A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Phase 3 Study of Bionetide for the Treatment of Girls and Women With Rett Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 210
- 试验地点
- 24
- 主要终点
- Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
研究概览
简要总结
Primary Objective • To investigate the efficacy of treatment with oral Bionetide versus placebo in girls and women with Rett syndrome
Key Secondary Objective
• To investigate the efficacy of treatment with oral Bionetide versus placebo on ability to communicate in girls and women with Rett syndrome
详细描述
Co-Primary Endpoints
- Rett Syndrome Behavior Questionnaire (RSBQ) total score - Change from Baseline to Week 12
- Clinical Global Impression-Improvement (CGI-I) Score at Week 12
Key Secondary Endpoint
Change from Baseline to Week 12 in:
• Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist- Social Composite Score (CSBS-DP-IT Social)
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
Quadruple Participant/Care Provider/ Investigator/ Outcome Assessor
入排标准
- 年龄范围
- 5 Years 至 20 Years（Child, Adult）
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Bionetide
Experimental: Bionetide Bionetide solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
干预措施: Bionetide (Drug)
Placebo
Placebo: Bionetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
时间窗: Baseline and Week 12
The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behavior.
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
时间窗: 12 Weeks Treatment Duration
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.
次要结局
- Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)(12 Weeks Treatment Duration)
- Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)(12 Weeks Treatment Duration)