DoD Amyotrophic Lateral Sclerosis, Therapeutic/Biomarker Pilot Trial Award

Description

The ALSRP Therapeutic/Biomarker Pilot Trial Award supports the rapid implementation of clinical trials with the potential to have a significant impact on the treatment or management of ALS. Clinical trials may be designed to evaluate promising drugs, biologics, or devices with anticipated therapeutic impact that is supported by strong scientific rationale and existing preclinical data. Projects may range from Phase 1 to small-scale Phase 2 trials and should be designed to de-risk and inform the design of more advanced trials, by investigating safety, feasibility, biomarker application, and therapeutic efficacy in relevant patient populations. Funding from this award mechanism must support a clinical trial and the trial design must include plans to generate compelling biomarker data. Principal Investigators (PIs) seeking funding for a preclinical research project or standalone biomarker development should alternatively consider one of the other FY21 ALSRP Funding Opportunity Announcements. Biomarker development and characterization can include target engagement biomarkers, pharmacodynamic biomarkers to measure the biological effect of an investigational therapeutic, and/or predictive/cohort-selective biomarkers that indicate whether a specific therapy will be effective in an individual patient or patient subgroup. Applicants must clearly describe the biomarker(s) and its potential to de-risk and improve the design of anticipated later-stage trials. For further description, see Attachment 8: Biomarker Statement. A clinical trial is defined as a research study in which one or more human subjects are prospectively assigned to one or more interventions (which may include placebo or other control) to evaluate the effects of the interventions on biomedical or behavioral health-related outcomes. More information is provided in the Human Subject Resource Document at https://cdmrp.army.mil/pubs/pdf/Human%20Subjects%20Resource%20Document.pdf. The following are important aspects of the FY21 ALSRP Therapeutic/Biomarker Pilot Trial Award: • Impact: The outcomes of the proposed project should directly and substantially de-risk and inform the design of anticipated later phase trials of the intervention under investigation. The intervention itself must offer significant potential impact for individuals affected by ALS, to include genetic or phenotypic patient subpopulations. • Rationale: Both the anticipated therapeutic impact of the intervention and the proposed clinical trial outcomes must be supported by strong scientific rationale that is established through critical review and analysis of the relevant literature and preliminary data. • Preliminary Data: Inclusion of preliminary data relevant to the proposed clinical trial is required. • Access to Patient Population: The application must demonstrate access to a suitable patient population to accomplish the project goals within the proposed timeline. The application should include a discussion of how accrual goals will be achieved and how standards of care may impact the study population. • Access to Therapeutic: The application should include documentation attesting to the availability of the drug/compound, device, and/or other materials needed, as appropriate, for the proposed duration of the study. The quality and stability of the product should be documented and commensurate with current FDA manufacturing standards applicable to the type and phase of product being developed (i.e., Quality System Regulation, Good Manufacturing Practice [GMP] guidelines). • Trial Design: The proposed clinical trial design should include clearly defined objectives and appropriate endpoints/outcome measures, and comply with current Good Clinical Practice (GCP) guidelines. • Statistical Plan: The application should include a clearly articulated statistical analysis plan, appropriate statistical expertise on the research team, and a power analysis reflecting sample size projections that will answer the objectives of the study. • Data Management Plan: The application should include a clearly articulated data management plan and use of an appropriate database to safeguard and maintain the integrity of the data. If FDA-regulated, the trial must use a 21 CFR 11-compliant database and appropriate data standards. For more on data standards, see https://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/ FormsSubmissionRequirements/ElectronicSubmissions/UCM511237.pdf. • Safety Management Plan: The application should include a clearly articulated safety management plan outlining how safety pharmacovigilance will be conducted, as applicable. • Clinical Monitoring Plan: The application should include a clearly articulated clinical monitoring plan outlining how the study will be monitored for GCP compliance. • Study Coordinator: 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. • FDA Experience: The application should detail the study team’s interactions to date with the FDA, including previous FDA submissions, if applicable. • Transition Plan: The application should include a Transition Plan (including potential funding and resources) showing how the product will progress to the next clinical trial phase and/or delivery to the market after the successful completion of the FY21 ALSRP Therapeutic/Biomarker Pilot Trial Award. • Institutional Support: The application should clearly demonstrate strong institutional support and, if applicable, a commitment to serve as the FDA regulatory sponsor, ensuring all sponsor responsibilities described in 21 CFR 312, Subpart D, are fulfilled. • Informed Consent Form: Funded trials are required to post a copy of the informed consent form used to enroll subjects on a publicly available Federal website in accordance with Federal requirements described in 32 CFR 219.

Eligibility

Grants.gov lists this opportunity under eligibility category code 99. These codes correspond to applicant types (state/local government, tribal organization, nonprofit, educational institution, individual, small business, etc.) defined in Grants.gov's own eligibility reference. See the current Grants.gov eligibility categories or check the official listing below for this opportunity's exact eligibility statement.

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Key Dates

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