Summary: The fiscal year 2026 (FY26) Amyotrophic Lateral Sclerosis Research Program (ALSRP) Pilot Clinical Trial Award (PCTA) supports the rapid implementation of clinical trials with the potential to have a significant impact on the treatment or management of amyotrophic lateral sclerosis (ALS). Projects may range from phase 1 to small-scale phase 2 trials.

Applications must address one of the following focus areas:

·        Biomarker-Driven Interventions: Disease-modifying interventions, with mechanism-specific biomarkers to predict which clinical trial participants are likely to respond, demonstrate target engagement, and effects on the intended biological pathway.

·        Clinical Care: Improving aspects of clinical care and symptom management for ALS.

Distinctive Features: Funding from this award mechanism must support a clinical trial. The clinical trial should begin no later than 12 months after the award date or 18 months for U.S. Food and Drug Administration (FDA)-regulated studies.

Projects proposing a therapeutic intervention (drug, biologic, and/or device) must incorporate biomarkers specific to the intervention into the trial design.

All pre-applications and applications are required to incorporate community collaboration, as described in Section 3.2.2, to optimize research impact.

Applications must include a detailed Regulatory Strategy plan that outlines the approach for obtaining regulatory approvals, if required, specifically for the funded portion of the study. In addition, applications must provide a separate Transition Plan that describes how the outcomes of the study will be advanced to the next phase of development, beyond the scope of the funded work.

Miscellaneous

• Unrestricted