Administrative Component of the Global Leukodystrophy Initiative Clinical Trials Unit (GLIA-CTN)

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Linked publications, trials & patents

Abstract

Summary/Abstract for the GLIA-CTN Administrative Core The mission of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) is to advance the diagnosis, management, and treatment of individuals affected by leukodystrophies, consistent with the broader principles of the RDCRN. The GLIA-CTN Administrative Core provides administrative support to accomplish these aims by uniting a global consortium across academic centers, patient advocacy groups, and industry partners. Aim 1 will maintain and expand the activities of the GLIA-CTN Administrative Core. We will use DMCC provided tools such as REDCap and Ambra Image repository as well as continued interactions with the NIH, RDCRN, and DMCC. Expansion plans include a novel DMCC/CAVATICA-based leukodystrophy registry, accessibility for a large legacy biosample set (>40,000 samples) via the NINDS-funded BioSEND repository, and dashboarding of key disease metrics for accessibility. Aim 2 will refine procedural approaches for rigorous FAIR data sharing. To ensure data is findable, we will work with the DMCC and deposit data in the NCATS ITRB. We ensure that demographic data is sufficiently detailed to allow for harmonization via global identifiers, and that it is accessible with proper authentication and authorization that protect patient privacy via the DMCC, retaining data sets. Interoperability will be ensured by aligning and integrating all data, images, genomic datasets, and samples contributed to various DMCC platforms. We use standard data language supported by SOPs to ensure that data is reusable by industry, advocacy, and clinical partners. Together, these efforts will create a secure and accessible data repository consistent with the broader goals of the RDCRN. Aim 3 will enable a community of advocacy, clinical and industry stakeholders. We will continue to work with patient advocacy groups and industry partners in projects such as dashboarding, individualized patient registries, key candidate drug trials and upcoming gene therapy trials. With RDCRN’s continued support and resources, the GLIA-CTN will continue to build a robust and collaborative translational research environment that will benefit the leukodystrophy community. The overall expected outcome of the Administrative Core is maintenance and creation of resources to support and engage a diverse group of leukodystrophy stakeholders across the common goals of improving the diagnosis, management and treatment of individuals living with leukodystrophy. We expect our team-based approach to continue to advance our common goals with broad stakeholder engagement