A Double-Blind, Randomized, Placebo-Controlled, Dose Titration Study of ANAVEX2-73 in Patients With Rett Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 31
- 试验地点
- 8
- 主要终点
- Incidence of Adverse Events
研究概览
简要总结
Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.
7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.
详细描述
This Phase 2 safety, tolerability and efficacy study is designed as a double-blind, randomized, placebo-controlled study.
This is a 7-week placebo-controlled study of ANAVEX2-73 oral solution for the treatment of patients with RTT 18 years or older. A voluntary option will be offered for all patients who meet the exposure criteria for ANAVEX2-73 to continue a 12-week open label extension.
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)
盲法说明
Double-Blind, Randomized, Placebo-Controlled
入排标准
- 年龄范围
- 18 Years 至 45 Years（Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Aged ≥ 18 years, inclusive.
- •Diagnosis of classic RTT, according to 2010 criteria (Neul et al., 2010), and a MECP2 mutation.
- •Current pharmacological treatment regimen, including supplements, has been stable for at least 4 weeks.
- •If on antiepileptic drugs (AEDs), 1-4 AEDs allowed. Treatment must be stable (drug, dose, interval of administration) for 30 days prior to enrollment.
- •Ability to keep accurate seizure diaries or have caregiver who can keep accurate seizure diaries.
- •Confirmation from the participant that, if of childbearing potential is not pregnant through urine pregnancy testing. Female patients of childbearing potential and at risk for pregnancy must agree to abstinence.
- •Prior to the conduct of study-specific procedures, the subject's parent/caregiver/LAR must provide written informed consent. If applicable, the research team must attempt to obtain consent from both parents.
排除标准
- •Patients who have a progressive medical or neurological condition that in the opinion of the Investigator would interfere with the conduct of the study.
- •Current clinically significant systemic illness that is likely to result in deterioration of the patient's condition or affect the patient's safety during the study.
- •History of clinically evident stroke or clinically significant carotid or vertebrobasilar stenosis or plaque or other history of neurologic (e.g., head trauma with loss of consciousness) or psychiatric condition that the Investigator deems may interfere with interpretability of data.
- •Indication of liver disease, defined by serum levels of ALT (SGPT), AST (SGOT), or alkaline phosphatase above 3x upper limit of normal (ULN) as determined during screening.
- •Treatment with immunosuppressive medications (e.g., systemic corticosteroids) within the last 90 days (topical and nasal corticosteroids and inhaled corticosteroids for asthma are permitted) or chemotherapeutic agents for malignancy within the last 3 years.
- •Other clinically significant abnormality on physical, neurological, laboratory, or electrocardiogram (ECG) examination (e.g., atrial fibrillation) that could compromise the study or be detrimental to the participant.
- •Any known hypersensitivity to any of the excipients contained in the study drug or placebo formulation.
- •Other co-morbid or chronic illness beyond that known to be associated with RTT.
- •Subjects who plan to initiate or change pharmacologic or nonpharmacologic intervention during the course of the study.
- •Subjects on potent CYP 3A4 and CYP2C19 inhibitors and inducers.
- •Subjects taking another investigational drug currently or within the last 30 days.
- •Any other criteria (such as a clinically significant screening blood test result), which in the opinion of the Investigator could interfere with the study conduct or outcome.
- •Patients with hepatic and renal impairment.
研究组 & 干预措施
Active arm
Week 0-7: Take 1 ml orally of the product daily (solution of ANAVEX2-73)
干预措施: ANAVEX2-73 (Drug)
Placebo arm
Week 0-7: Take 1 ml orally of the product daily (placebo)
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence of Adverse Events
时间窗: 7 weeks
Incidence of Adverse Events
Maximum Plasma Concentration [Cmax] of ANAVEX2-73
时间窗: 7 weeks
PK of ANAVEX2-73 and metabolite
Lipid panel
时间窗: 7 weeks
Significant laboratory findings
Area Under the Curve [AUC] of ANAVEX2-73
时间窗: 7 weeks
PK of ANAVEX2-73 and metabolite
次要结局
- RSBQ(7 weeks)
- CGI-I(7 weeks)