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

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Disease Information

Descriptive Information
Brief Title † Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
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
Brief Summary This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome.
Detailed 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.
Study Phase Phase 2/Phase 3
Study Type † Interventional
Study Design †
Primary Outcome Measure † Reduction in respiratory abnormality in patients with Rett syndrome
Secondary Outcome Measure † Efficacy of sarizotan assessed by the caregiver
Condition † Rett Syndrome
Intervention † DrugSarizotan
Study Arms / Comparison Groups Sarizotan Between 2 to 10 mg bid based on age and weight criteria. Placebo Placebo bid respectively
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment † 129
Start Date † August 2016
Completion Date October 2017
Primary Completion Date July 2017
Eligibility Criteria † Inclusion Criteria: - Body weight ≥ 10 kg - Age ≥ 6 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. Exclusion Criteria: - 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.
Gender All
Ages 6 Years - N/A
Accepts Healthy Volunteers No
Contacts †† Ravi Anand, MD, +39 026103461,
Location Countries † India
Administrative Information
NCT ID † NCT02790034
Organization ID Sarizotan/001/II/2015
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Newron
Collaborators ††
Investigators † Study Director: Ravi Anand, MD, Newron Pharmacueticals
Information Provided By
Verification Date April 2017
First Received Date † May 24, 2016
Last Updated Date April 6, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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