NHLBI Progenitor Cell Translational Consortium – Clinical Translation Studies

NHLBI Progenitor Cell Translational Consortium – Clinical Translation Studies (Revised as of Oct 2020)

Background

The goal of the NHLBI Progenitor Cell Translational Consortium (PCTC) is to translate advances in progenitor cell biology towards application to heart, lung, and blood diseases. The initiative will focus on the use of progenitor cell-based disease models to understand disease mechanisms and to develop novel therapies, and the application of progenitor cell-based therapies for the treatment of heart, lung, and blood diseases.

Clinical Translation Studies

NHLBI is making funds available to obtain approval to proceed with or actual conduct of first-in-human or Phase I studies to foster innovative approaches to translate diagnostic or therapeutic interventions based on compelling pre-clinical work to clinical applications in collaboration with well qualified clinicians. Animal rule translation to non-human primates will be allowed. Prior to the release of funds for studies in human subjects, the Principal Investigator must present US Food and Drug Administration (FDA) IND (Investigational New Drug) or IDE (Investigational Device Exception) approval or a letter from the FDA stating that the protocol is exempt from IND/IDE regulations. Or, for pre-clinical studies the Principal Investigator must present a pre-IND meeting summary letter from the FDA and the investigator’s statement specifying which of the FDA requirements that must be completed for approval of an IND or IDE application the Clinical Translation Study would address.

PCTC Clinical Translation Studies must have potential for unusually high impact to advance the field significantly. PCTC Clinical Translation Studies will be supported for a maximum of two years at $100,000/year direct costs total. PCTC Clinical Translation Studies that bridge between two or more Consortium Research Hubs will be given higher priority in order to enhance the development of synergies between Consortium investigators. The PCTC Clinical Translation Studies are also expected to be helpful in developing young investigators; we encourage applicants to include junior faculty members at participating institutions. Applications in any research area falling within the mission of the PCTC are appropriate for this funding opportunity announcement (FOA).

Requirements

The funding period for PCTC Clinical Translation Studies will be up to 2 years; it is anticipated that successful Clinical Translation Studies will be funded in continuation after completion through the home institution existing U01 funding or serve as the basis for applications for external funding from the NIH or other funding sources. The total direct cost for Clinical Translation Study applications should not exceed $100,000/year direct costs for up to 2 years (maximum award $200,000 direct costs). Principal Investigators from domestic institutions should include the accurate institutional facilities and administrative rate in their application; international institutions should include a facilities and administrative rate of 8% in their budgets. All data and meta-data generated by the Clinical Translation Study must be provided to the PCTC through the PCTC Administrative Coordinating Center (PCACC). A final Progress Report will be required. Brief interval progress summaries at six month intervals are expected (6-month Progress Reports). At the conclusion of the first year of funding and at the conclusion of the funded activity, the Clinical Translation Study PI must submit to the PCTC Administrative Coordinating Center (PCACC) a Progress Report that includes a description of activities, outcomes, and an assessment of success in meeting the stated objectives. All Progress Reports must be submitted in standard, NIH progress report format. All recipients of these funds will be required to sign an agreement that outlines PCTC rules for the sharing of information and reagents before any funds will be disbursed. Recipients will also be expected to attend the annual meeting of the PCTC. Studies must be both novel and of high quality to succeed in competition for PCTC Clinical Translation Study awards.

Eligibility

Applications for PCTC Clinical Translation Studies may come from a single institution or from a collaboration of investigators from two or more institutions. Applicants for funding of a NHLBI PCTC Clinical Translation Study must be current participants in the NHLBI PCTC. Applications will be limited to a maximum of one under review at any one time from each U01 award and must be approved by the U01 PI. PI and collaborator salary support is not permitted.

Funds available

Clinical Translation Studies for this solicitation will be funded from funds set aside through the PCACC; funding of up to 5 applications is anticipated. Depending on the number and quality of applications received, additional funds set aside through the PCACC may be added for additional meritorious applications.

Application process

Review and funding procedure

Applications should include the following information (page limits are single spaced, using font size as specified for the SF 424 application):

·     Face Page – (use the PHS 398 face page)

·     Program summary/abstract (limited to ½ page)

·     Research Plan           

·  Specific Aims (limited to 1 page)

·  Research Strategy (limited to 6 pages)

·       Significance

·       Innovation

·       Approach

·     Milestones and timeline for their accomplishment consistent with the proposal scope and award period.

·     Budget – (detailed budget for initial budget period and budget for entire proposed budget period using the PHS 398 budget forms)

·     Budget justification

·     Description of facilities, resources and equipment available to perform the proposed work

·     Biographical sketches for key personnel (including up to date personal statements)

·     Human Subjects: if non-exempt human research is included in the application, the Human Subject sections of the SF 424 should be included – see instructions

·     Vertebrate animals – if vertebrate animals are involved, address the five key points outlined in the instructions

·     hESC – if human embryonic stem cells are involved in the project, list the 4-digit NIH Registration Number of the specific cell line(s) from the NIH Human Embryonic Cell Registry, or indicate that the specific stem cell line cannot be referenced at this time.

Applications will be reviewed by a panel consisting of external experts and NHLBI extramural staff scientists. The review criteria are:

·     Significance

·     Innovation

·     Approach

·     Investigator(s)

·     Environment

To achieve the goals of the PCTC Clinical Translation Study program, increased emphasis will be placed on innovation.

If projects include human subject or animal research, the relevant sections of the application will be considered as part of the scoring process.

Funding for the Clinical Translation Studies will be provided via a subcontract from the NHLBI PCTC Administrative Coordinating Center at the University of Maryland, Baltimore.

Submitting an application

Each proposal must be submitted as a separate electronic document, i.e., a PDF, to the PCTC Administrative Coordinating Center as a PDF email attachment, with PCTC2021ClinicalTranslation01 in the file name. The title (NHLBI Progenitor Cell Translational Consortium – Clinical Translation Studies) and number (RFP PCTC2021ClinicalTranslation01) must appear on the title page of the submission. Each application must have a D&B Data Universal Numbering System (DUNS) number as the universal identifier on the title page and completed face page, check list, biosketches and budget justification in the format of the U.S. Department of Health and Human Services Public Health Service Grant Application (PHS 398). 

The mechanism of awards will be fixed cost subcontracts from the NHLBI Progenitor Cell Translational Consortium (PCTC) Administrative Coordinating Center Cooperative Agreement with the National Heart, Lung, and Blood Institute (NHLBI). Base budgets on a January 1, 2021, start date. Justified pre-award expenditures up to 90 days may be allowed. The subcontract will require compliance with the U.S. Public Health Service (PHS) Policy on Humane Care and Use of Laboratory Animals, Protection of Human Subjects, Research Using Human Embryonic Stem Cells, and other required federal citations. All data and meta-data generated by the Clinical Translation Study must be provided to the PCTC through the PCTC Administrative Coordinating Center. Principal Investigators of funded Clinical Translation Studies will be expected to attend annual PCTC meetings. If the proposal involves more than one Project Director (PD)/Principal Investigator (PI) or consortium arrangements, a Multiple PD/PI plan and consortium plan as necessary must be included. The contact PI must be specified in the Multiple PD/PI plan and with contact information on the title page.

Questions

Questions concerning submission of proposals for the Clinical Translation Studies should be submitted in writing to Dr. Terrin by email (mterrin@som.umaryland.edu) or FAX (410-706-4400). Dr. Terrin may be contacted at (410) 262-7841.