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

全球脑白质营养不良倡议临床试验网络 (GLIA-CTN)

基本信息

批准号：
10266084
负责人：
FLORIAN S EICHLER
金额：
$ 140.57万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-09-30 至 2024-06-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10266084
关键词：
Adrenomyeloneuropathy Adult Advocacy Advocate Affect Alexander Disease Ataxia Awareness Axon Bioinformatics Biological Markers Birth Blood Brain Caring Classification Clinical Clinical Trials Design Clinical Trials Network Collaborations Collection Communities Companions Computerized Medical Record Cost Sharing Data Diagnosis Disease Documentation Early Diagnosis Early identification Early treatment Epidemiologist Equilibrium Etiology Family Foundations Future Gait Gene Expression Glial Fibrillary Acidic Protein Health Expenditures Health Personnel Healthcare Hereditary Disease Individual Institution Interferons Joints Knowledge Leadership Longterm Follow-up Measurement Measures Membrane Lipids Metachromatic Leukodystrophy Methodology Mission Modeling Molecular Morbidity - disease rate Myelin Natural History Neonatal Screening Nervous system structure Outcome Pathway interactions Patient Outcomes Assessments Patient advocacy Patients Pelizaeus-Merzbacher Disease Performance Phase Pilot Projects Positioning Attribute Program Development Program Sustainability Publications Questionnaires Rare Diseases Recording of previous events Reproducibility Research Research Personnel Resources Scientist Severity of illness Signal Transduction Standardization Subgroup Surrogate Markers Syndrome System Technology Therapeutic Therapeutic Trials Time Training Translating base care burden career development clinical care clinical center clinical development clinical outcome assessment clinical research site clinical trial readiness data integration data sharing disease classification epidemiology study improved industry partner innovation leukodystrophy meetings next generation next generation sequencing novel patient advocacy group patient oriented preclinical development programs prospective pseudotoxoplasmosis syndrome rate of change sharing platform tool wearable device white matter working group

项目摘要

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.

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