The MSRP CTA supports the rapid implementation of clinical trials with the potential to have a significant impact on the treatment or management of multiple sclerosis. Clinical trials may be designed to evaluate promising new products, pharmacologic agents (drugs or biologics), devices, clinical guidance, and/or emerging approaches and technologies. Proposed projects may range from small proof-of-concept trials (e.g., pilot, first-in-human, phase 0) to demonstrate the feasibility or inform the design of more advanced trials, through large-scale trials to determine efficacy in relevant patient populations.
The FY24 MSRP CTA offers two funding levels (Refer to Section II.D.5, Funding Restrictions). Only one funding level may be chosen per application and the choice of application category is at the discretion of the applicant. The requested budget at each funding level must be justified and appropriate to the scope of the clinical trial proposed. The following are generalized descriptions of the scope of research appropriate for each funding level:
• Funding Level 1 (CTA-FL1) supports small-scale, early-phase, proof-of-principle clinical trials to demonstrate feasibility or inform the design of more advanced trials, or other clinical trials that are appropriate for this funding level. Preliminary data relevant to the proposed clinical trial, preferably in subjects with MS, are required.
• Funding Level 2 (CTA-FL2) supports larger-scale clinical trials at phase 1 or phase 2 that seek to show preliminary evidence of safety or efficacy (i.e., benefit of clinical or paraclinical outcomes) in relevant patient populations. Strong justification should be provided, which could include, but is not limited to, intervention type, trial duration, sample size, outcome measures, assessment tools, and frequency of assessment. Preliminary data relevant to the proposed clinical trial, preferably in subjects with MS, are required.
For the purposes of this funding opportunity, Regulatory Agency refers to the U.S. Food and Drug Administration (FDA) or any relevant international regulatory agency unless otherwise noted.
Key aspects of the FY24 MSRP CTA Award Mechanism:
• Clinical Trial Start Date: The proposed clinical trial is expected to begin no later than 12 months after the award date or 18 months after the award date for studies regulated by the Regulatory Agency.
• Preliminary Data Are Required: Inclusion of preliminary data relevant to the proposed clinical trial is required.
• Study Population: The application should demonstrate the availability of and access to a suitable patient population that will support a meaningful outcome for the study. The application should include a discussion of how accrual goals will be achieved, as well as the strategy for inclusion of women and minorities in the clinical trial appropriate to the objectives of the study. Studies utilizing human biospecimens or datasets that cannot be linked to a specific individual, gender, ethnicity, or race (typically classified as exempt from Institutional Review Board [IRB] review) are exempt from this requirement.
• Intervention Availability and Safety: The application should demonstrate the documented availability of and access to the drug/compound, device, and/or other materials needed, as appropriate, for the proposed duration of the study. Clearly identify all study risks, including potential safety concerns and adverse events. Describe all safety measures to minimize and/or eliminate risks to human subjects and study personnel or to manage unpreventable risks. Discuss the overall plan for the provision of emergency care or treatment for an adverse event for study-related injuries, including who will be responsible for the cost of such care.
• Personnel and Environment: The application should demonstrate the study team’s expertise and experience in all aspects of conducting clinical trials, including appropriate statistical analysis, knowledge of FDA processes (if applicable), and data management. The application should include a study coordinator(s) who will guide the clinical protocol through the local IRB of record and other federal agency regulatory approval processes, coordinate activities from all sites participating in the trial, and coordinate participant accrual. The application should show strong institutional support and, if applicable, a commitment to serve as the FDA regulatory sponsor, ensuring all sponsor responsibilities described in the Code of Federal Regulations, 21 CFR 312, Subpart D, are fulfilled.
• Statistical Analysis and Data Management Plans: The application should include a clearly articulated statistical analysis plan, a power analysis reflecting sample size projections that will answer the objectives of the study, and a data management plan that includes the use of an appropriate database to safeguard and maintain the integrity of the data. If required by a Regulatory Agency, the trial must use a 21 CFR 11-compliant database and appropriate data standards.
