An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 7
- 主要终点
- Safety and Tolerability of Treatment With Oral Trofinetide
研究概览
简要总结
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 5 Years（Child）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Female subject
- •2 to 4 years of age and body weight ≥9 kg and <20 kg at Screening OR
- •5 years of age and body weight ≥9 kg and <12 kg at Screening
- •Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
- •The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
- •Has classic/typical Rett syndrome (RTT) or possible RTT according to the Rett Syndrome Diagnostic Criteria
- •Has a documented disease-causing mutation in the MECP2 gene
- •Has a stable pattern of seizures, or has had no seizures, within 8 weeks prior to Screening
- •Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 4 weeks prior to Screening
排除标准
- •Has been treated with insulin within 12 weeks of Baseline
- •Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
- •Has a history of, or current, cerebrovascular disease or brain trauma
- •Has significant, uncorrected visual or uncorrected hearing impairment
- •Has a history of, or current, malignancy
- •Has any of the following:
- •QTcF interval of >450 ms at Screening or Baseline
- •History of a risk factor for torsades de pointes (e.g., heart failure or family history of long QT syndrome)
- •History of clinically significant QT prolongation that is deemed to put the subject at increased risk of clinically significant QT prolongation
- •Other clinically significant finding on ECG at Screening or Baseline
- •Additional inclusion/exclusion criteria apply. Patients will be evaluated at baseline to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all prespecified entry criteria).
研究组 & 干预措施
Drug - trofinetide
Oral dose of trofinetide
干预措施: Trofinetide (Drug)
结局指标
主要结局
Safety and Tolerability of Treatment With Oral Trofinetide
时间窗: Mean study drug exposure 434 days, corresponding to 1.2 years
Percentage of patients with treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), withdrawals due to AEs, potentially clinically important (PCI) changes in other safety assessments (laboratory values, vital signs, or ECGs, following protocol-defined PCI criteria)
AUC0-12,ss (Area Under the Concentration-time Curve From Time 0 to 12 h at Steady State)
时间窗: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Area under the concentration-time curve from time 0 to 12 h at steady state as obtained from population pharmacokinetic (PK) modelling
Cmax,ss (Maximum Observed Drug Concentration at Steady State)
时间窗: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling
Cmin,ss (Minimum Observed Drug Concentration at Steady State of Oral Trofinetide)
时间窗: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Minimum observed drug concentration at steady state of oral trofinetide as obtained from population pharmacokinetic (PK) modeling
Tmax (Time of the Maximum Observed Drug Concentration at Steady State)
时间窗: PK samples were taken predose and at Weeks 2, 4, 8, and 12
Time of the maximum observed drug concentration at steady state as obtained from population pharmacokinetic (PK) modelling
次要结局
未报告次要终点