Phase 3 Study Launched to Investigate Bumetanide to Treat Autism | Kenneth Roberson, Ph.D.
Servier, an independent, international pharmaceutical company, and Neurochlore, a French biotechnology, announce the launch in Europe of phase 3 studies to evaluate the use of bumetanide in children suffering from Autism Spectrum Disorders (ASD).
The main aim of these trials is to evaluate the efficacy and safety of bumetanide, compared with placebo, on the core symptoms (persistent impairment in reciprocal social communication and interaction and restricted and repetitive patterns of behavior) of ASD after a 6-month treatment. Overall the total duration of each of these two trials will be one year. Following the 6-month evaluation period versus placebo all the patients will be treated with bumetanide for a period of 6 months. Each patient will thus have the opportunity to be on active treatment for at least 6 months.
For each trial, the design, the outcome measures, the eligibility criteria and the contacts and locations of the clinical centers are available at https://clinicaltrials.gov/ct2/show/NCT03715153 (study of 2- to 6-year-olds) and https://clinicaltrials.gov/ct2/show/NCT03715166 (study of 7- to 17-year-olds).
Bumetanide reduces the high concentrations of chloride observed in neurons in certain neurodevelopmental disorders like ASD. In 2017, Neurochlore published results supporting efficacy and safety from its phase 2b study with bumetanide in 6 centers in France in almost 90 children (aged 2-18 years). Bumetanide’s favorable effects on several aspects of ASD allow to envision management of autism, particularly of its core symptoms.