NCT05145010Enrolling 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 Achondroplasia: PROPEL OLE
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 入组人数
- 300
- 试验地点
- 31
- 主要终点
- Incidence of treatment emergent adverse events (TEAE) and serious TEAE
研究概览
简要总结
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 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
- •Pediatric subjects with ACH who have completed a previous QED-sponsored interventional study with infigratinib.
- •Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
- •Subjects are able to swallow oral medication.
- •Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- •If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
- •The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed.
- •Key Rollover Subjects
排除标准
- •Subject has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
- •Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication.
- •Subjects prematurely discontinued a prior QED-sponsored interventional study with infigratinib
- •Current participation in an ongoing clinical study with a sponsor other than QED
- •Subjects that have reached final height or near final height.
- •Key Inclusion Criteria for Treatment Naïve Subjects
- •Subject must be 3 to <18 years of age at screening and have growth potential.
- •Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
- •Subjects are able to swallow oral medication.
- •Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
- •Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) before study entry.
- •Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- •If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
- •The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed.
- •Key Exclusion Criteria for Treatment Naïve Subjects
- •Subjects who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
- •Subjects who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subject or would interfere with study participation or safety evaluations.
- •Subjects who have a history of malignancy.
- •Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A
- •Subjects who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
- •Subjects who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months.
- •Subjects who have significant abnormality in screening laboratory results.
- •Subjects who have had a fracture within 12 months of screening.
研究组 & 干预措施
Arm 1: Rollover subjects
Experimental
Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)
干预措施: Infigratinib (Drug)
Arm 2: Treatment naïve subjects
Experimental
Children naïve to 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 height Z-score in relation to ACH and non-ACH growth charts
时间窗: 10 years
次要结局
- Changes over time in range of motion (elbow)(10 years)
- Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview(10 years)
- Changes over time in weight z-score(10 years)
- Age of puberty onset and time to Tanner stage ≥4(10 years)
- Changes over time in number of episodes of otitis media per year(10 years)
- Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts(10 years)
- Changes over time in body proportions(10 years)
- Changes overtime in BMI(10 years)
- Changes over time in number of episodes and/or severity of sleep apnea(10 years)
- Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)(10 years)
- Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)(10 years)
- Changes in cognitive functions assessed by age-appropriate computerized tests(10 years)
- Changes over time in skeletal abnormalities of the lower extremities and spine(10 years)
- Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)(10 years)
- Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)(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)
研究者
研究点 (31)
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