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 nine 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 will explore whether glial fibrillary acidic protein (GFAP) can be used as a biomarker for future clinical trials in Alexander Disease. Investigators working on this project also seek to create new clinical outcomes assessments tailored to this leukodystrophy. Project 3 will be conducted at all GLIA-CTN study sites.
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.
The goal of this project is to create a collection of clinical outcomes assessments specific to adrenomyeloneuropathy (AMN) that can be used in the context of future multi-center clinical trials. Project 2 will be conducted at Massachusetts General Hospital (Boston, MA) and the Kennedy Krieger Institute (Baltimore, MD).
This project will assess the ability of a novel rating scale to assess longitudinal change in Aicardi-Goutières Syndrome (AGS) and will also focus on exploring the relationship between a potential biomarkers and established clinical outcome measures. Project 4 will be conducted at all GLIA-CTN study sites.
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
