GLIA-CTN Pilot Project Award

Pilot projects, also known as demonstration projects, are key to identifying new clinical research opportunities within the field. Our consortium will be providing funding and resources for studies proposed by scientists within the GLIA-CTN, as well as external research collaborators. There will be at least one project running at all times, and no project will take more than two years.

The primary goal of the GLIA-CTN Pilot Project Award is to generate preliminary research data needed to secure more substantial funding through traditional federal, institutional, and/or industry grant mechanisms. Recognizing the importance of creating a sustainable infrastructure for collaborative research and excellence in clinical care, the GLIA-CTN Pilot Project Award is designed to enhance clinical trial readiness in the leukodystrophies through clinical/translational research in biomarkers, or assays to assess therapeutics, or other initiatives directed towards clinical trial readiness

Applications must be received no later than February 21, 2023 (11:59PM EST).

At least two GLIA-CTN Pilot Project Awards will be funded annually. Additional awards may be granted during a single funding period at the discretion of the GLIA-CTN Career Development Committee, and as permitted by available funding.

Support will be provided for a one-year period.

The GLIA-CTN Pilot Project Award will provide up to $20,000 for research and salary, inclusive of appropriate fringe and indirect costs. We recommend that the applicant ask for a waiver of indirect costs. No greater than 8% indirect costs are permitted.

Candidates for the award must have a doctoral degree within the health professions (eg, MD, DO, DDS, DMD, OD, DC, PharmD) and be actively affiliated with a US-based academic and/or health care institution. Individuals with degrees in nursing research and/or practice, who are licensed to practice clinically, may be eligible. There are also certain circumstances in which individuals with a PhD may be eligible, outlined below.

Innovative, early-stage applications addressing key knowledge gaps that are without evidence of alternate means of funding (ie, “high-risk/high-yield”) are encouraged.

Eligible applications will meet the following key criteria.

• The application must relate to a specific established leukodystrophy (see Vanderver et al., Mol Genet Metab. 2015; doi: 10.1016/j.ymgme.2015.01.006);
• All projects must be performed using patient samples, imaging or clinical data. Please contact the GLIA-CTN to assess that the funding proposal is responsive to the overall funding structure of the Rare Disease Clinical Research Network (RDCRN) prior to submission;
• The applicant must demonstrate willingness to collaborate with the GLIA-CTN in a sustainable way, including adoption of centralized/standardized data collection tools managed by the GLIA-CTN Data Integration Core (DIC);
• Applicants holding a PhD only must demonstrate integration with the clinical team at their site, as well as partnership with clinicians to facilitate the establishment of a local Leukodystrophy Center;
• The applicant must describe scientific methods that demonstrate adequate rigor and replication to translate the proposed research project into future clinical studies (ie, description of alternative approaches or “Go/No-Go” criteria).

Applications should also meet the following feasibility criteria.

• The applicant must demonstrate approval by their local Institutional Review Board (IRB) and, if appropriate, their Institutional Animal Care and Use Committees (IACUC) prior to accepting award funding.
• For pilot projects involving interventional approaches, the application must include a protocol synopsis and evidence of submission to appropriate regulatory bodies prior to submission.

At this time, only US-based investigators are eligible to apply for funding through a GLIA-CTN Pilot Project Award. We hope to be able to include international applicants in future award cycles. In the meantime, alternative funding mechanisms may be available to support international researchers interested in proposing either a new pilot project or collaborating on an existing project within one of the key disease areas outlined in Appendix A of the Request for Applications (RFA), available for download here. Please contact GLIA-CTN Program Manager, Omar Sherbini, MPH, at email@theglia.org for additional details.

Applications that include a plan to leverage central GLIA-CTN resources in one or more of the following ways will be prioritized:

• Use of existing phenotype and longitudinal natural history previously collected through the consortium’s central repository, known as the Myelin Disorders Biorepository Project (MDBP);
• Use of banked and/or prospectively collected biospecimen obtained by one or more clinical research centers currently participating in the GLIA-CTN;
• Inclusion of one or more GLIA-CTN Principal and/or Site Investigators in pilot project design and/or execution;
• Inclusion of one or more GLIA-CTN Advocacy Committee Members/Organizations in pilot project design and/or execution.

Standard reporting requirements include the following.

• A progress report will be expected at 6 months following award, and a final report will be expected at 12 months following award;
• Funds will be disbursed at the end of each 6-month reporting period;
• Publication in the form of an abstract at a national or international meeting or submission of a manuscript for publication is expected within 12 months of completion of the award;
• The awardee is expected to attend, and be prepared to discuss their pilot project, at the GLIA-CTN Annual Instigator Meeting (Summer 2023) and attend the next GLIA Scientific Meeting (Spring 2024) after completion of their award.

IRB/IACUC protocols must include language that permits protected health information (PHI) to be shared with the Children’s Hospital of Philadelphia (CHOP), and with the Rare Diseases Clinical Research Network (RDCRN) Data Management and Coordinating Center (DMCC) designated by the National Institutes of Health.

The GLIA-CTN Career Development and Pilot Project Committee recommends the following application structure.

• Pilot Proposal (2-3 Pages): Background, Innovation, Approach (inc. Specific Aims, Preliminary Data and Methods), Project Timeline, and Future Directions;
• Bibliography (1 Page)

The following criteria will be considered during the review process.

• Does the application meet the eligibility requirements?
• Does the proposed research have the potential to improve care of patients?
• Are the aims well defined?
• Is the approach innovative?
• Are the methods appropriate?
• Will the collected data answer the stated aims? 
• Is the budget adequate and the timeline realistic?
• Is there strong institutional support for creation of a local Leukodystrophy Center?

All applications will undergo the following two-stage process.

• Applicants will submit a first-round application for review by the GLIA-CTN Career Development and Pilot Project Committee. Feedback will be provided to all applicants.
• Applicants with strong proposals will be invited to incorporate the Committee’s feedback and submit a revised application for a second round of review.

Questions regarding the application requirements, submission guidelines, etc. may be directed to GLIA-CTN Program Manager, Omar Sherbini, MPH at email@theglia.org.