RECRUITING

The Myelin Disorders Biorepository Project

Conditions

LeukodystrophyWhite Matter DiseaseLeukoencephalopathies4H SyndromeAdrenoleukodystrophyAMNALDALD Gene MutationALD (Adrenoleukodystrophy)X-linked AdrenoleukodystrophyX-ALDAdrenomyeloneuropathyAicardi Goutieres SyndromeAGSAlexander DiseaseAlexanders LeukodystrophyAxDADLDCanavan DiseaseCTXCerebrotendinous XanthomatosesKrabbe DiseaseGALC DeficiencyGloboid LeukodystrophyTUBB4A-Related LeukodystrophyH-ABC - Hypomyelination, Atrophy of Basal Ganglia and CerebellumHBSLHBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg SpasticityLBSLLeukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder)Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate ElevationALSPCSF1R Gene MutationHCC - Hypomyelination and Congenital CataractMLC1Megalencephalic Leukoencephalopathy With Subcortical CystsMLDMetachromatic LeukodystrophyPMDPelizaeus-Merzbacher DiseasePLP1 Null SyndromePLP1 Gene Duplication | Blood or Tissue | MutationsPelizaeus Merzbacher Like DiseasePeroxisomal Biogenesis DisorderZellweger SyndromeRefsum DiseaseSalla DiseaseSialic Storage DiseaseSjögrenSjogren-Larsson SyndromeVan Der Knapp DiseaseVanishing White Matter DiseaseCharcot-Marie-ToothCMTMct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter DeficiencyAllan-Herndon-Dudley SyndromeCadasilCockayne SyndromeMultiple Sulfatase DeficiencyGangliosidosesGM2 GangliosidosisBPANLabrune SyndromeLCCMucopolysaccharidosesTBCK-Related Intellectual Disability Syndromeleukoencephalopathymyelindemyelinatingmdbp

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network

Quick Facts

Study Start:2016-12-08
Study Completion:2030-12-08
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT03047369

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Omar S. Sherbini, MPH
CONTACT
215-590-3068
sherbinio@chop.edu

Principal Investigator

Adeline Vanderver, MD
PRINCIPAL_INVESTIGATOR
Children's Hospital of Philadelphia

Study Locations (Sites)

Children's Hospital of Los Angeles
Los Angeles, California, 90027
United States
Children's Hospital of Orange County
Orange, California, 92868
United States
Stanford University (Lucile Packard Children's Hospital)
Palo Alto, California, 94304
United States
University of California, Davis (UC Davis Health)
Sacramento, California, 95817
United States
University of California, San Diego (Rady Children's Hospital)
San Diego, California, 92123
United States
UCSF Benioff Children's Hospital
San Francisco, California, 94158
United States
Children's National Medical Center
Washington, District of Columbia, 20010
United States
Emory University (Children's Healthcare of Atlanta)
Atlanta, Georgia, 30342
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205
United States
Massachusetts General Hospital (MGH)
Boston, Massachusetts, 02114
United States
University of Minnesota
Minneapolis, Minnesota, 55454
United States
Mayo Clinic
Rochester, Minnesota, 55905
United States
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, 27157
United States
Akron Children's Hospital
Akron, Ohio, 44308
United States
Nationwide Children's Hospital
Columbus, Ohio, 43205
United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15219
United States
Baylor College of Medicine (Texas Children's Hospital)
Houston, Texas, 77030
United States
UT Health Houston
Houston, Texas
United States
University of Utah (Primary Children's Hospital)
Salt Lake City, Utah, 84112
United States
Seattle Children's Hospital
Seattle, Washington, 98105
United States

Collaborators and Investigators

Sponsor: Children's Hospital of Philadelphia

  • Adeline Vanderver, MD, PRINCIPAL_INVESTIGATOR, Children's Hospital of Philadelphia

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2016-12-08
Study Completion Date2030-12-08

Study Record Updates

Study Start Date2016-12-08
Study Completion Date2030-12-08

Terms related to this study

Keywords Provided by Researchers

  • leukodystrophy
  • white matter disease
  • leukoencephalopathy
  • myelin
  • demyelinating
  • mdbp

Additional Relevant MeSH Terms

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