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
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 129
- 试验地点
- 14
- 主要终点
- Reduction in Respiratory Abnormality in Patients With Rett Syndrome
研究概览
简要总结
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).
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
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)
干预措施: Sarizotan low dose (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)
干预措施: Sarizotan high dose (Drug)
Placebo
Placebo bid for 24 wks DB age 4 and above
干预措施: Placebo (Drug)
结局指标
主要结局
Reduction in Respiratory Abnormality in Patients With Rett Syndrome
时间窗: 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.
次要结局
- Efficacy of Sarizotan Assessed by the Caregiver-rated Impression of Change(24 weeks)