NCT07393373Enrolling By Invitation2 期
Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Hypochondroplasia: ACCEL OLE
QED Therapeutics, a BridgeBio company24 个研究点 分布在 10 个国家目标入组 135 人开始时间: 2026年4月23日最近更新:
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 135
- 试验地点
- 24
- 主要终点
- Incidence of treatment emergent adverse events (TEAE) and serious TEAE
研究概览
简要总结
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.
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
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Inclusion Criteria for Participants Rolling Over from ACCEL 2/3
- •Pediatric participants with HCH who have completed ACCEL 2/3
- •Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche
排除标准
- •Exclusion Criteria for Participants Rolling Over from ACCEL 2/3
- •Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
- •Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
- •Participants who prematurely discontinued ACCEL 2/3
- •Participants who have reached final height or near final height
- •Current participation in an ongoing clinical study with a sponsor other than QED
研究组 & 干预措施
Arm 1: Rollover subjects
Experimental
Children who have completed QED-sponsored interventional study with infigratinib
干预措施: Infigratinib (Drug)
结局指标
主要结局
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
时间窗: 10 years
Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts
时间窗: 10 years
次要结局
- Changes over time in AHV Z-score(10 years)
- Changes over time in body proportions(10 years)
- Changes over time in weight Z-score(10 years)
- Changes overtime in BMI(10 years)
- Age of puberty onset and time to Tanner stage ≥4(10 years)
- Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans(10 years)
- Changes over time in bone morphology/density by x-ray and DXA(10 years)
- Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test)(10 years)
- Change in attention assessed by age-appropriate computerized tests (Identification Test)(10 years)
- Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)(10 years)
- Change in working memory assessed by age-appropriate computerized tests (One Back Test)(10 years)
- Changes over time in severity of epilepsy measured by frequency and adverse event grading(10 years)
- Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)(10 years)
- Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)(10 years)
- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)(10 years)
- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)(10 years)
- Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview(10 years)
研究者
研究点 (24)
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