mary Kay Koenig, MD
University of Utah (Primary Children's Hospital)Salt Lake CityUtah84112
Joshua Bonkowsky, MD, PhD
Seattle Children's HospitalSeattleWashington98105
Emily Shelkowitz, MD

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Children's Hospital of Los Angeles· Los Angeles, CAChildren's Hospital of Orange County· Orange, CAStanford University (Lucile Packard Children's Hospital)· Palo Alto, CAUniversity of California, Davis (UC Davis Health)· Sacramento, CAUniversity of California, San Diego (Rady Children's Hospital)· San Diego, CAUCSF Benioff Children's Hospital· San Francisco, CA

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