A 40-Week, Open-label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 154
- 试验地点
- 21
- 主要终点
- Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG
研究概览
简要总结
To investigate the safety and tolerability of 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 21 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •Has completed the Week 12/End-of-treatment visit of the antecedent study, Study ACP-2566-003
- •Met all entry criteria for the antecedent study
- •May benefit from long-term 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
- •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 3 months prior to Baseline
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) or has major surgery planned during the study
- •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 a QTcF interval of >450 ms on the Baseline ECG performed before the first dose of trofinetide is given in the present study
- •Has developed a clinically significant ECG finding during the antecedent study
- •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
Trofinetide
干预措施: Trofinetide (Drug)
结局指标
主要结局
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in ECG
时间窗: 40 Weeks Treatment Duration
Potentially clinically important ECG changes were defined in the study protocol as absolute QTcF interval \>500 ms or QTcF interval change from the baseline value of previous study ACP-2566-003 of \>60 ms
Percentage of Subjects With Treatment-emergent Adverse Events (TEAEs), Percentage of Subjects With Serious Adverse Events (SAEs), and Percentage of Subjects With Withdrawals Due to AEs
时间窗: 40 Weeks Treatment Duration
Percentage of subjects with treatment-emergent adverse events (TEAEs), percentage of subjects with serious adverse events (SAEs), and percentage of subjects with withdrawals due to AEs
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Body Weight
时间窗: 40 Weeks Treatment Duration
Potentially clinically important changes in body weight were defined in the study protocol as: Weight increase ≥7% from baseline; Weight decrease ≥7% from baseline
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes in Vital Signs
时间窗: 40 Weeks Treatment Duration
Potentially clinically important changes in vital signs were defined in the study protocol 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 ≥120 bpm and increased ≥15 bpm from baseline; Pulse ≤50 bpm and decreased ≥15 bpm from baseline
Subjects (N, %) With Post-baseline Potentially Clinically Important Changes
时间窗: 40 Weeks Treatment Duration
Potentially clinically important changes in laboratory parameters were defined in the study protocol 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 \>2.0 mmol/L; Blood urea nitrogen ≥10.71 mmol/L; Creatinine \>1.5 x upper limit of normal (ULN); Uric acid ≥505.75 μmol/L; Lactate dehydrogenase ≥3 x 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 aminotransferase ≥3 x ULN; Aspartate aminotransferase ≥3 x ULN; Gamma glutamyl transpeptidase ≥3 x ULN; Alkaline phosphatase ≥3 x ULN; Bilirubin ≥1.5 x ULN
次要结局
- Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Change From Baseline to Week 40 in Clinical Global Impression-Severity (CGI-S)(40 Weeks Treatment Duration)
- Rett Syndrome Clinician Rating of Hand Function (RTT-HF) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Clinical Global Impression-Improvement (CGI-I) Score at Week 40(40 Weeks Treatment Duration)
- Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM) Change From Baseline to Week 40(40 Weeks Treatment Duration)
- Impact of Childhood Neurologic Disability Scale (ICND) Total Score Change From Baseline to Week 40(40 Weeks Treatment Duration)