Daniel Glaze, Medical Director of the Blue Bird Circle Rett Center and a Professor in the Departments of Pediatrics and Neurology at Baylor College of Medicine in Houston, discusses ongoing Rett syndrome research and clinical trials, including the ongoing phase 3 STARS trial.
The STARS clinical study (Sarizotan Treatment of Apneas in Rett Syndrome) in patients with Rett syndrome is expected to complete enrollment in H2 2018, and Newron expects to report results from the STARS study in H1 2019.
Currently over 100 patients, aged six years and over, have qualified for inclusion into the trial. Based on the current rate of screening and qualification, Newron anticipates that randomization of the target number of 129 patients will be completed in H2 2018. The STARS study is being performed in patients who present with clinically significant apneas during the course of the disease. Apneas are a cardinal feature of Rett syndrome, present in approximately 70% of patients, and contribute significantly to other co-morbidities, as well as leading to a reduced quality of life.
Sarizotan is a highly selective compound modulating the activity of specific serotonin or dopamine receptors in the brain. It is currently in a the potentially pivotal STARS study for Rett syndrome and has the potential to become the first drug approved for this orphan disease, and the first therapy to become independently commercialized by Newron.
Rett syndrome is a severe neurodevelopmental disorder primarily affecting females, with an estimated prevalence ranging from 1 in 10,000 to 1 in 20,000 females. There are no approved treatments available. Rett syndrome is characterized by a loss of acquired fine and gross motor skills and the development of neurological, cognitive and autonomic dysfunction, which leads to loss of ability to conduct daily life activities, walk or communicate. Rett syndrome also is associated with a reduced life expectancy.