Pilot Core of the Global Leukodystrophy Initiative Clinical Trial Network (GLIA-CTN)

Children'S Hosp Of Philadelphia, Philadelphia PA

Investigators

Linked publications, trials & patents

Abstract

Pilot/Feasibility Core The aim of the GLIA-CTN Pilot/Feasibility Core is to support the development of critical pilot/feasibility studies related to the improved diagnosis, care, and treatment of individuals with leukodystrophies. The Pilot/Feasibility Committee will ensure that innovative pilot/feasibility studies aimed at advancing the diagnosis, clinical trial readiness, management or treatment of leukodystrophies are selected in a fair and transparent fashion. Aim 1 will Support Novel Pilot Studies with GLIA-CTN Innovation Awards. A pilot projects will be selected annually to support pilot data generation in addition to one Career Development Award given annually to an early-stage clinical researcher. Selected projects will be reviewed by the GLIA-CTN as well as the NIH program officials for congruence with the overarching aims of the RDCRN with additional scrutiny by the DMCC to ensure consistency with FAIR data principles. The expected outcome is the creation of preliminary data sets to support future NIH funding proposals, supporting future translational research in the leukodystrophies and expanding the translational research community. Aim 2 will create enhanced Collaboration with Patient Community through Advocacy Advancement Awards. To address the evolving needs of the leukodystrophy community and patient advocacy groups, we will create the opportunity for focused engagement with concrete deliverables with a goal to support two six-month projects annually. The expected outcome is increased collaboration with the advocacy community with a renewed focus on patient-driven research. Aim 3 will offer In-Kind GLIA-CTN Community Awards. To encourage network growth, diverse membership, and an expanded leukodystrophy workforce, we will offer two annual in-kind infrastructure support pilots to groups external to the GLIA-CTN. The expected outcome is early feasibility studies for new natural history studies that can later be expanded to funded projects within the GLIA-CTN. With this portfolio of awards, the GLIA-CTN Pilot/Feasibility Core hopes to contribute to a new generation of NIH-funded research while expanding the GLIA-CTN network and support the research goals of the advocacy community. This multi-stakeholder approach, with open access, reflects the unshakeable commitment of the GLIA-CTN to enhance high quality research tools for the broader leukodystrophy community.