To provide evidence-based recommendations for the selection of MRI end-points in clinical trials for spinocerebellar ataxias (SCA1, SCA2, SCA3) and Friedreich ataxia, in the context of emerging disease-modifying therapies.
Key Findings:
Quantitative neuroimaging measures can serve as viable candidate biomarkers for ataxia pathology and progression.
Current clinical outcome assessments (COAs) require large sample sizes, which poses challenges for clinical trial feasibility.
MRI end-points can provide objective, reproducible, and sensitive measures that may correlate with clinical outcomes.
Interpretation:
Limitations:
Further research is needed to address knowledge gaps regarding the clinical relevance of imaging measures, including specific areas where evidence is lacking.
The consensus focuses on specific ataxia types, which may limit generalizability to other forms of ataxia.
Conclusion:
The consensus statement provides a framework for integrating MRI end-points into clinical trials for hereditary ataxias, emphasizing the need for further validation and research.
