DOD Amyotrophic Lateral Sclerosis, Clinical Outcomes and Biomarkers Award

The FY24 ALSRP Clinical Outcomes and Biomarkers Award (COBA) supports the development and/or validation of clinical outcomes and biomarkers to enrich clinical trials in Amyotrophic Lateral Sclerosis (ALS). Projects can be relevant to a specific therapy, a class of therapeutics, or to a specific ALS subtype (such as a particular genetic mutation) and do not have to broadly apply to all patients.

Research may include, but is not limited to:

• Target engagement biomarkers.
• Objective pharmacodynamic biomarkers to measure the biological effect of an investigational therapeutic.
• Predictive/cohort-selective biomarkers that indicate whether a specific therapy will be effective in an individual patient or patient subgroup.
• Diagnostic, prognostic, or disease progression
• Validate clinician-, observer-, patient-reported and/or performance outcomes to better support clinical trial success metrics.
• Define ALS subtypes using patient-based resources to link biosamples and/or digital data elements to rigorous molecular and clinical data.
• Realize improved strategies that better measure disease progression for people living with ALS.
• Augment biospecimens, outcome, or digital health data to an on-going clinical trial.
• Correlate clinical-trial related data (e.g., biosample, imaging, digital health data) with clinical outcomes or responses to therapies.

Use of existing well-characterized and highly curated clinical resources is encouraged. Examples of patient-based ALS resources include ongoing or completed clinical trial datasets, biorepositories of clinical specimens, registries (e.g., Centers for Disease Control and Prevention National ALS Registry and/or Biorepository; https://www.cdc.gov/als/Default.html), large omics datasets, patient-report outcomes, digital biomarker datasets, and databases of clinical data and/or metadata. Active-duty military and/or Veteran patient populations or resources should be considered. A list of suitable resources can be found on the ALSRP web page (https://cdmrp.health.mil/alsrp/resources/ALSRPresources). Other resources may be used, provided they have an adequate description of repository parameters and mechanisms for broad access.