2024-516822-67-00招募中3 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
Qed Therapeutics Inc.7 个研究点 分布在 5 个国家目标入组 32 人开始时间: 2025年7月15日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 32
- 试验地点
- 7
- 主要终点
- ACCEL 2: Change from baseline (BL) in height velocity (HV) at Week 26 (annualized to cm/year). ACCEL 3: Change from BL to Week 52 in AHV compared to placebo.
研究概览
简要总结
ACCEL 2: To obtain preliminary evidence of the efficacy of infigratinib in pediatric participants 5 to 11 years of age with HCH who have short stature. ACCEL 2: To evaluate the safety and tolerability of infigratinib in children with HCH. ACCEL 3: To evaluate the efficacy of infigratinib in pediatric participants 3 to <18 years of age with HCH who have short stature with potential to grow.
研究设计
- 分配方式
- Randomized
- 主要目的
- Accel 3
- 盲法
- Double (Carer, Subject, Analyst, Monitor, Investigator)
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
- •Participants must have completed the Week 26 visit in the observational study (QBGJ398-004).
- •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 while taking study drug and for 30 days after the last dose of study drug.
- •Signed informed consent.
- •ACCEL 2: Participants 5-11 years of age (inclusive).
- •Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
- •AHV >X cm/year over a period ≥26 weeks prior to screening based on measurements obtained during observational Study QBJG398-004 (ACCEL) (Phase 2 portion only). NOTE: Visit window may be applied to the 26-week period of the observational ACCEL study as per the ACCEL SoA (ie. the Week 26 visit in ACCEL may occur slightly earlier than 26 weeks).
- •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.
排除标准
- •Participants who have ACH or a short stature condition other than HCH. Participants with variants in FGFR3 known to cause ACH or other FGFR3-related conditions will be excluded.
- •Children with epilepsy who meet certain additional criteria.
- •Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib. Complete list referenced in protocol.
- •Current evidence of clinically significant corneal or retinal disorder/keratopathy.
- •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 or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit.
- •Current participation in any other ongoing clinical study with other sponsor.
- •Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study; or previous guided growth surgery with plates still in place or removed within the 12 months prior to screening.
- •Currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP)3A or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract.
- •Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
- •Clinically significant abnormality in any laboratory test result at screening as specified in the protocol.
- •Having had a fracture of the long bones or spine within 12 months prior to screening.
- •Females who have had their menarche (ACCEL 2 only).
- •Pregnant or breastfeeding at the screening visit.
- •Allergy to any components of the study drug.
结局指标
主要结局
ACCEL 2: Change from baseline (BL) in height velocity (HV) at Week 26 (annualized to cm/year). ACCEL 3: Change from BL to Week 52 in AHV compared to placebo.
ACCEL 2: Change from baseline (BL) in height velocity (HV) at Week 26 (annualized to cm/year). ACCEL 3: Change from BL to Week 52 in AHV compared to placebo.
次要结局
- ACCEL 2: Change from BL in height Z-score (in relation to both HCH and average height tables for age and sex) compared to placebo.
- ACCEL 3: Change from BL to Week 52 in upper to lower body segment ratio, compared to placebo.
研究者
QED Clinical Development
Scientific
Qed Therapeutics Inc.
研究点 (7)
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