# The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN)

> **NIH NIH U54** · CHILDREN'S HOSP OF PHILADELPHIA · 2022 · $1,397,307

## Abstract

Abstract:
Leukodystrophies are inherited diseases that affect the white matter of the brain due to the loss or absence of
myelin, the lipid membrane that insulates axons in the nervous systems. The leukodystrophy community is at a
key turning point. Novel molecular approaches have increased disease recognition. At the same time key
disorders now have potential treatment pathways. There is urgent unmet need in clinical trial readiness, lest
the promise in pre-clinical development be unable to be translated to well-designed clinical trials. This gap in
knowledge has galvanized stakeholders in the disease community. In collaboration with advocacy partners, we
have formed a research-based consortium, the Global Leukodystrophy InitiAtive Clinical Trials Network
(GLIA-CTN). In this proposal, the GLIA-CTN will embark on a longitudinal natural history, focused on but not
limited to five leukodystrophy with clinical trial readiness: Adrenomyeloneuropathy (AMN), Aicard Goutieres
Syndrome (AGS), Alexander Disease (AxD), Metachromatic Leukodystrophy and Pelizaeus Merzbacher
Disease. Approaches will include Clinical Outcome Assessments (COA) and Patient Reported Outcomes
(PRO) with a focus on reliability, reproducibility and validity. Additionally, we will develop methodology around
Electronic Medical Record (EMR) extraction of natural history data for the leukodystrophies. Companion
projects around novel tools and scales to define the ataxia seen in AMN, novel disease classification strategies
in AGS and their correlation with disease severity and biomarkers, and evidentiary criteria around GFAP (glial
fibrillary acidic protein) as a biomarker for AxD complete the proposal. Oversight for these projects will occur
under a well-developed Administrative Unit which includes a Data Integration Core staffed by bioinformatics,
epidemiologists and biostatisticians thanks to significant cost sharing with participating institutions.
Sustainability of the program is supplemented with a strong career development and pilot project program. All
aspects of the program are carefully aligned with our patient advocacy partners who have provided meaningful
input and will be stakeholders in data sharing platforms. Together, these approaches will establish a platform
to develop clinical trial readiness across the leukodystrophies, in close partnership between clinician scientists,
advocacy groups and industry partners, enabling transformation of the field.

## Key facts

- **NIH application ID:** 10442668
- **Project number:** 5U54NS115052-04
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** FLORIAN S EICHLER
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $1,397,307
- **Award type:** 5
- **Project period:** 2019-09-30 → 2024-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10442668

## Citation

> US National Institutes of Health, RePORTER application 10442668, The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) (5U54NS115052-04). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10442668. Licensed CC0.

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