Department of Defense (DoD) Neurofibromatosis Clinical Trial Award

Funding from this award mechanism must support a clinical trial and may not be used for preclinical research studies. A clinical trial is defined as a prospective accrual of human subjects where an intervention (e.g., device, drug, biologic, surgical procedure, rehabilitative modality, behavioral intervention, or other) is tested on a human subject for a measurable outcome with respect to exploratory information, safety, effectiveness, and/or efficacy. This outcome represents a direct effect on the human subject of that intervention or interaction. Principal Investigators (PIs) seeking funding for a preclinical research project should consider one of the other award mechanisms/Funding Opportunities being offered by the FY16 NFRP. The term “human subjects” is used in this Program Announcement/Funding Opportunity to refer to individuals who will be recruited for or who will participate in the proposed clinical trial. For more information, a Human Subject Resource Document is provided at https://ebrap.org/eBRAP/public/Program.htm.

If the clinical trial involves the use of a drug that has not been approved by the U.S. Food and Drug Administration (FDA) for the proposed investigational use, then evidence that an Investigational New Drug (IND) application that meets all requirements under the Code of Federal Regulations, Title 21, Part 312 (21 CFR 312) has been submitted to the FDA prior to the application submission deadline, or that the drug is exempt from an IND, is required. If the investigational product is a device, evidence that an Investigational Device Exemption (IDE) application that meets all requirements under 21 CFR 812 has been submitted to the FDA prior to the application submission deadline, or that the device is exempt from an IDE, is required. The NFRP IND/IDE Documentation Form (Attachment 13) should be included in the application. Documentation of approval of the IND or IDE must be obtained and submitted to the CDMRP Help Desk (help@eBRAP.org) prior to Programmatic Review; otherwise, the Government reserves the right to withdraw the application.

The following are important aspects of submission for the Clinical Trial Award:
• The proposed clinical trial is expected to begin no later than 6 months after the award date. The 6 months includes the time required for regulatory review and approval processes.
• The application should include a clearly articulated statistical analysis plan appropriate to the phase of the clinical trial, a power analysis reflecting sample size projections that will clearly answer the objectives of the study, and appropriate statistical expertise.
• The application must demonstrate documented availability and accessibility of the drug/compound, device, and/or other materials needed, e.g., a letter from the manufacturer assuring an adequate supply of the agent (and placebo, if necessary).
• The application must demonstrate documented availability of, and access to, a suitable patient population that will support a meaningful outcome for the study. The PI should discuss how accrual goals will be achieved and maintained and how standards of care may impact the study population.
• The application must include, as appropriate, a study coordinator(s) who will guide the clinical protocol through the local Institutional Review Board (IRB) of record and other regulatory approval processes, coordinate activities from all sites participating in the clinical trial, and coordinate participant accrual.
• The application should include a transition plan (including potential funding and resources) showing how the product will progress to the next clinical trial phase and/or delivery to the market after the successful completion of the NFRP Clinical Trial Award.
• The application should clearly demonstrate strong institutional support and commitment.
• The proposed clinical trial design should include clearly defined and appropriate endpoints, and follow Good Clinical Practice (GCP) guidelines.
• The application should include a clearly articulated clinical monitoring plan, outlining how the study will be monitored for GCP compliance.
• The application should include a clearly articulated data management plan and use of an appropriate database to safeguard and maintain the integrity of the data.
• The application should include a clearly articulated safety management plan, outlining how safety pharmacovigilance will be conducted as applicable.
• The application should acknowledge commitment to filing the study in the NIH clinical trials registry, www.clinicaltrials.gov.
Multi-Institutional Clinical Trials: If the proposed clinical trial is multi-institutional, plans for communication and data transfer among the collaborating institutions, as well as how specimens and/or imaging products obtained during the study will be handled, should be included in the appropriate sections of the application. A separate intellectual and material property plan agreed upon by all participating institutions is also required for multi-institutional clinical trials. Refer to Attachments 8 and 9 for additional information.