An Open-Label, Dose Finding, International Phase 2 Study With Once Monthly Subcutaneous VRS-317 in Adult Growth Hormone Deficiency (GHD)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 18
- 主要终点
- Safety as measured by the number of subjects with adverse events, concomitant medications, safety labs, vital signs and physical exams
研究概览
简要总结
A Phase 2, open-label dose-finding safety study of individualized monthly VRS-317 dosing for five months in adults with GHD.
详细描述
A Phase 2, dose finding safety study to evaluate an individualized monthly VRS-317 dosing regimen in adults with GHD. This is an open-label, international, multicenter study with VRS-317 treatment for five months. This treatment period will include monthly dose titrations until a subject's mean IGF-I SDS value is within a target range for two consecutive months. Subjects will be stratified into three cohorts based on sensitivity to rhGH.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 23 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Female subjects of childbearing potential must have negative pregnancy test and use appropriate contraceptive methods
- •Subjects must have documented GHD during adulthood.
- •Subjects taking other hormone replacement therapy must have been on a stable course of treatment for at least 3 months.
- •Subjects with underlying disorders responsible for the subject's GHD must have been clinically stable for at least 6 months.
- •Subjects receiving daily rhGH injections must washout for 14 days.
- •Subjects must provide signed informed consent.
- •Subjects must have a BMI (kg/m2) between 19.0 and 35.0.
排除标准
- •Subjects with diabetes mellitus or inadequate glucose control
- •Subjects with untreated adrenal insufficiency.
- •Subjects with free thyroxine outside the normal reference range.
- •Subjects currently taking oral glucocorticoids, except for physiological maintenance doses of oral glucocorticoids in subjects with multiple pituitary hormone deficiencies.
- •Subjects with current significant cardiovascular disease, heart insufficiency of NYHA class >
- •Subjects with current significant cerebrovascular, pulmonary, neurological, renal, inflammatory, or hepatobiliary disease.
- •Subjects with current papilledema.
- •Subjects with a history of persistent or recurring migraines.
- •Subjects with current edema (≥ CTCAE Grade 2).
- •Subjects with current drug or alcohol abuse.
- •Subjects with a documented history of HIV, current HBV or HCV infection
- •Subjects with a prior history of malignancy excluding adequately treated non-melanoma skin cancers or in situ carcinoma of the cervix.
- •Women who are pregnant or breastfeeding.
- •Subjects with a significant abnormality in Screening laboratory results
研究组 & 干预措施
Cohort A: Somavaratan in Older Adults
Long-acting recombinant human growth hormone therapy administered subcutaneously once monthly in subjects >= 35 years of age
干预措施: somavaratan (Drug)
Cohort B: Somavaratan in Younger Adults
Long-acting recombinant human growth hormone therapy administered subcutaneously once monthly in subjects < 35 years of age
干预措施: somavaratan (Drug)
Cohort C: Somavaratan in Women on Estrogen
Long-acting recombinant human growth hormone therapy administered subcutaneously once monthly in female subjects on oral estrogen (regardless of age)
干预措施: somavaratan (Drug)
结局指标
主要结局
Safety as measured by the number of subjects with adverse events, concomitant medications, safety labs, vital signs and physical exams
时间窗: 5 months
Safety observations include adverse events, concomitant medications, safety labs, vital signs and physical exams.
Starting doses (proportion of subjects who achieve normalization of IGF-I SDS response during the first dosing interval )
时间窗: 5 months
To evaluate the starting doses of VRS-317 for each cohort as measured by the proportion of subjects who achieve normalization of IGF-I SDS response during the first dosing interval (one month after the first dose)
Dose titration plan (proportion of subjects who achieve a mean IGF-I SDS within the defined target range after each dose titration)
时间窗: 5 months
To evaluate the dose titration plan of VRS-317 for each cohort as measured by the proportion of subjects who achieve a mean IGF-I SDS within the defined target range after each dose titration
次要结局
- Immunogenicity of VRS-317 by detection of neutralizing antibodies (NAbs)(5 months)
- Immunogenicity of VRS-317 by measurement of serum anti-drug antibody (ADA) titers(5 months)