# Global Leukodystrophy Initiative Clinical Trials Network Gap Year Administrative Supplement

> **NIH NIH U54** · CHILDREN'S HOSP OF PHILADELPHIA · 2024 · $1,009,720

## 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) that is focused on, but not limited, to a core set of ten leukodystrophies that are at or approaching
stages of clinical trial readiness. Approaches will include Clinical Outcome Assessments (COA) and Patient
Reported Outcomes (PRO) with a focus on reliability, reproducibility and validity. Additionally, we will expand
advanced methodologies for Electronic Medical Record (EMR) extraction of natural history data for the
leukodystrophies. Oversight for these projects will occur under a well-developed Administrative Unit at the
Children’s Hospital of Philadelphia (CHOP), which includes the GLIA-CTN Data Integration Core staffed by
bioinformatics, epidemiologists, and biostatisticians. All aspects of the program are carefully aligned with our
patient advocacy partners, represented by the Chair and Co-Chair of the GLIA-CTN Patient Advocacy
Committee, who have provided meaningful input and will be stakeholders in data sharing platforms. Together,
these approaches will expand our clinical trial readiness platform across the leukodystrophies, enabling
transformation of the field.

## Key facts

- **NIH application ID:** 11084865
- **Project number:** 3U54NS115052-05S4
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** FLORIAN S EICHLER
- **Activity code:** U54 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $1,009,720
- **Award type:** 3
- **Project period:** 2024-07-01 → 2025-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11084865

## Citation

> US National Institutes of Health, RePORTER application 11084865, Global Leukodystrophy Initiative Clinical Trials Network Gap Year Administrative Supplement (3U54NS115052-05S4). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/11084865. Licensed CC0.

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