Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms
This study is currently recruiting participants.
Verified June 2017 by Newron
Information provided by (Responsible Party):
First received: May 24, 2016
Last updated: June 23, 2017
Last verified: June 2017
This study evaluates the safety, tolerability and efficacy of Sarizotan in reducing respiratory abnormalities in Rett Syndrome.
|Study Design:||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|
Resource links provided by NLM:
U.S. FDA Resources
Further study details as provided by Newron:
Primary Outcome Measures:
- 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.
Secondary Outcome Measures:
- 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.
|Actual Study Start Date:||August 2016|
|Estimated Study Completion Date:||December 2018|
|Estimated Primary Completion Date:||July 2018 (Final data collection date for primary outcome measure)|
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
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.
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02790034
Please refer to this study by its ClinicalTrials.gov identifier: NCT02790034
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Sponsors and Collaborators
|Study Director:||Ravi Anand, MD||Newron Pharmacueticals|