EUCTR2015-004448-20-GB进行中（未招募）1 期
A Randomized, Double-Blind, Placebo-Controlled, Six-Month Study to Evaluate the Efficacy, Safety and Tolerability of Sarizotan in Patients with Rett Syndrome with Respiratory Symptoms
ewron Pharmaceuticals S.p.A.0 个研究点目标入组 129 人开始时间: 2016年9月23日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 129
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •The patient must meet all of the following inclusion criteria to be eligible for enrolment into the study:
- •Demographics
- •1.Female or male = 4 years of age.
- •2.Body weight = 10 kg, and within the expected range for an RTT patient, based on age and height.
- •3.Diagnosis of Rett syndrome based on consensus clinical criteria; a test for MECP2 mutations (Xq28), will be performed at screening if results from an accredited laboratory are not available; selection for the trial is not contigent on the result of MECP2 test. Patients with known MECP2 duplications will not be eligible.
- •4.One or more of the following breathing dysfunctions: periodic apnea during wakefulness; intermittent hyperventilation; breath holding spells; air swallowing; forced expulsion of air or saliva.
- •5.Patient meets all of the following criteria related to breathing abnormalities:
- •a.Parent report of 10 episodes or more of breathing abnormality per day during wakefulness in the week prior to the screening visit;
- •b.Time per hour spent on normal breathing is less than 90% of the total time per hour of wakefulness (i.e., =10% of the time should be abnormal breathing);
- •c.Has at least 10 episodes of breathing dysrhythmia, defined by episodes =10 seconds of breath holding (apnea), per hour during cardiorespiratory monitoring (performed with home/ambulatory monitoring system during screening period).
- •6.Stable medication regimen for 4 weeks prior to beginning the study (if receiving services - physical, occupational, or speech therapy - subjects must be on a stable regimen of these services for 3 months prior to beginning the study). Female patients of childbearing potential are to use adequate contraception as recommended by their Health Care Provider.
- •7.Parent/legal guardian/representative has provided written consent prior to the patient participating in the study. Where feasible, consent or assent for patients less than 18 years of age, has also been provided by the patient.
- •8.Ability to take study medication provided either as capsules or combined with food/drink.
- •9.Patient is cooperative, willing to complete all aspects of the study, and capable of doing so with assistance of a caregiver.
- •10.Caregiver is able to understand the instructions and fully participate.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 90
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 39
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •The presence of any of the following will exclude a patient from study enrollment:
- •1.Meets any of the diagnostic exclusion criteria for Rett syndrome, Typical;
- •2.Patient is participating in a clinical trial with another investigational drug or has taken an investigational drug within one month or 5 half-lives (whichever is longer) prior to screening;
- •3.Hypersensitivity to sarizotan or other 5-HT1a agonists, [UK only: or to the capsule material gelatin or microcrystalline cellulose used in the placebo capsules];
- •4.Current clinically significant (as determined by Investigator) a) 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 [for US, Italy, India
- •and Australia];
- •b) cardiovascular, respiratory (e.g. severe asthma), or gastrointestinal
- •disease, renal impairment (as indicated by creatinine >2X ULN), hepatic
- •impairment (as indicated by total bilirubin >2X ULN), history of
- •moderate or severe hepatic insufficiency or moderate or severe liver cirrhosis, or hematologic or other medical disorders, in addition to those directly related to the patient's Rett syndrome [for UK only];
- •5.QTcF interval on the ECG is greater than 450 msec.
- •6.Surgery planned during the study (except for insertion of gastrostomy tube);
- •7.Severe diabetes mellitus or fatty acid oxidation disorder.
- •8.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.
- •9.Females who are pregnant, breastfeeding, or of childbearing potential and not using adequate contraception, as recommended by their Health Care Provider.
- •10.[UK only: Subjects who have an inborn error of metabolism.]
- •11.Evidence of clinically significant malnutrition.
研究者
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