Newron Pharmaceuticals has completed patient enrolment in the pivotal Sarizotan for the Treatment of Apneas in Rett Syndrome (STARS) clinical trial.
Rett syndrome is a severe neuro-developmental orphan disease that currently has no approved treatment options. It usually affects girls at a very young age.
The STARS trial has enrolled more than 130 patients aged four years or older and who present clinically significant apneas during the course of the disease.
Around 70% of the patients present apneas, a cardinal feature of Rett syndrome, as well as cause co-morbidities and reduce quality of life.
The STARS trial is being conducted at 14 centres across the US, Europe, Asia and Australia. Top-line results from the trial are expected to be available in the last quarter of this year.
Newron in a statement said: “Only patients who experienced clinically significant apnea, ie. at least ten episodes of more than ten seconds each per hour while awake, qualified for inclusion in the study.
“The apneas were monitored and recorded using a state-of-the-art medical device providing an objective measure of breathing dysfunction.
“The recordings were performed over a five to six-hour period per day, for three days per week, with the opportunity for patients to repeat the procedure in the subsequent three weeks in case they did not qualify in the first week of screening.”
As part of the six-month-long STARS trial, Newron randomised patients to receive daily doses of 10mg or 20mg sarizotan or placebo.
Newron recorded respiration of the patients at their homes at four different time-points during the trial’s ongoing 24-week double-blind period.
The primary objective of the trial is the percentage reduction in the episodes of apnea during waking time compared to placebo.
Sarizotan has so far been found to be well tolerated among the patients with a very low rate of discontinuation due to adverse events or lack of efficacy.