Our shared research infrastructure supports collection and analysis of robust clinical data and biological specimen to pave the way for transformative therapeutic trials across the leukodystrophies.
Who are we?
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.
The GLIA-CTN is fortunate to work with an incredible group of advocacy partners around the world.
The Global Leukodystrophy Initiative Clinical Trials Network is the culmination of over a decade of collaborative research efforts and partnership with advocacy organizations worldwide. These stakeholders are united in their efforts to bring increased awareness to the leukodystrophy community as they continue pursuing new clinical and scientific advances.
The GLIA-CTN currently includes nearly two dozen of the leading pediatric research institutions across the United States.
The Global Leukodystrophy Initiative actively collaborates with over a dozen other researchers around the world.
We have partnered with dozens of advocacy organizations to promote education and awareness initiatives, and to fund cutting-edge research in the leukodystrophies.
Individuals participating in a shared biorepository have helped researchers address some of the most pressing questions in the effort to develop therapies for the leukodystrophies.
The GLIA-CTN is lead by multiple investigators with with decades of clinical research expertise and extensive leadership experience.
The GLIA-CTN award is comprised of four well-defined projects designed to address specific needs within key leukodystrophies. Even though each project is distinct and highly specific, they will all rely on a single shared research platform to collect and share data in a multi-center fashion. This platform will be used to support groundbreaking research on additional leukodystrophies.
This is a large-scale natural history study intended to develop robust data collection and analysis tools - as well as validated outcome measures - that can be applied to all leukodystrophies and even other rare diseases. Project 1 will be conducted at all GLIA-CTN study sites.
This project seeks to develop and validate Clinical Outcome Assessments (COA) for Leukoencephalopathy with Brainstem and Spinal Cord Involvement and Lactate Elevation (LBSL) and explore whether Magnetic Resonance Spectroscopy (MRS) lactate levels may serve as a useful biomarker in the context of future clinical trials. Project 3 will be conducted at the Kennedy Krieger Institute (KKI) and Stanford University.
Pilot projects - also known as demonstration projects - are key to identifying new clinical research opportunities within the field. Our consortium will providing funding and resources for studies proposed by scientists within the GLIA-CTN, as well as external research collaborators. There will be at least one project running at all times, and no project will take more than two years.
This project seeks to develop newborn screening (NBS) approaches to support early diagnosis and intervention to improve outcomes for children affected by Aicardi-Goutières Syndrome (AGS).Project 2 will be conducted at the Children’s Hospital of Philadelphia (Philadelphia, PA) and the University of Washington (Seattle, WA).
This project will redefine approaches to clinical trial readiness in ultrarare disorders using Canavan Disease as a template. This includes developing predictive models, measuring comorbidities to prepare for real-world safety monitoring, and establishing real-world protocols to capture AAV toxicities. Project 4 will be conducted at the Children’s Hospital of Philadelphia (Philadelphia, PA) and Massachusetts General Hospital (Boston, MA).
Borrowing a model developed by the Leukodystrophy Care Network (LCN), the GLIA-CTN will establish a unique educational core focused on training the future generation of clinician scientists. Selected trainees will execute a pilot project in order to build disease-specific expertise, and are expected to establish a Leukodystrophy Center at their home institution that meets standard LCN criteria.
Devon Cordova (AGS Americas Association) / Read Full Letter of Support
Dave Manley (PMD Foundation) / Read Full Letter of Support
Maria Kefalas (The Calliope Joy Foundation) / Read Full Letter of Support
David McIntyre (The Bethanys Hope Foundation) / Read Full Letter of Support
