About this trial
RADIAL is an algorithm which has been developed following a review of the literature on 67 autosomal recessive cerebellar ataxias (ARCA) and personal clinical experience. Frequency and specificity of each feature were defined for each autosomal recessive cerebellar ataxia, and corresponding prediction scores were assigned. Clinical and paraclinical features of patients are entered into the algorithm, and a patient's total score for each ARCA is calculated, producing a ranking of possible diagnoses. Sensitivity and specificity of the algorithm were assessed by blinded analysis of a multinational cohort of 834 patients with molecularly confirmed autosomal recessive cerebellar ataxia. The performance of the algorithm was assessed versus a blinded panel of autosomal recessive cerebellar ataxia experts. The correct diagnosis was ranked within the top 3 highest-scoring diagnoses at a sensitivity and specificity of \>90% for 84% and 91% of the evaluated genes, respectively. Mean sensitivity and specificity of the top 3 highest-scoring diagnoses were 92% and 95%, respectively. Our aim is now to validate in a prospective cohort of ARCA, the performance of RADIAL to predict the correct genetic diagnosis.
Eligibility criteria
Qualifiers
Patient, male or female, over 5 years old (no upper age limit)
Patient with cerebellar ataxia who started before the age of 40
Patient with a family history compatible with autosomal recessive inheritance (sporadic case, consanguinity, several cases in siblings)
Patient in which an acquired cause of cerebellar ataxia has been excluded
Disqualifiers
Patient in whom targeted sequencing of a panel of PMDA genes and/or exome/genome sequencing have already been performed.
Subject of a legal protection measure
Subject in exclusion period (determined by previous or current study)
Trial design
Treatments tested in this trial
- Genetic diagnosis (PMDA panel)
- Use of RADIAL algorithm