An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children With Idiopathic Growth Hormone Deficiency
试验速览
- 阶段
- 2 期
- 状态
- Enrolling By Invitation
- 发起方
- Lumos Pharma
- 入组人数
- 60
- 试验地点
- 7
- 主要终点
- Incidence of treatment-emergent adverse events
研究概览
简要总结
This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.
详细描述
An Extension Study to Monitor Long-Term Safety of LUM-201 Treatment in Children with Idiopathic Growth Hormone Deficiency. This study will last for up to 36 months to allow collection of additional long-term safety and efficacy data related to LUM-201 treatment in the pediatric patient population. Subjects will enter into this trial after successful participation in a prior LUM-201 study. This study will consist of visits every 6 months through three years.
There are a total of 7 in-person visits with a follow-up phone call between visits. At the clinic visits, subjects will have a physical exam and blood collection as well as efficacy assessments.
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
- 5 Years 至 14 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 successfully participated in a pediatric LUM-201 GHD study through at least the 12-month visit, and be eligible for continuation of treatment, pending all other enrollment criteria are met.
排除标准
- •Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201
- •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-
- •Subjects receiving shorter-term (two weeks or less) treatment with these medications should be evaluated on case-by-case basis by the PI in consultation with the MMs.
研究组 & 干预措施
LUM-201 (1.6 mg/kg/day)
干预措施: LUM-201 (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events
时间窗: Day 1 to Month 36
Number of events
次要结局
- Change in Weight SDS(Day 1 to Month 36)
- Annualized Height Velocity (AHV) achieved by daily dosing 1.6mg/kg of LUM-201(Day 1 to Month 36)
- Change in Bone Age (BA)(Day 1 to Month 36)
- Growth Hormone Concentrations in subjects(Day 1 to Month 36)
- Insulin-like growth factor 1 concentration(Day 1 to Month 36)
- Height standard deviation score (SDS)(Day 1 to Month 36)