FY16 Tuberous Sclerosis Complex Pilot Clinical Trial Award

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The PCTA mechanism supports exploratory studies involving limited human exposure (e.g., small sample size) that produce information on diagnostic or therapeutic effectiveness, safety, tolerability, or mechanisms of action. These studies should be aimed at obtaining preliminary data leading to the development of interventions with the potential to improve TSC outcomes. Preclinical studies will not be supported by this mechanism. Examples of acceptable studies include, but are not limited to the following: • Studies designed to identify an appropriate population or subpopulation for future clinical trials • 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 Funding from this award mechanism must support a pilot clinical trial and may not be used for preclinical research studies. A clinical trial is defined as a prospective accrual of patients 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 being offered (Exploration – Hypothesis Development Award, Funding Opportunity Number: W81XWH-16-TSCRP-EHDA; Idea Development Award, Funding Opportunity Number: W81XWH-16-TSCRP-IDA, or Synergistic Idea Development Award, Funding Opportunity Number: W81XWH-16-TSCRP-SIDA). 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 proposed 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 TSCRP IND/IDE Documentation Form (Attachment 8) should be included in the application. Documentation of approval or exemption of the IND or IDE must be obtained and submitted to the CDMRP Help Desk (help@eBRAP.org) by 5:00 p.m. ET, November 1, 2016; otherwise, the Government reserves the right to withdraw the application.

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