DOD Amyotrophic Lateral Sclerosis, Pilot Clinical Trial Award

The Department of Defense (DOD) has introduced the Amyotrophic Lateral Sclerosis (ALS) Research Program (ALSRP) Clinical Trial Award for the fiscal year 2024. This award is meant to support clinical trials that can greatly impact ALS treatment or management. The trials could range between phase 1 and small-scale phase 2 trials.

These projects should aim to inform and direct the design of future advanced trials by studying safety, feasibility, application of biomarkers, and the therapeutic efficacy in relevant patient populations. The clinical trials should evaluate promising drugs, biologics, or devices that have a strong scientific rationale and are backed by preliminary studies or preclinical data. Also, trials focusing on patient care and ALS symptom management are encouraged.

In order for a study to receive funding from this award, it must be a clinical trial as defined by the DOD. There are other programs available for those seeking funding for preclinical research projects. Studies do not qualify as clinical trials if they do not aim to measure the safety and effectiveness of an intervention.

If a project proposes a therapeutic intervention, it must include specific biomarkers in the design of the trial. Applicants need to clearly describe how these biomarkers will improve and inform the design of future trials.

The impact of a pilot clinical trial should not be measured by whether an intervention is ready by the end of the trial, but whether outcomes can improve and speed up future larger trials or clinical care. The submitted applications can focus on specific subpopulations of ALS patients or potentially individual patients.

Efforts to improve clinical care and symptom management should have a positive effect on patients in the near term. All interventions must have a significant potential impact for those affected by ALS.

Community collaborations are crucial in optimizing the impact of the research funded by the FY24 ALSRP Pilot Clinical Trial Award. The research should cater to the needs of people with ALS, their families, and their care partners. Research teams are required to establish collaborations with community members to ensure that the research is accessible, efficient, and respectful of the patients' needs.

Applications must include at least one community partner, such as a person with ALS, a family member, caregiver, or a representative of a community-based organization. This person will provide advice and consultation throughout the project. The applicant team and the community members are expected to work together on all aspects of the project, not just attend seminars and meetings.

The clinical trial is expected to start within 12 to 18 months from the award date. The application should show that the necessary drugs, devices, and materials are readily available for the duration of the study.

Access to a suitable patient population that can support meaningful outcomes for the study is required. The application should also demonstrate the research team's expertise in conducting clinical trials and show strong institutional support.

A statistical analysis plan, a data management plan, and a transition plan showing how the interventions will progress to the next clinical trial phase or improve current standards of care after the trial is also required. All these plans should be present in the application.

The Principal Investigator (PI) will be required to provide an update on the project's progress at annual meetings, with their community collaboration partners ideally included. These meetings will be attended by the ALSRP Programmatic Panel, CDMRP staff, the USAMRAA Grants/Contracts Officer, and other stakeholders.