Rett syndrome is a rare neurodevelopmental disorder. It affects females disproportionately, at a far greater rate than males. It causes cognitive, autonomic, and neurological dysfunction as well as a loss of motor skills. Patients have difficulty walking, communicating, and doing everyday activities. Life expectancy of those living with Rett syndrome is reduced, with quarter of patients succumbing to cardio-respiratory dysrhythmias. 70% of patients deal with episodes of apnea (disordered breathing and hyperventilation).
There are no therapies currently approved for the treatment of Rett syndrome.
A New Study
Newron Pharmaceuticals has recently announced that they have completed enrollment in their study examining sarizotan as a potential treatment for apnea within Rett syndrome. It’s called STARS which stands for Sarizotan for the Treatment of Apneas in Rett Syndrome.
Apnea is a severe symptom of this condition. Data has shown that 70% of patients, at a minimum, spend 10% of their time awake not breathing. Between 4.2 and 24 times every hour the oxygen saturation of these individuals is below 90%. Combined, this level of saturation could last up to 48 minutes every hour. Not only can this lead to other complications associated with the disease, but it significantly impacts the quality of life of patients.
The STARS study included only individuals who were experiencing a minimum of 10 episodes of apnea each hour they were awake. These episodes also had to last more than 10 seconds for them to qualify. Patients were monitored before their inclusion was granted, however they were given the opportunity to repeat the monitoring process if they did not meet the criteria initially.
In total, there were 130 patients enrolled across 14 trial centers located in Asia, Australia, Europe, as well as the United States. Patients were all at least 4 years old. They were randomized to receive sarizotan or placebo each day for 24 weeks. At 4 points during this time period, respiration was recorded. 90% of the patients who have already completed the 24 weeks have enrolled in the extension study which is open-label and long-term.
So far, sarizotan has shown tolerability, safety, and efficacy in preliminary baseline data.
Results from STARS are expected by the fourth quarter of 2019. Currently, the trial is still ongoing for the last few patients who were enrolled. If this data continues to show positive indications for sarizotan, Newron plans to file for FDA marketing authorization as well as approval from the European and Canadian regulatory bodies.
Hopefully we will continue to see positive results from sarizotan through the end of this study, which could lead to the very first approved treatment for Rett syndrome. Stay tuned to hear the final results of Newron’s trial at the end of this year.
You can read more about this trial and sarizotan’s development here.