Itaian pharma company Newron Pharmaceuticals has completed enrollment of patients in its STARS clinical trial of Sarizotan for the treatment of apneas in Rett Syndrome.
Newron Pharmaceuticals is targeting to release top line results of the STARS clinical trial in the fourth quarter of this year.
Rett syndrome is a severe neuro-developmental disease, which currently does not have any approved treatment options. The disorder affects girls starting at a very early age.
Newron Pharmaceuticals said that over 130 patients, aged four years or older, have qualified for the , STARS clinical trial, which is being held across 14 centers in the US, Europe, Australia and Asia.
The STARS clinical study is being carried out in patients who present with clinically significant apneas during the course of the disorder.
Apneas are a primary feature of Rett syndrome, seen in nearly 70% of patients, which contribute to a great extent to other co-morbidities, and also to a lowered quality of life.
According to the Italian pharma company, only patients who had faced clinically significant apnea, that is more than 10 episodes of over 10 seconds each per hour while awake, qualified for inclusion in the STARS clinical trial.
By using a medical device providing an objective measure of breathing dysfunction, the apneas were monitored and recorded. The recordings were conducted over a 5- to 6-hour period a day, for three days per week, with the scope for patients to repeat the procedure in the following three weeks if they did not qualify in the first week of screening.
Through the six-month clinical trial, patients who met the eligibility criteria were randomly grouped for treatment with daily doses of 10 or 20 mg of sarizotan or placebo.
Recordings of respiration are taken at home, at four separate time-points through the 24-week double-blind period of the clinical trial, which is still going on for the last enrolled patients.
The primary endpoint of the STARS clinical trial is the percentage reduction in the apnea episodes during waking time in comparison to placebo.
Newron Pharmaceuticals said that so far treatment with sarizotan has been tolerated very well with a very low rate of discontinuation caused by adverse events or lack of efficacy.
Nearly 90% of the enrolled patients who had completed the 24-week double-blind period have continued in the long-term open-label extension study, said the Italian pharma company.
Newron Pharmaceuticals believes that if the results from the STARS clinical trial turn out to be positive, then it could move ahead to seek marketing authorization with regulatory agencies in the US, Canada and the European Union .