Summary

for people ages 4 years and up (full criteria)
at San Diego, California and other locations
study started
estimated completion:
Richard Haas

Description

Summary

This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome.

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

Details

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.

Keywords

Rett Syndrome Syndrome Signs and Symptoms, Respiratory Sarizotan

Eligibility

You can join if…

Open to people ages 4 years and up

  • 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 CAN'T join 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.

Locations

  • University of California accepting new patients
    San Diego California 92093 United States
  • Baylor College of Medicine accepting new patients
    Houston Texas 77030 United States

Lead Scientist

  • Richard Haas
    Professor, Neurosciences. Authored (or co-authored) 34 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Newron Pharmaceuticals SPA
ID
NCT02790034
Phase
Phase 2/3
Study Type
Interventional
Last Updated