NCT06873035Enrolling By Invitation2 期
A Phase 2/3, Multicenter, Open-Label Phase Followed by a Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children With Hypochondroplasia: ACCEL 2/3
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
- 24
- 试验地点
- 45
- 主要终点
- Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
研究概览
简要总结
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses, who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).
详细描述
ACCEL 2/3 is a Phase 2/3 study that comprises of 2 portions. The Phase 2 portion is an open-label, portion in children with HCH aged 5 to 11 years old followed by a Phase 3 portion which is double-blind, placebo-controlled in children with HCH aged >3 years old to <18 years old.
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
- 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
- •Participants must have completed at least 26 weeks and still be on the observational study (QBGJ398-004).
- •Phase 2 portion: Participants 5-11 years of age (inclusive).
- •Phase 3 portion: Participants 3 to <18 years of age at screening with growth potential
- •Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
- •Participants are able to swallow oral medication.
- •Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
- •Participants are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements
- •Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- •If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception, as relevant, while taking study drug and for 1 month after the last dose of study drug.
- •Signed informed consent.
排除标准
- •Participants who have ACH or a short stature condition other than HCH.
- •Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
- •Current evidence of clinically significant corneal or retinal disorder/keratopathy confirmed by ophthalmic examination.
- •Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
- •History and/or current evidence of extensive ectopic tissue calcification.
- •History of malignancy.
- •Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.
- •Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid.
- •Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study.
- •Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
- •Clinically significant abnormality in any laboratory test result at screening.
- •Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
- •Allergy to any components of the study drug.
- •Concurrent circumstance, disease, or condition that would interfere with study participation.
研究组 & 干预措施
Phase 2 Cohort 2
Experimental
infigratinib (0.25 mg/kg/day)
干预措施: infigratinib 0.25 mg/kg/day (Drug)
Phase 2 Cohort 1
Experimental
Oral infigratinib 0.128 mg/kg/day
干预措施: infigratinib 0.128 mg/kg/day (Drug)
结局指标
主要结局
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
时间窗: 26 weeks
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation
时间窗: at least 26 weeks
次要结局
- Changes in cognitive function (as assessed by age appropriate computerized tests)(52 weeks)
- Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)(52 weeks)
- Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)(52 weeks)
- Change from BL in collagen X marker (CXM) levels(52 weeks)
- Change from BL in height Z-score (in relation to both HCH and average height tables)(52 weeks)
- Change from BL in upper to lower body segment ratio (cm)(52 weeks)
- Changes in cognitive function (as assessed by age appropriate computerized tests)(52 weeks)
- Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)(52 weeks)
- Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)(52 weeks)
- Change from BL in collagen X marker (CXM) levels(52 weeks)
研究者
研究点 (45)
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