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Rett Syndrome Treatment Study

Clinical Trial Title: 
A randomized, double-blind, placebo-controlled, six-month study to evaluate the efficacy, safety and tolerability of sarizotan in patients with Rett syndrome with respiratory symptoms.
Clinical Trial Protocol ID: 
Clinical Trial Investigator Name: 
Peter T. Heydemann, MD
Clinical Trial Protocol Description: 

This is a prospective, 24-week, randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and efficacy of multiple oral doses of sarizotan in patients with Rett syndrome (RTT) with respiratory abnormalities.

Clinical Trial Eligibility Criteria: 

In order to participate you must meet the following criteria:

  • Are 13 years of age or older.
  • Have a body weight ≥ 25 kg.
  • Have a diagnosis of Rett syndrome based on consensus clinical criteria, confirmed by a test for MECP2 mutations (Xq28). Test will be performed at screening if results from an accredited laboratory are not available. Selection for the trial is not contingent on the results of the MECP2 test.
  • Have one or more of the following breathing dysfunctions:
    • Periodic apnea during wakefulness.
    • Intermittent hyperventilation.
    • Breath holding spells.
    • Air swallowing.
    • Forced expulsion of air or saliva.

You will be excluded from the study if any of the following criteria apply to you:

  • Have MECP2 duplications.

This is a partial list of inclusion and exclusion criteria.

Clinical Trial Area: 
Pediatric Neurological Disorders
Contact Phone: 
(312) 942-0079
Contact Name: 
Susan Rohde