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Trial record 1 of 1 for:    NCT02790034
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Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms (STARS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02790034
Recruitment Status : Terminated (The study did not demonstrate evidence of efficacy on the primary or secondary efficacy variables)
First Posted : June 3, 2016
Results First Posted : December 21, 2021
Last Update Posted : December 21, 2021
Sponsor:
Information provided by (Responsible Party):
Newron Pharmaceuticals SPA

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).

Condition or disease Intervention/treatment Phase
Rett Syndrome Drug: Sarizotan low dose Drug: Sarizotan high dose Drug: Placebo Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 129 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : October 26, 2016
Actual Primary Completion Date : August 6, 2019
Actual Study Completion Date : May 4, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rett Syndrome

Arm Intervention/treatment
Experimental: Sarizotan low dose
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)
Drug: Sarizotan low dose
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). Assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.

Experimental: Sarizotan high dose
5 mg or 10 mg bid based on age and weight criteria for 24 wks DB 5 mg bid (4 to <13 years; ≥13 years of age and weighing <25 kg 10 mg bid (≥13 years of age and weighing ≥25 kg)
Drug: Sarizotan high dose
5 mg or 10 mg bid based on age and weight criteria for 24 wks DB 5 mg bid (4 to <13 years; ≥13 years of age and weighing <25 kg; 10 mg bid (≥13 years of age and weighing ≥25 kg) Assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients

Placebo Comparator: Placebo
Placebo bid for 24 wks DB age 4 and above
Drug: Placebo
Placebo BID followed by assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.




Primary Outcome Measures :
  1. Reduction in Respiratory Abnormality in Patients With Rett Syndrome [ Time Frame: Baseline up to week 24 ]
    Measured as the percent change in the number of apnea episodes per hour during awake time, calculated using an ambulatory data acquisition system (BioRadioTM) as part of home monitoring procedure. BioRadioTM record specific respiratory and cardiac parameters.


Secondary Outcome Measures :
  1. Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change [ Time Frame: 24 weeks ]

    Caregiver-rated Impression of Change (CIC): 7-point scale requiring the caregiver to rate how much the patient's illness has improved or worsened relative to the baseline state.

    7-point Likert-type scale for which ratings range from 1 = very much improved to 7 = very much worse, with 4 = no change. This caregiver-rated measure considered activities, behavior, mood and functioning. This rating was performed in consultation with the study Investigator but was based largely on the caregivers' evaluation during the reporting period. The single rating of the CIC was to be based on changes in the following domains: • Activities (watching TV, interest in conversations around her, cooperation during toileting, dressing/bathing, etc.), • Communication (verbal or by eye movements, hand movements, or head movements), • Behavior (agitation, refusal to feed, scratching, social avoidance), • Participation in family/outdoor/social events)




Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02790034


Locations
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United States, Alabama
University of Alabama
Birmingham, Alabama, United States, 35233
United States, California
University of California
San Diego, California, United States, 92093
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Minnesota
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States, 55101
United States, Texas
Baylor College of Medicine
Houston, Texas, United States, 77030
Australia, Western Australia
South Metropolitan Health Service Fiona Stanley Hospital
Murdoch, Western Australia, Australia, 6961
India
Amrita Institute of Medical Sciences
Kochi, Kerala, India, 682041
Vijaya Health Centre
Chennai, Tamilnadu, India, 600 026
P.D. Hinduja National Hospital and Medical Research Centre
Mumbai, India, 400 016
Jaslok Hospital and Research centre
Mumbai, India, 400 026
All India Institute of Medical Sciences
New Delhi, India, 110 029
Italy
A.O.U. Senese Policlinico Santa Maria alle Scotte
Siena, Tuscany, Italy, 53100
U.O. Neuropsichiatria Infantile
Milano, Italy, 20142
United Kingdom
King's College Hospital
London, United Kingdom, SE5 8AF
Sponsors and Collaborators
Newron Pharmaceuticals SPA
Investigators
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Study Director: Ravi Anand, MD Newron Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Newron Pharmaceuticals SPA:
Study Protocol  [PDF] March 17, 2020
Statistical Analysis Plan  [PDF] March 30, 2020

Publications:
Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0, May 28, 2009 (v4.03 June 14, 2010), U.S. Department of Health and Human Services.
Guy W (Ed). Clinical Global Impressions. In ECDEU Assessment Manual for Psychopharmacology, revised, U.S. Department of Health, Education and Welfare Pub. No. (ADM) 76-338. Rockville, MD: NIMH, 1976, 217-222.
Landon C. Respiratory monitoring: Advantages of inductive plethysmography over impedance pneumograpy. VivoMetrics, VMLA-039-02, 2003.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Newron Pharmaceuticals SPA
ClinicalTrials.gov Identifier: NCT02790034    
Other Study ID Numbers: Sarizotan/001/II/2015
First Posted: June 3, 2016    Key Record Dates
Results First Posted: December 21, 2021
Last Update Posted: December 21, 2021
Last Verified: November 2021
Additional relevant MeSH terms:
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Rett Syndrome
Syndrome
Signs and Symptoms, Respiratory
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System