DoD Tuberous Sclerosis Complex Pilot Clinical Trial Award

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The TSCRP Pilot Clinical Trial Award mechanism was offered for the first time in FY13. Since then, four Pilot Clinical Trial Award applications have been received and one has been recommended for funding.The TSCRP Pilot Clinical Trial Award mechanism supports exploratory studies involving limited human exposure that produce diagnostic or therapeutic information (e.g., screening studies, microdose studies), toxicity studies of an intervention, and studies to determine the mechanism of action and side effects of an intervention. These studies should be aimed at obtaining preliminary data leading to the development of interventions, as well as clinical biomarkers, and endpoints, with the potential to improve TSC outcomes.Examples of studies include but are not limited to the following:• Identification of an appropriate population.• Identification of the dosage, duration, and/or delivery strategy of an intervention.• Evaluation of the feasibility of the intervention in TSC.• Evaluation of efficacy and safety.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 an Investigational New Drug (IND) application to the FDA that meets all requirements under the Code of Federal Regulations, Title 21, Part 312 (21 CFR 312) may be required and must be submitted to the FDA prior to the application submission deadline. 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. Documentation of approval of the IND or IDE prior to Programmatic Review must be obtained and submitted to the CDMRP Help Desk (help@eBRAP.org), otherwise the Government reserves the right to withdraw the application.

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