A Multicenter, Open-Label, Phase 2 Study to Evaluate Growth and Safety of LUM-201 Following 12 Months of Daily rhGH Treatment in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed the LUM-201-01 Trial
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Lumos Pharma
- 入组人数
- 15
- 试验地点
- 19
- 主要终点
- Annualized height velocity (AHV) measured as standing height with stadiometer
研究概览
简要总结
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the LUM-201-01 trial.
详细描述
This trial will have one screening/baseline visit with tests to assess if subjects are eligible to start study therapy. The screening/baseline visit can coincide with the Month 12 visit in the LUM-201-01 trial. Subjects will receive the same dose of LUM-201 as a daily oral dose each morning. LUM-201 will be adjusted for body weight increments at each clinic visit, as applicable.
The trial consists of 12 months of treatment. After screening, subjects will return to the clinic for 5 visits. During four of these clinic visits, safety laboratory samples will be collected.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 13 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.
- •Must have previously completed 12 months of daily rhGH treatment as part of the LUM-201-01 PGHD trial.
- •Is eligible for study participation as confirmed by the principal investigator (PI)
排除标准
- •Has a medical condition that, in the opinion of the PI and/or MM, adds unwarranted risk to use of LUM-
- •Uses any medication that, in the opinion of the PI and/or MM, can independently cause short stature or limit the response to exogenous growth factors.
- •Has planned or is receiving current long-term treatment with medications known to act as substrates, inducers, or inhibitors of the cytochrome system CYP3A4 that metabolizes LUM-201
研究组 & 干预措施
LUM-201 (3.2 mg/kg/day)
干预措施: LUM-201 (Drug)
结局指标
主要结局
Annualized height velocity (AHV) measured as standing height with stadiometer
时间窗: Day 1 to Month 12
Annualized Height Velocity (AHV) measured as standing height with stadiometer
Annualized Height Velocity (AHV) Measured as Standing Height With Stadiometer
时间窗: Day 1 to Month 12
Mean change from day 1 to month 12, Annualized Height Velocity (AHV) Height is measured in triplicate, by a calibrated stadiometer and taking the mean value AVH equation is (h2-h1/t2-t1)\*365.25; h1 = height measured at day 1 h2 = height measured at month 12 t1 = day 1 t2 = month 12
次要结局
- Incidence of adverse events in children with GHD(Day 1 to Month 12)
- Insulin-like growth factor 1 standard deviation score (SDS)(Day 1 to Month 12)
- GH Concentration on maintenance treatment(Day 1 to Month 12)
- Insulin-like growth factor 1(Day 1 to Month 12)
- Insulin-like growth factor binding protein 3(Day 1 to Month 12)
- Height standard deviation score (SDS)(Day 1 to Month 12)
- Change in Weight-SDS(Day 1 to Month 12)
- Change in BMI(Day 1 to Month 12)
- Change in BMI SDS(Day 1 to Month 12)
- Change in Weight(Day 1 to Month 12)
- Change in Bone Age(Month 6)
- Incidence of Adverse Events in Children With GHD(Day 1 to Month 12)
- Growth Hormone (GH) Values(Day 1 to Month 12)
- Insulin-Like Growth Factor 1 (IGF-1) Values(Day 1 to Month 12)
- Insulin-Like Growth Factor Binding Protein 3 (IGFBP-3) Values(Day 1 to Month 12)
- Height Standard Deviation Score (SDS)(Day 1 to Month 12)
- Change in Weight - Standard Deviation Score (SDS)(Day 1 to Month 12)
- Change in Body Mass Index (BMI)(Day 1 to Month 12)
- Change in Body Mass Index (BMI) - Standard Deviation Score (SDS)(Day 1 to Month 12)
- Insulin-Like Growth Factor 1 (IGF-1) - Standard Deviation Score (SDS)(Day 1 to Month 12)
- Occurrence of Adverse Events in Children With Growth Hormone Deficiency (GHD)(Day 1 to Month 12)
- Height (HT) Standard Deviation Score (SDS)(Day 1 to Month 12)