2025-523509-13-00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 Hypochondroplasia: ACCEL OLE
Qed Therapeutics Inc.3 个研究点 分布在 2 个国家目标入组 22 人开始时间: 2026年4月1日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 22
- 试验地点
- 3
- 主要终点
- Incidence, type, severity, and causality of AEs; SAEs; AEs that require dose reduction or discontinuation per study protocol; and changes over time in vital signs, laboratory assessments and special investigations (ophthalmic and dental evaluations), and imaging (x-rays, , dual x-ray absorptiometry [DXA] scan).
研究概览
简要总结
To evaluate the safety and tolerability of long-term, once-daily oral administration of infigratinib for the treatment of participants living with HCH. To evaluate the efficacy of long-term, once-daily oral administration infigratinib in participants with HCH assessed by changes over time in standing height Z-score.
研究设计
- 分配方式
- Na
- 主要目的
- Open Label Extension
- 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
- 否
入选标准
- •for Participants Rolling Over from ACCEL 2/3:
- •Pediatric participants with HCH who have completed ACCEL 2/
- •Inclusion Criteria for Participants Rolling Over from ACCEL 2/3:
- •Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- •Inclusion Criteria for Participants Rolling Over from ACCEL:
- •Participant must be 3 to <18 years of age at screening and have growth potential (defined as X in females and X in males).
- •Inclusion Criteria for Participants Rolling Over from ACCEL:
- •Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
- •Inclusion Criteria for Participants Rolling Over from ACCEL:
- •Participants have at least a 6-month period of growth assessment in the ACCEL study before study entry.
- •Inclusion Criteria for Participants Rolling Over from ACCEL:
- •Participants are ambulatory and able to stand without assistance.
- •Inclusion Criteria for Participants Rolling Over from ACCEL:
- •Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
排除标准
- •for Participants Rolling Over from ACCEL 2/3:
- •Participant has concurrent medical condition (ie, circumstance, syndrome, symptom, sign, etc) that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Currently receiving treatment with agents that are known strong X or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Participants receiving medications which could increase X
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Having had a fracture of the long bones or spine within 12 months of screening.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Current participation in an ongoing clinical study with a sponsor other than QED.
- •Exclusion Criteria for Participants Rolling Over from ACCEL 2/3:
- •Current evidence of clinically significant X
- •Exclusion Criteria for Participants Rolling Over from ACCEL 2/3:
- •Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication.
- •Exclusion Criteria for Participants Rolling Over from ACCEL 2/3:
- •Participants who prematurely discontinued ACCEL 2/
- •Exclusion Criteria for Participants Rolling Over from ACCEL 2/3:
- •Participants who have reached final height or near final height (AHV X for a minimum 6-month observation period and X for males and X for females).
- •Exclusion Criteria for Participants Rolling Over from ACCEL 2/3:
- •Current participation in an ongoing clinical study with a sponsor other than QED.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Participants who have a clinically significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the participant or would interfere with study participation or safety evaluations. Complete list referenced in protocol.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Participants who have a history and/or current evidence of extensive ectopic tissue calcification.
- •Exclusion Criteria for Participants Rolling Over from ACCEL:
- •Participants who have a history of malignancy.
研究组 & 干预措施
INFIGRATINIB, INFIGRATINIB, Infigratinib, INFIGRATINIB, INFIGRATINIB
Test
干预措施: Infigratinib (Drug)
INFIGRATINIB, INFIGRATINIB, Infigratinib, INFIGRATINIB, INFIGRATINIB
Test
干预措施: INFIGRATINIB (Drug)
结局指标
主要结局
Incidence, type, severity, and causality of AEs; SAEs; AEs that require dose reduction or discontinuation per study protocol; and changes over time in vital signs, laboratory assessments and special investigations (ophthalmic and dental evaluations), and imaging (x-rays, , dual x-ray absorptiometry [DXA] scan).
Incidence, type, severity, and causality of AEs; SAEs; AEs that require dose reduction or discontinuation per study protocol; and changes over time in vital signs, laboratory assessments and special investigations (ophthalmic and dental evaluations), and imaging (x-rays, , dual x-ray absorptiometry [DXA] scan).
Changes over time in standing height Z-score (in relation to both HCH and average height tables for age and sex).
Changes over time in standing height Z-score (in relation to both HCH and average height tables for age and sex).
次要结局
- Changes over time in AHV and AHV Z-score in relation to average height tables for age and in body proportions including upper to lower body segment ratio, upper arm to forearm length ratio, upper leg to lower leg length ratio, arm span to standing height ratio, and head circumference to standing height ratio, in weight Z-score and BMI in relation to average height tables (and HCH tables, if and when they become available), age of puberty onset and time to X, in body composition
- Change over time in various quality of life scales including Pediatric Quality of Life, Quality of Life in Short Stature Youth, Patient/ Parent Global Impression of Severity and Change
- Treatment benefit as reported by parent and participant during qualitative interview.
- Changes over time in body composition as assessed by dual x-ray absorptiometry (DXA) scans.
- Changes over time in bone morphology/density by x-ray and DXA.
研究者
QED Clinical Development
Scientific
Qed Therapeutics Inc.
研究点 (3)
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