A Randomized, Phase 2/3, Open-Label, Multi-center Study of the Pharmacokinetics, Pharmacodynamics, Safety and Efficacy of A Long-acting Human Growth Hormone (Somavaratan, VRS-317) in Pre-pubertal Japanese Children With Growth Hormone Deficiency (GHD)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 41
- 试验地点
- 1
- 主要终点
- Efficacy (Annual Height velocity)
研究概览
简要总结
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. Upon completion of the PK/PD stage, the PK/PD profiles for the GHD children in this study will be compared to the PK/PD profiles for the GHD children treated in the Western study Phase 1b/2a study (Protocol 12VR2) and identify the somavaratan dose to be used in the Phase 3 stage in Japan. The Phase 3 stage will continue dosing for 12 months to obtain safety and efficacy data on 48 subjects.
详细描述
The trial will consist of three stages: 1) a 30 day Phase 2 PK and PD evaluation of somavaratan, 2) an optional Phase 2 Extension and 3) a 12 month Phase 3 safety and efficacy stage. The study is a randomized, multi-center, open label study. The primary endpoint is height velocity at 12 months.
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
- None
入排标准
- 年龄范围
- 3 Years 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological Age ≥ 3.0 years and ≤ 9.0 years (girls) or ≤ 10.0 years (boys)
- •Pre-pubertal status
- •Diagnosis of GHD as documented by two or more GH stimulation test results
- •Height SD score ≤ -2.0 at screening
- •Weight for Stature ≥ 10th percentile
- •IGF-I SD score ≤ -1.0 at screening
- •Delayed bone age
排除标准
- •Prior treatment with any growth promoting agent
- •History of, or current, significant disease
- •Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome
- •Birth weight and/or birth length less than 5th percentile for gestational age
- •A diagnosis of Attention Deficit Hyperactivity Disorder
- •Daily use of anti-inflammatory doses of glucocorticoid
- •Prior history of leukemia, lymphoma, sarcoma or cancer
- •Ocular findings suggestive of increased intracranial pressure and/or retinopathy at screening
- •Significant spinal abnormalities including scoliosis, kyphosis and spina bifida variants
- •Significant abnormality in screening laboratory studies
研究组 & 干预措施
Phase 2: Somavaratan (VRS-317)
Active treatment arm
干预措施: Somavaratan (VRS-317) (Drug)
Phase 3: Somavaratan (VRS-317)
Somavaratan long acting recombinant human growth hormone administered subcutaneously twice-monthly
干预措施: Somavaratan (VRS-317) (Drug)
结局指标
主要结局
Efficacy (Annual Height velocity)
时间窗: 12 months
Annual Height velocity.
次要结局
- Pharmacodynamics (IGF-I responses to study drug administration)(12 months)
- Pharmacodynamics (IGFBP-3 responses to study drug administration)(12 months)
- Safety (Number of subjects with adverse events )(12 months)
- Safety (Concomitant medications)(12 months)
- Safety (Safety labs)(12 months)
- Safety (Vital signs)(12 months)
- Safety (Physical Exams)(12 months)