Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
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|ClinicalTrials.gov Identifier: NCT02790034|
Recruitment Status : Active, not recruiting
First Posted : June 3, 2016
Last Update Posted : February 4, 2019
|Condition or disease||Intervention/treatment||Phase|
|Rett Syndrome||Drug: Sarizotan Drug: Placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||129 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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 :||August 2016|
|Estimated Primary Completion Date :||April 2019|
|Estimated Study Completion Date :||April 2019|
Active Comparator: Sarizotan
Between 2 to 10 mg bid based on age and weight criteria.
2 to 10 mg per day of Sarizotan followed by assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.
Other Name: sarizotan hydrochloride
Placebo Comparator: Placebo
Placebo bid respectively
placebo BID followed by assessment of safety, tolerability and efficacy on reducing the respiratory symptoms in patients.
Other Name: Placebo capsules with microcrystalline cellulose
- Reduction in respiratory abnormality in patients with Rett syndrome [ Time Frame: 3 days prior to Baseline up to week 24 ]Measured as the percent reduction 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.
- Efficacy of sarizotan assessed by the caregiver [ 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.
- Safety and tolerability of sarizotan in patients with Rett syndrome with respiratory symptoms. [ Time Frame: 24 weeks ]Adverse events (AEs),Vital signs (systolic/diastolic blood pressure, pulse, body weight, body temperature, respiratory rate),Laboratory evaluations (blood chemistry, hematology, urinalysis, plasma ACTH, cortisol and prolactin),Electrocardiogram (ECG) - 12-lead standard,Physical examination Neurological examination, Ophthalmology examination (including OCT if feasible), Tanner staging
- Respiratory symptoms - Percent time spent with breathing dysrhythmia per hour [ Time Frame: 24 weeks ]Percent time spent with breathing dysrhythmia per hour. These respiratory outcomes will be determined by assessment of changes in intra-subject values and group mean change values.
- Respiratory symptoms - Number of hyperventilation episodes [ Time Frame: 24 weeks ]Number of hyperventilation episodes. These respiratory outcomes will be determined by assessment of changes in intra-subject values and group mean change values.
- Respiratory symptoms - Oxygen saturation [ Time Frame: 24 weeks ]Oxygen saturation. These respiratory outcomes will be determined by assessment of changes in intra-subject values and group mean change values.
- Respiratory symptoms - Respiratory Distress Index; [ Time Frame: 24 weeks ]Respiratory Distress Index; These respiratory outcomes will be determined by assessment of changes in intra-subject values and group mean change values.
- Respiratory symptoms - Incidence of breathing dysrhythmia episodes [ Time Frame: 24 weeks ]Incidence of breathing dysrhythmia episodes. These respiratory outcomes will be determined by assessment of changes in intra-subject values and group mean change values.
- Motor behaviour [ Time Frame: 24 weeks ]
Assessed by Motor-Behavioral Assessment Scale:
I. Behavioral/Social Assessment - 16 items II. Orofacial/Respiratory Assessment - 7 items III. Motor Assessment/Physical Signs - 14 items.
- Global change from baseline [ Time Frame: 24 weeks ]assessed by the Clinical Global Impression of Change (CGI-C): 7-point scale requiring the clinician to rate how much the patient's illness has improved or worsened relative to the baseline state.
- Caregiver burden [ Time Frame: 24 weeks ]Caregiver Top 3 Concerns: Visual Analogue Scale-based evaluation of three priority concerns identified by caregivers related to the patient's RTT syndrome which they would like to see change as a result of treatment. The severity of these concerns is rated by the caregiver at baseline and is evaluated again at subsequent follow-up visits.
- Overall assessment of symptoms of Rett syndrome [ Time Frame: 24 weeks ]Assessed by Rett Syndrome Clinical Severity Scale (RCSS): Frequency and manageability of seizures, respiratory irregularities, scoliosis, ability to walk (gait apraxia), hand use, speech and sleep; yielding total and feature-specific scores.
- Pharmacokinetics profile of sarizotan and its comparison with the profile in adults [ Time Frame: Baseline, 1 and 4 hr post-dose on Day 1, and 1 and 4 hr post-dose on Day 15 ]measurement of plasma levels of Sarizotan and its major metabolites from blood samples collected from the patients at the specified time points.
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
|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|
|Amrita Institute of Medical Sciences|
|Kochi, Kerala, India, 682041|
|Vijaya Health Centre|
|Chennai, Tamilnadu, India, 600 026|
|Apollo Gleneagles Hospitals|
|Kolkata, West Bengal, India, 700054|
|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|
|A.O.U. Senese Policlinico Santa Maria alle Scotte|
|Siena, Tuscany, Italy, 53100|
|U.O. Neuropsichiatria Infantile|
|Milano, Italy, 20142|
|King's College Hospital|
|London, United Kingdom, SE5 8AF|
|Study Director:||Ravi Anand, MD||Newron Pharmaceuticals|