Consensus Recommendations for Clinical Outcome Assessments and Registry Development in Ataxias: Ataxia Global Initiative (AGI) Working Group Expert Guidance

Abstract

To accelerate and facilitate clinical trials, the Ataxia Global Initiative (AGI) was established as a worldwide research platform for trial readiness in ataxias. One of AGI’s major goals is the harmonization and standardization of outcome assessments. Clinical outcome assessments (COAs) that describe or reflect how a patient feels or functions are indispensable for clinical trials, but similarly important for observational studies and in routine patient care. The AGI working group on COAs has defined a set of data including a graded catalog of COAs that are recommended as a standard for future assessment and sharing of clinical data and joint clinical studies. Two datasets were defined: a mandatory dataset (minimal dataset) that can ideally be obtained during a routine clinical consultation and a more demanding extended dataset that is useful for research purposes. In the future, the currently most widely used clinician-reported outcome measure (ClinRO) in ataxia, the scale for the assessment and rating of ataxia (SARA), should be developed into a generally accepted instrument that can be used in upcoming clinical trials. Furthermore, there is an urgent need (i) to obtain more data on ataxia-specific, patient-reported outcome measures (PROs), (ii) to demonstrate and optimize sensitivity to change of many COAs, and (iii) to establish methods and evidence of anchoring change in COAs in patient meaningfulness, e.g., by determining patient-derived minimally meaningful thresholds of change.

Effects of non-invasive brain stimulation for degenerative cerebellar ataxia: a protocol for a systematic review and meta-analysis

“…The primary clinical outcome will be cerebellar ataxia, as measured by SARA42 and ICARS scores 41. The use of these scales is recommended for assessment of cerebellar ataxia as a clinician-reported outcome measure 56. The secondary outcomes will include gait speed, functional ambulatory capacity (FAC)57 and functional independence measure (FIM),58 as well as any other reported outcomes that the reviewer considers important.…”

“… 41 The use of these scales is recommended for assessment of cerebellar ataxia as a clinician-reported outcome measure. 56 The secondary outcomes will include gait speed, functional ambulatory capacity (FAC) 57 and functional independence measure (FIM), 58 as well as any other reported outcomes that the reviewer considers important. The weighted mean difference and the mean and SD will be used for continuous data.…”