NCT06948214招募中3 期
A Multicenter, 12-Month, Randomized, Double Blind, Placebo-Controlled Phase 3 Efficacy and Safety Study of Daily Oral LUM-201 in Naïve-to-Treatment, Prepubertal Children With Growth Hormone Deficiency (GHD)
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Lumos Pharma
- 入组人数
- 150
- 试验地点
- 52
- 主要终点
- AHV after 12 months on LUM-201 compared to placebo
研究概览
简要总结
The OraGrowtH Phase 3 Trial is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate the LUM-201 predictive enrichment marker (LUM-201 PEM) strategy to select subjects likely to respond to therapy with daily oral LUM-201.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 3 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must be naïve to treatment and prepubertal
- •Subjects must have a maximal GH response of < 10 ng/mL from 2 prior GH stimulation tests conducted within the preceding 12 months
- •Impaired height defined as ≥ 2.0 standard deviations (SDs) below the mean height for chronological age and sex
- •Morning or random cortisol level of ≥ 7.0 μg/dL
- •≥ 3.0 years and age ≤ 10.0 years for girls and ≤ 11.0 years for boys
- •Baseline height velocity (HV) based on ≥ 6 months of growth assessments < 25th percentile for age and sex
- •Bone Age delay of ≥ 12 months compared to the chronological age
- •In girls, have genetic testing results to rule out Turner syndrome. If SHOX genetic testing results are available, they need to be negative.
- •Have normal thyroid function. Subjects diagnosed with hypothyroidism must have documented successful treatment for at least 3 months prior to Day 1
- •Baseline IGF-1 standard deviation score (SDS) ≤ -1.0
排除标准
- •Any medical or genetic condition which, in the opinion of the Investigator or Medical Monitor (MM), can be an independent cause of short stature and/or limit the response to exogenous growth factor treatment.
- •Arm span to height ratio > 2 SDs below the mean for age and sex
- •A medical or genetic condition that, in the opinion of the Investigator and/or MM, adds unwarranted risk to use of LUM-201
- •Use of any medication that, in the opinion of the Investigator and/or MM, can independently cause short stature or limit the response to exogenous growth factors
- •Current inflammatory diseases requiring systemic corticosteroid treatment for > 2 consecutive weeks within the last 3 months prior to the Screening Visit
- •Use of hormone replacement therapy for any hormone deficiency other than thyroid deficiency
- •Any ECG at the Screening Visit noted to have a clinically significant abnormality, as confirmed by the MM
- •Any subjects suspected of having past or present intracranial tumor growth as confirmed by brain imaging prior to the Screening or Day 1 Visit
- •Any subject suspected of having intracranial hypertension (IH) as confirmed by fundoscopy and other assessments
- •Any subject with serum alanine transaminase (ALT), aspartate transaminase (AST), or total bilirubin > upper limit of normal (ULN)
- •Suspicion of absent pituitary function as evidenced by a maximal stimulated GH ≤ 3.0 ng/mL on any prior standard of care GH stimulation test completed within 12 months
- •Body weight ≤ 14.0 kg
- •BMI < -2 or > +2 SDs for age and sex based on WHO standards
- •Birth weight for gestational age < 3rd percentile based on WHO standards
- •Treatment with medications known to be moderate or strong inhibitors or strong inducers of cytochrome P450 (CYP) 3A/4
- •History of spinal, cranial, or total body irradiation
- •Attention deficit hyperactivity disorder (ADHD) diagnosis
研究组 & 干预措施
LUM-201
Experimental
干预措施: LUM-201 (Drug)
Placebo
Placebo Comparator
干预措施: Matched Placebo (Capsules) (Other)
结局指标
主要结局
AHV after 12 months on LUM-201 compared to placebo
时间窗: Day 1 to Month 12
Annualized height velocity (AHV) measured as standing height with stadiometer
次要结局
未报告次要终点
研究者
研究点 (52)
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