Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms — STARS  

Rett Syndrome Clinical Trial

Official title: A Randomised, Double-Blind, Placebo-Controlled 6-month Study to Evaluate the Efficacy, Safety, and Tolerability of Sarizotan in Patients With Rett Syndrome With Respiratory Symptoms

Status Terminated

Clinical Trial Summary

This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome in an initial double blind 24 week period followed by an open label treatment phase of up to 168 weeks (the latter for patients with no safety and tolerability issues).

Clinical Trial Description

This is a randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and efficacy of multiple doses of sarizotan in patients with Rett syndrome with respiratory abnormalities. The study participants will be randomized to either sarizotan between 2 and 10 mg bid or placebo bid, based on age and weight criteria.

All patients who have completed the final evaluations at Week 24 (Day 168) and have no safety or tolerability issues that would preclude continuing on the study medication and have been compliant with the trial requirements will have the option of continuing open-label treatment with Sarizotan for up to 168 weeks. ;

Related Conditions & MeSH terms

• Rett Syndrome
• Signs and Symptoms, Respiratory
• Syndrome

• NCT number: NCT02790034
• Study type: Interventional
• Source: Newron Pharmaceuticals SPA
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: October 26, 2016
• Completion date: May 4, 2020