DoD Neurofibromatosis Clinical Trial Award

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The NFRP Clinical Trial Award (CTA) mechanism was first offered in FY99. Since then, 23 CTA applications have been received, and 6 have been recommended for funding. DOD FY10 Neurofibromatosis Clinical Trial Award 3 The NFRP CTA supports research with the potential to have a major impact on the treatment or management of neurofibromatosis and/or Schwannomatosis. Funding from this award mechanism must support a clinical trial. In general, a clinical trial is defined as a prospective accrual of patients for a study where an intervention (e.g., device, drug, behavioral, surgical procedure, or other) is tested on human subjects for a measurable outcome. Refer to the General Application Instructions, Appendix 5, for additional information about studies involving human subjects. The proposed clinical trial is expected to begin no later than 12 months after the award date. Preliminary data, unpublished results from the laboratory of the Principal Investigator (PI) or collaborators named on this application, that is relevant to the proposed research project is required. Proposals should also be based on a sound scientific rationale that is established through logical reasoning and critical review and analysis of the literature. Funding from this award mechanism cannot be used for preclinical research studies. PIs seeking funding for a preclinical research project should consider one of the other award mechanisms/funding opportunities being offered. Each application should contain only one clinical trial with a distinct study design. Investigational New Drug (IND) or Investigational Device Exemption (IDE) applications should be submitted or approved prior to application submission. If the study is in support of an application to the U.S. Food and Drug Administration (FDA), Investigational New Drug (IND) or Investigational Device Exemption (IDE) applications should be submitted prior to the grant application submission. The Government reserves the right to withdraw funding if an active exemption from marketing approval for the IND or IDE has not been acquired within 6 months of the award date. If an IND or IDE is required to conduct the proposed research, but is not received within 6 months of the award date, the Government reserves the right to revoke funding. For descriptions of each type of clinical trial, please refer to http://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/ Guidances/default.htm and http://www.clinicaltrials.gov. Refer to the General Application Instructions, Appendix 5, for helpful information about distinguishing clinical trials and research utilizing human anatomical substances. Again, the proposed clinical trial is expected to begin no later than 12 months after the award date. The following are important aspects of submission for the CTA: • Include a prospective accrual of subjects for a study where an intervention (e.g., drug, device, behavioral, surgical procedure, or other) is tested on human subjects for a measurable outcome. • Demonstrate availability of, and access to, a suitable volunteer population that will support a meaningful outcome for the study. Discuss how accrual goals will be achieved and how standards of care may impact the study population. • Describe clearly defined and appropriate endpoints for the proposed clinical trial. • Clearly articulate the statistical analysis plan. Include a power analysis reflecting sample size projections that will clearly answer the objectives of the study. • Discuss the potential impact of the study results for patients with neurofibromatosis or Schwannomatosis. DOD FY10 Neurofibromatosis Clinical Trial Award 4 • Include a study coordinator(s) who will guide the clinical protocol through Institutional Review Board (IRB), Human Subjects Research Review Board, and other regulatory approval processes, coordinate activities from all sites participating in the trial, and coordinate participant accrual. • Demonstrate institutional support.

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