Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)


Leukodystrophies are a complex, often progressive group of disorders affecting the white matter of the brain due to the loss or absence of myelin, the lipid membrane that insulates axons in the central nervous system. Despite advances in the diagnosis of these disorders, they remain widely under-recognized, with unmet gaps in clinical care and curative therapeutics. The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) is a consortium of scientists, industry stakeholders, and patient advocacy leaders working together to promote advances in the diagnosis and treatment of leukodystrophies. Specifically, it seeks to create a robust research infrastructure that will allow for collection and analysis of longitudinal natural history data, development of novel clinical outcome assessments, and identification of surrogate biomarkers, ultimately paving the way for transformative therapeutic trials across the leukodystrophies. In parallel to these approaches, the GLIA-CTN will work closely with a diverse group of stakeholders to promote disease awareness and education—including newborn screening and early diagnosis—and establish clinical guidelines to support the short- and long-term care of individuals living with leukodystrophies.

The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) is part of the Rare Diseases Clinical Research Network (RDCRN), which is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). GLIA-CTN is funded under grant number U54NS115052 as a collaboration between NCATS and the National Institute of Neurological Disorders and Stroke (NINDS). All RDCRN consortia are supported by the network’s Data Management and Coordinating Center (DMCC) (U2CTR002818). Funding support for the DMCC is provided by NCATS and NINDS.

Baylor College of Medicine
Stanford University
Children’s Hospital of Philadelphia
Emory University
Kennedy Krieger Institute
University Of Colorado Denver
University Of Utah
University Of Wisconsin-Madison

  • Global Leukodystrophy Initiative Clinical Trials Network
  • Rare Diseases Clinical Research Network (RDCRN)
  • Rare Disease
  • Aicard Goutieres Syndrome (AGS)
  • Alexander Disease (AxD)
  • Adrenoleukodystrophy
  • Adrenomyeloneuropathy (AMN)
  • Krabbe Disease
  • Metachromatic Leukodystrophy
  • Pelizaeus-Merzbacher Disease (PMD)