A Long-acting Human Growth Hormone (VRS-317) in Pre-pubertal Children With Growth Hormone Deficiency: A Randomized, Open-label, Multi-center, Phase 1b/2a Study of Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Efficacy After Subcutaneous Administration for 6 Months
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 64
- 试验地点
- 23
- 主要终点
- Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
研究概览
简要总结
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317. During the Phase 2a stage, patients will receive 6 months of VRS-317 treatment at dose levels selected from the Phase 1b stage. The primary endpoints for the study are to determine the safety and efficacy of repeat dose VRS-317.
详细描述
In Phase 1b, separate cohorts of patients will be tested in a single ascending dose format. Safety review committee meetings will take place prior to escalating to each increasing dose level. Enrolled patients will be monitored for 60 days for safety with PK/PD and safety labs collected.
Two dose levels will be selected after completion of Phase 1b to be tested during Phase 2a (6 months of continuous VRS-317 treatment). Safety and PK assessments will be made during the Phase 2a stage. Patient heights will be measured by stadiometer.
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 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological Age ≥ 3.0 years and ≤ 11.0
- •Diagnosis of GHD as documented by GH stimulation test
- •Below average height SDS at screening
- •Appropriate weight for Stature
- •Decreased IGF-I SDS at screening
- •Delayed bone age
- •Normal thyroid function test results at screening visit
- •Legally authorized representative informed consent.
排除标准
- •Prior treatment with any growth promoting agent
- •Documented history of, or current, significant disease
- •Chromosomal aneuploidy, significant gene mutations
- •Diagnosis of Attention Deficit Hyperactivity Disorder
- •Daily use of anti-inflammatory doses of glucocorticoid
- •Prior history of leukemia, lymphoma, sarcoma or cancer
- •Known allergy to constituents of the study drug formulation
- •Abnormal ocular findings at screening
- •Significant abnormality in screening laboratory studies
结局指标
主要结局
Phase 1b and 2a: Evaluate the safety and tolerability of multiple dosing levels of subcutaneous (SC) VRS-317 in pediatric growth hormone deficient (GHD) patients. Phase 2a: Evaluate the efficacy of multiple dose levels of VRS-317
时间窗: 8 Months
Safety observations will include the number of patients with adverse events, concomitant medications, safety labs, vital signs, physical exams, single dose immunogenicity (Phase 1b), and repeat dose immunogenicity (Phase 2a). The primary efficacy outcome measure is the height velocity over 6 months as measured by standing height taken with stadiometer at baseline and after 6 Months of VRS-317 dosing in Phase 2a.
次要结局
- Evaluate pharmacokinetic measures of VRS-317.(8 months)