IB1001-304: Effects of N-Acetyl-L-Leucine on CACNA1A Disorders: A Phase III, randomized, placebo-controlled, double-blind, crossover study

Condition studied

CACNA1A Disorders

Investigational medicinal product(s)

Eligibility

Primary endpoint

The primary endpoint measure is the Scale for the Assessment and Rating of Ataxia (SARA). SARA is an eight-item clinical rating scale (range 0–40, where 0 is the best neurological status and 40 the worst). It is a reliable and valid clinical scale with a high internal consistency that measures the severity of ataxia and increases with ataxia disease stage.

Endpoint detail

• Spinocerebellar Ataxia Functional Index (SCAFI) • International Cooperative Ataxia Rating Scale (ICARS) • Quality of Life EQ-5D-5L for patients ≥18; EQ-5D-Y for children <18 years • Neuro Quality of Life – Upper Extremity Function (NeuroQOL-UEF) assessed by the Patient or the Caregiver • Investigator, Caregiver (if applicable), and Patient (if able) Clinical Global Impression of Improvement (CGI-I) comparing end of Treatment Period I (Visit 4) to baseline (Visit 2).