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

Stopped early · Phase 2/Phase 3 · Has a placebo group

Conditions studied: Rett Syndrome

In brief

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).

Key facts

Study ID

NCT02790034

Run by

Newron Pharmaceuticals SPA

People needed

129

Starts

2016-10-26

Expected to finish

2020-05-04

Last updated by the study team

2021-12-21

Who can join

Age: 4 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Body weight ≥ 10 kg
• Age ≥ 4 years
• Diagnosis of Rett syndrome based on consensus clinical criteria and patients with MECP2 duplications will not be eligible.
• Has at least 10 episodes of breathing dysrhythmia, defined by episodes ≥10 seconds of breath holding (apnea), per hour during cardiorespiratory monitoring
• Ability to take study medication provided either as capsules or combined with food/drink.
• Patient is cooperative, willing to complete all aspects of the study, and capable of doing so with assistance of a caregiver.

You may not qualify if…

• Meets any of the diagnostic exclusion criteria for Rett syndrome, Typical (Neul et al, 2010);
• Patient is participating in a clinical trial with another investigational drug
• Hypersensitivity to sarizotan or other 5-HT1a agonists;
• Current clinically significant (as determined by Investigator) cardiovascular, respiratory (e.g. severe asthma), gastrointestinal, renal, hepatic, hematologic or other medical disorders, in addition to those directly related to the patient's Rett syndrome;
• QTcF interval on the ECG is greater than 450 msec.
• Surgery planned during the study (except for insertion of gastrostomy tube);
• Severe diabetes mellitus or fatty acid oxidation disorder.
• Ophthalmologic history including any of the following conditions: albino patients, family history of hereditary retinal disease, retinitis pigmentosa, any active retinopathy or severe diabetic retinopathy.
• Females who are pregnant, breastfeeding, or of childbearing potential and not using a hormonal contraceptive.

Where it is running

• University of Alabama — Birmingham, Alabama, United States
• University of California — San Diego, California, United States
• Rush University Medical Center — Chicago, Illinois, United States
• Gillette Children's Specialty Healthcare — Saint Paul, Minnesota, United States
• Baylor College of Medicine — Houston, Texas, United States
• South Metropolitan Health Service Fiona Stanley Hospital — Murdoch, Western Australia, Australia
• Amrita Institute of Medical Sciences — Kochi, Kerala, India
• Vijaya Health Centre — Chennai, Tamil Nadu, India
• P.D. Hinduja National Hospital and Medical Research Centre — Mumbai, India
• Jaslok Hospital and Research centre — Mumbai, India
• All India Institute of Medical Sciences — New Delhi, India
• A.O.U. Senese Policlinico Santa Maria alle Scotte — Siena, Tuscany, Italy
• U.O. Neuropsichiatria Infantile — Milan, Italy
• King's College Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

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