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

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 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).
    

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.

      All patients who have completed the final evaluations at Week 24 (Day 168) and have no safety
      or tolerability issues that would preclude continuing on the study medication and have been
      compliant with the trial requirements will have the option of continuing open-label treatment
      with Sarizotan for up to 168 weeks.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional

Primary Outcome

Reduction in Respiratory Abnormality in Patients With Rett Syndrome

Secondary Outcome

 Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change

Condition

Rett Syndrome

Intervention

Sarizotan low dose

Study Arms / Comparison Groups

 Sarizotan low dose

Description:  2 mg or 5 mg bid based on age and weight criteria for 24 wks DB 2 mg bid (4 to <13 years; ≥13 years of age and weighing <25 kg 5 mg bid (≥13 years of age and weighing ≥25 kg)

Publications

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

Recruitment Status

Drug

Estimated Enrollment

129

Start Date

October 26, 2016

Completion Date

May 4, 2020

Primary Completion Date

August 6, 2019

Eligibility Criteria

        Inclusion Criteria:

          -  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.

        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

4 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Ravi Anand, MD, , 

Location Countries

Australia

Location Countries

Australia

NCT ID

NCT02790034

Organization ID

Sarizotan/001/II/2015

Responsible Party

Sponsor

Study Sponsor

Newron Pharmaceuticals SPA

Study Sponsor

Ravi Anand, MD, Study Director, Newron Pharmaceuticals

Verification Date

November 2021