Italian-based biopharmaceutical company Newron Pharmaceuticals has begun the Sarizotan Treatment of Apneas in Rett Syndrome (STARS) study of sarizotan to treat Rett syndrome.
Sarizotan is a new chemical entity and a highly selective compound for specific serotonin or dopamine receptors that modulate the activity of these neurotransmitters in the brain.
STARS is a randomised, double-blind, placebo-controlled trial designed to assess the efficacy, safety and tolerability of sarizotan in treating Rett syndrome.
The trial will run for a duration of six months and is planning to enrol 129 patients who will be divided into three groups of 43 people.
Newron chief medical officer Ravi Anand said: "The initiation of the STARS study is an exciting milestone in our development programme for sarizotan, for the treatment of cardinal abnormal respiratory symptoms of Rett syndrome including episodes of apnea, hyperventilation and breathing dysrhythmia.
“Newron looks forward to a further collaboration with leading Rett syndrome researchers, experts and advocacy groups to carry out the study and help address patients’ alarming and debilitating symptoms that may contribute to morbidity and mortality in the long term.”
Rett syndrome is a severe neurodevelopmental disorder mainly affecting females, causing loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction.
Most of the patients suffering from Rett syndrome experience apnea, hyperventilation and disordered breathing.
The trial is primarily focused to minimise instances of clinically significant apneas during waking time.
Patients older than 13 years will be administered with 10mg and 20mg doses of sarizotan, or placebo on a daily basis.
Newron is expecting to release top-line results of the study next year.