Engaging ALS patients and caregivers (the ALS research ambassadors) to help design the REFINE-ALS biomarker study

Figures & data

Table 1 Summary of information exchange with the ALS Research Ambassadors on the REFINE-ALS study design.

Information provided to the Ambassadors	Topics for feedback
Study goals	Best ways to recruit patients for the study
Study design, including schedule of study visits	Which clinical assessments are of most interest to patients
Number of patients to be enrolled and enrollment criteria	Anticipated interest level by patients in the ALS community
Clinical assessments to be used in the study (reviewed in greater detail during the second meeting) Definition of a biomarker, types of biomarkers that are generally available, and types of biomarkers being used in the study Option for DNA analysis and genome sequencing to identify the presence of known genes that may increase risk for ALS (reviewed during the second meeting)	How patients would prefer being informed of individual assessment results and overall study outcomes, and the best timing for communication of individual results Level of interest in obtaining DNA sequencing and genome analysis results

Figure 1 Changes to the original study protocol based on feedback from advisory board meetings with the Research Ambassadors.

Table 2 Summary of actionable key feedback from the Research Ambassadors and changes implemented to the REFINE-ALS study design as a result.

Research Ambassador feedback		Revisions to study design and procedures
Advisors were disappointed to learn that patients currently receiving edaravone would be ineligible	→	Develop a speaking points document to explain the exclusion of patients already receiving edaravone
Clarify which experimental treatments (eg, coconut oil) would be disqualifying	→	Develop a list of investigational drugs that would preclude enrollment in the study
Reduce the frequency of blood collections	→	Institute a protocol amendment to eliminate visits at cycles 2, 4, and 5
Travel expenses are a concern	→	Revise the budget to cover patient travel, as appropriate
Permit patients to receive clinical results in real time	→	Develop a template for return of clinical results to participants
Permit patients to receive genetic results and prediction model	→	Work with New York Genome Center and Origent to include these options