DoD Tuberous Sclerosis Research Pilot Clinical Trial Award
The mission of the TSCRP is to encourage innovative research aimed at understanding the pathogenesis and manifestations of TSC to improve the lives of individuals with TSC. Within this context, the FY13 TSCRP encourages applications that address one or more of these vital program Focus Areas: • Genetic, epigenetic, and non-genetic modifiers of TSC. • Preclinical models and therapeutic strategies (e.g., cytotoxic agents, combination therapies). • Biomarkers for early detection, prognosis, and prediction of treatment outcomes (such as serum markers, imaging, electrophysiology, prenatal testing, and pharmacogenetics). • Impact of TSC manifestations in adults (e.g., care management, age-specific pathogenesis, epidemiology, renal, reproductive issues, and lymphangioleiomyomatosis [LAM]). • Long-term benefits and effects of mTOR inhibitors or other agents. • Novel strategies for diagnosis, treatment, and prevention of TSC manifestations including those geared toward early identification and intervention. • Cellular and molecular mechanisms of TSC and LAM pathogenesis. • Causes and treatment of epilepsy in TSC. • Causes and treatment of TSC-associated neurocognitive disorders including cognitive impairment, and psychiatric, behavioral, and sleep disorders. The TSCRP Pilot Clinical Trial Award mechanism supports studies of interventions (e.g., device, drug, biologic, surgical procedure, rehabilitative modality, behavioral intervention, or other) with the potential to have a major impact on the treatment or management of TSC. Funding from this award mechanism must support a pilot study aimed at obtaining preliminary data to support the rationale and design of subsequent clinical trials. Examples of research topics include but are not limited to the following: • Studies to identify an appropriate population. • Studies to identify the dosage, duration, and/or delivery strategy of the intervention. • Studies to evaluate the feasibility of the intervention in TSC. • Studies to better understand efficacy and safety. Pilot clinical trials may include 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.
Office: U.S. Army Medical Research Acquisition Activity
301-682-5507
help@cdmrp.org
