2024-513857-55-00招募中2 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
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 68
- 试验地点
- 10
- 主要终点
- Incidence of treatment emergent adverse events (TEAE) and serious TEAE
研究概览
简要总结
- To evaluate the safety and tolerability of long-term administration of daily doses of oral infigratinib in subjects with ACH.
- To evaluate the efficacy of long-term administration of daily doses of oral infigratinib in subjects with ACH as assessed as changes over time in standing height Z-score.
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 主要目的
- Safety Follow-Up
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Inclusion Criteria for Rollover Subjects
- •Pediatric subjects with ACH who have completed a previous QEDsponsored interventional study with infigratinib.
- •Subjects and parent(s), legal guardians, or caregivers 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 parents(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed.
- •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.
排除标准
- •Exclusion Criteria for Rollover Subjects
- •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 that 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
- •Exclusion Criteria for Treatment Naïve Subjects
- •Subjects who have hypochondroplasia or short stature condition other than ACH (eg, 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) 3A4
- •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, insulinlike 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.
结局指标
主要结局
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Changes over time in height Z-score in relation to ACH and non-ACH growth charts.
Changes over time in height Z-score in relation to ACH and non-ACH growth charts.
次要结局
- Change over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non-ACH growth charts.
- Changes over time in body proportions
- Changes over time in weight Z-score
- Changes over time in BMI
- Age of puberty onset and time to Tanner stage ≥4.
- Changes over time in number of episodes of otitis media per year.
- Changes over time in number of episodes and/or severity of sleep apnea.
- Changes over time in range of motion (elbow).
- Changes over time in skeletal abnormalities of the lower extremities and spine
- Changes in health-related quality of life (HRQoL) as assessed by Pediatric Quality of Life Inventory (PedsQL)
- Changes in health-related quality of life (HRQoL) as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
- Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
- Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
- Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview
- Changes in cognitive functions assessed by age-appropriate computerized tests
研究者
QED Therapeutics SVP Regulatory
Scientific
Qed Therapeutics Inc.
研究点 (10)
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