An Open-Label Extension Study of Continuing Treatment With Trofinetide for Rett Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 78
- 试验地点
- 21
- 主要终点
- Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs
研究概览
简要总结
To investigate the safety and tolerability of continued long-term treatment with oral trofinetide in girls and women 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
- 5 Years 至 22 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Has completed the EOT visit of the antecedent trofinetide Study ACP-2566-004 (i.e., has completed 40 weeks)
- •May benefit from continued treatment with open-label trofinetide in the judgment of the Investigator
- •Can still 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
- •Childbearing Potential
- •Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter. If a subject is sexually active or becomes sexually active during the study, she must use 2 clinically acceptable methods of contraception (e.g., oral, intrauterine device [IUD], diaphragm plus spermicide, injectable, transdermal or implantable contraception) for the duration of the study and for at least 30 days thereafter. Subject must not be pregnant or breastfeeding.
排除标准
- •Began treatment with growth hormone during the antecedent study
- •Began treatment with IGF-1 during the antecedent study
- •Began treatment with insulin during the antecedent study
- •Has developed a 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)
- •Subject is judged by the Investigator or the Medical Monitor to be inappropriate for the study due to AEs, medical condition, or noncompliance with investigational product or study procedures in the antecedent study
- •Has a clinically significant abnormality in vital signs at Baseline
- •Has an average QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study (i.e., the ECG performed at the EOT visit of the antecedent study)
- •Has developed a clinically significant ECG finding during the antecedent study
- •Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening 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 pre-specified entry criteria).
研究组 & 干预措施
Drug - trofinetide
trofinetide oral solution
干预措施: trofinetide (Drug)
结局指标
主要结局
Percentage of Patients With Treatment-emergent Adverse Events (TEAEs), With Serious Adverse Events (SAEs), and With Withdrawals Due to AEs
时间窗: Mean study drug exposure was 426 days, corresponding to 1.2 years
Withdrawals due to AEs included both study drug discontinuation as well as study termination
Number (%) of Patients With Potentially Clinically Important Changes in ECG Post-baseline
时间窗: Mean study drug exposure was 426 days, corresponding to 1.2 years
Potentially clinically significant ECG changes were defined as QTcF \>500 ms or QTcF change from baseline (CFB) of \>60 ms
Number (%) of Patients With Potentially Clinically Important Changes in Vital Signs Post-baseline
时间窗: Mean study drug exposure was 426 days, corresponding to 1.2 years
Potentially clinically important changes from baseline in vital signs were defined as: Systolic blood pressure (SBP) ≥180 mmHg and increased ≥20 mmHg from baseline; SBP ≤90 mmHg and decreased ≥20 mmHg from baseline; Diastolic blood pressure (DBP) ≥105 mmHg and increased ≥15 mmHg from baseline; DBP ≤50 mmHg and decreased ≥15 mmHg from baseline; Pulse rate (PR) ≥120 bpm and increased ≥15 bpm from baseline; PR≤50 bpm and decreased ≥15 bpm from baseline. Baseline was the baseline value of previous study ACP-2566-003.
Number (%) of Patients With Potentially Clinically Important Changes in Body Weight Post-baseline
时间窗: Mean study drug exposure was 426 days, corresponding to 1.2 years
Potentially clinically important changes from baseline in body weight were defined as: Weight increase ≥7% from baseline Weight decrease ≥7% from baseline
Number (%) of Patients With Potentially Clinically Important Changes in Laboratory Parameters Post-baseline
时间窗: Mean study drug exposure was 426 days, corresponding to 1.2 years
Potentially clinically important changes in laboratory parameters were defined as: sodium ≤125 mmol/L; sodium ≥155 mmol/L; potassium ≤3.0 mmol/L ; potassium ≥5.5 mmol/L; chloride ≤85 mmol/L; chloride ≥120 mmol/L; calcium \<2.0 mmol/L; calcium \>2.75 mmol/L; blood urea nitrogen ≥10.71 mmol/L; creatinine \>1.5× upper limit of normal (ULN); uric acid ≥505.75 μmol/L; lactate dehydrogenase (LDH) ≥3×ULN; glucose ≤2.48 mmol/L; glucose ≥11 mmol/L; albumin ≤26 g/L; albumin ≥60 g/L; protein ≤50 g/L; protein ≥100 g/L; alanine transaminase (ALT) ≥3×ULN; aspartate transaminase (AST) ≥3×ULN; gamma glutamyl transferase (GGT) ≥3×ULN; alkaline phosphatase (ALP) ≥3×ULN; bilirubin ≥1.5×ULN
次要结局
未报告次要终点