Italian pharma company Newron completes patient enrollment for sarizotan STARS trial in Rett syndrome

Newron Pharmaceuticals, an Italian biotech company, has successfully completed patient enrollment in its pivotal STARS clinical trial, which is investigating sarizotan as a potential treatment for apneas in Rett syndrome. This rare and severe neuro-developmental disorder, which predominantly affects young girls, currently has no approved therapies.

The company aims to release top-line results from the trial by the fourth quarter of this year, potentially bringing much-needed hope to patients suffering from the debilitating effects of Rett syndrome.

Over 130 patients enrolled globally

The STARS clinical trial is a significant step forward, enrolling over 130 patients, aged four years and older, across 14 centers in the US, Europe, Australia, and Asia. The study targets patients experiencing clinically significant apneas, a primary symptom of Rett syndrome, which affects nearly 70% of patients. Apneas, or breathing interruptions, often contribute to other severe health complications and significantly degrade the quality of life.

Inclusion in the trial was based on strict criteria, with only patients experiencing more than 10 apnea episodes lasting over 10 seconds per hour during wakefulness qualifying. This high threshold ensures that the study specifically targets those with the most significant symptoms.

Monitoring apneas with advanced medical devices

To accurately assess the extent of the apneas, patients were monitored using medical devices that objectively measure breathing dysfunction. These measurements were taken over a 5- to 6-hour period each day for three days per week, with follow-up opportunities for patients who initially did not meet the screening criteria.

This six-month clinical trial involves a double-blind design, where eligible patients were randomly assigned to receive daily doses of sarizotan (either 10 mg or 20 mg) or a placebo. Respiration recordings were taken at home at four separate time-points throughout the 24-week period. The trial is still ongoing for the final enrolled patients.

Primary endpoint: Apnea reduction

The primary goal of the study is to measure the percentage reduction in apnea episodes during waking hours, comparing the effects of sarizotan to the placebo. Early signs from the trial suggest that sarizotan is well-tolerated by participants, with a very low rate of discontinuation due to adverse effects or lack of efficacy.

Encouragingly, nearly 90% of patients who completed the 24-week double-blind phase have opted to continue in the long-term open-label extension study. This high level of continued participation is a positive indicator of sarizotan’s potential as an effective treatment for Rett syndrome apneas.

Looking ahead: Regulatory approvals

If the STARS trial results prove successful, Newron Pharmaceuticals is poised to seek marketing authorization for sarizotan from regulatory bodies in the US, Canada, and the European Union. The company’s progress in this area could mark a pivotal moment in the treatment of Rett syndrome, offering the first approved therapy for apneas associated with this debilitating disorder.