Comparison of Somavaratan (VRS-317), a Long-acting Human Growth Hormone, to Daily rhGH in a Phase 3, Randomized, One-year, Open-label, Multi-center, Non-inferiority Trial in Pre-pubertal Children With Growth Hormone Deficiency
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 138
- 主要终点
- Annual Height Velocity
研究概览
简要总结
The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
详细描述
This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. 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 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).
- •Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in boys.
- •Diagnosis of growth hormone deficiency (GHD) as documented by two or more growth hormone (GH) stimulation test results ≤ 10.0 ng/mL.
- •Height standard deviation score (SDS) ≤ -2.0 at screening.
- •Weight for Stature ≥ 10th percentile.
- •Insulin-like growth factor-I (IGF-I) SDS ≤ -1.0 at screening.
- •Delayed bone age (≥ 6 months).
排除标准
- •Prior treatment with any growth promoting agent
- •History of, or concurrent significant disease (for example, diabetes, cystic fibrosis, renal insufficiency).
- •Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD) or confirmed diagnosis of a named syndrome.
- •A diagnosis of Attention Deficit Hyperactivity Disorder.
- •Daily use of anti-inflammatory doses of glucocorticoid.
- •Prior history of leukemia, lymphoma, sarcoma or cancer.
- •Treatment with an investigational drug in the 30 days prior to screening.
- •Known allergy to constituents of the study drug formulation.
- •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
Somavaratan
Participants will receive somavaratan 3.5 milligrams (mg)/kilogram (kg) subcutaneous (SC) bolus injection twice monthly for 12 months.
干预措施: Somavaratan (Drug)
rhGH
Participants will receive commercially available rhGH (genotropin) 34 micrograms (μg)/kg once daily SC bolus injection for 12 months.
干预措施: rhGH (Drug)
结局指标
主要结局
Annual Height Velocity
时间窗: 12 months
Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) \* 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.
次要结局
- Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader(Baseline, Month 12)
- Change From Baseline in Height Standard Deviation Score (SDS) at Month 12(Baseline, Month 12)
- Change From Baseline in Body Weight at Month 12(Baseline, Month 12)
- Change From Baseline in Body Mass Index (BMI) at Month 12(Baseline, Month 12)
- Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12(Baseline, Month 12)
- Number of Participants With Adverse Events (AEs)(Baseline up to Month 12)
- Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12(Baseline, Month 12)