Rett Syndrome Treatment Study
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.
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.