NCT05625568尚未招募2 期
Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients
Vyant Bio0 个研究点目标入组 48 人开始时间: 2023年3月最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 48
- 主要终点
- Adverse Events
研究概览
简要总结
This is an exploratory, Phase 2, multicenter, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, and efficacy of oral treatment with VYNT-0126 in female subjects 18-45 years of age with Rett syndrome.
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
- 18 Years 至 45 Years（Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Females age 18-45 (inclusive)
- •Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene
- •Severity rating of between 10 and 36 (Rett Syndrome Natural History/Clinical Severity Scale)
- •Concomitant medications (including approved medications for treatment of Rett syndrome) must be stable for >4 weeks prior to enrollment
- •Able to receive liquid study drug orally or via gastrostomy tube (G-tube)
排除标准
- •Actively undergoing neurological regression;
- •Abnormal QT interval, prolongation or significant cardiovascular history
- •Excluded concomitant medications
- •Current clinically significant (as determined by the investigator). cardiovascular, endocrine, hepatic, renal, or respiratory disease
- •Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication
- •History of, or current cerebrovascular disease or brain trauma
- •History of, or current, malignancy
- •Clinically significant abnormalities in safety laboratory tests, vital signs, or ECG, as measured at screening or baseline
- •Any condition which in the investigator's opinion would affect the ability of the subject to participate in the study
- •Allergy to VYNT-0126 or any ingredients of the liquid formulation
研究组 & 干预措施
10 mg VYNT-0126
Experimental
干预措施: VYNT-0126 (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
5 mg VYNT-0126
Experimental
干预措施: VYNT-0126 (Drug)
结局指标
主要结局
Adverse Events
时间窗: Through study completion, approximately 14 weeks
Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the two VYNT-0126 doses and placebo. SAEs and AEs will be examined throughout the study.
次要结局
- Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)(Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).)
- Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)(Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).)
- Change from Baseline in Clinical Global Impression of Severity (CGI-S)(Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).)
- Clinical Global Impression of Improvement (CGI-I)(Obtained at the end of dose titration and end of treatment (approximately 14 weeks).)
研究者
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