The CTRA supports studies that will move promising, well-founded preclinical and/or clinical research findings closer to clinical application, including diagnosis, prognosis, or treatment of TSC. Projects supported by this award mechanism may include, but are not limited to:
• Studies moving from preclinical to clinical research and/or the reverse, or analyzing human anatomical substances and/or data associated with completed clinical trials to understand the mechanism of action, or improve diagnosis, prognosis, or treatment.
• Studies advancing clinical trial readiness through development of biomarkers, clinical endpoints, and validation of pharmacokinetics/pharmacodynamics.
• Pilot clinical trials where limited clinical testing (e.g., small sample size) of a novel intervention to 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 may be appropriate but must include a clinical component. Projects that are strictly animal research will not be considered for funding and should consider other FY23 TSCRP funding opportunities.
Deadlines:
• Required Pre-Application Deadline: June 8, 2023
• Application Submission Deadline: July 14, 2023
The FY23 TSCRP Clinical Translational Research Award (CTRA) encourages applications that address one or more of the following Focus Areas:
• Understanding, preventing, and treating the features of TSC-Associated Neuropsychiatric Disorders (TAND) and reducing their impact, including pharmacological, behavioral, and surgical interventions
• Strategies for eradicating tumors associated with TSC and TSC-associated lymphangioleiomyomatosis (LAM), including gaining a deeper mechanistic understanding of tumor microenvironment, TSC signaling, and mTOR independent pathways
• Preventing epilepsy, improving treatment, and mitigating neurodevelopmental and adverse outcomes associated with TSC-related seizures
• Developing, assessing, and testing emerging technologies including imaging and molecular therapeutic strategies, such as gene therapy, to improve outcomes in TSC
Independent investigators at or above the level of Assistant Professor (or equivalent) are eligible to be named as a PI, Initiating PI, or Partnering PI. Each investigator may be named on only one FY23 TSCRP CTRA application as a PI, Initiating PI, or Partnering PI. An eligible PI, regardless of ethnicity, nationality, or citizenship status, must be employed by or affiliated with an eligible organization.
The anticipated direct costs budgeted for the entire period of performance for an FY23 CTRA should not exceed $1,000,000. The anticipated direct costs budgeted for the entire period of performance for an FY23 CTRA with Partnering PI Option Award should not exceed $1,150,000. Refer to Section II.D.5, Funding Restrictions, for detailed funding information.
