An Open-Label, Long-Term Extension Study of the Safety of Somavaratan (VRS-317) in Adults With Growth Hormone Deficiency (GHD)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 36
- 主要终点
- Number of Participants With Adverse Events (AEs)
研究概览
简要总结
Open-label extension study to evaluate the safety of long-term twice-monthly administration of somavaratan in adults with Growth Hormone Deficiency (GHD).
详细描述
An open-label extension study to evaluate the safety of long-term twice-monthly administration of somavaratan in adults with GHD. This multicenter study is open to participants completing a Versartis adult GHD study as well as approximately 40 new somavaratan naïve participants (either recombinant human growth hormone [rhGH] treatment naïve or currently receiving daily rhGH therapy). All participants will receive twice-monthly (every 15 days ± 2 days) subcutaneous (SC) somavaratan. Doses will be titrated to each participant's individual insulin-like growth factor-I (IGF-I) responses based on the IGF-I level 7 days post-dose until a maintenance dose is achieved. Participants receiving somavaratan in a previous somavaratan study will have their dose decreased by half (minimum dose of 20 milligrams [mg], 40 mg for women on estrogen, rounded down to the nearest even number) and will be titrated per the Dose Titration Plan. New participants enrolling in this study will be assigned to one of two cohorts based on sensitivity to rhGH and titrated per the Dose Titration Plan.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Female participants of childbearing potential must have negative pregnancy test and use appropriate contraceptive methods
- •Documented GHD during adulthood
- •Participants naive to somavaratan must have an IGF-1 SDS value ≤ 0 at Screening
- •Participants taking other hormone replacement therapy must have been on a stable course of treatment for at least 3 months
- •Underlying disorders responsible for the participant's GHD must have been clinically stable for at least 6 months
- •Participants receiving daily rhGH injections must washout for ≥ 14 days
- •Body mass index (BMI) (kilograms [kg]/meter square [m^2]) between 18.0 and 40.0
排除标准
- •Untreated adrenal insufficiency
- •Recently diagnosed thyroid dysfunction which is not being treated or has not been stable on therapy for at least 3 months
- •Currently taking anti-inflammatory dose of glucocorticoids that could potentially compromise safety or efficacy assessments
- •Currently taking a GHRH or IGF-I product
- •Current significant cardiovascular disease, heart insufficiency of New York Heart Association (NYHA) class > 2
- •Current significant disease thought to increase risk of receiving growth hormone or confound assessment of study outcomes
- •History of diabetes mellitus or inadequate glucose control
- •Current drug or alcohol abuse
- •Current human immunodeficiency virus (HIV) wasting syndrome (HIV testing not required)
- •History of malignancy in adulthood (participants with a history of childhood malignancy that were subsequently treated with rhGH in childhood and remain GHD in adulthood may be enrolled)
- •Women who are pregnant or breastfeeding
- •Treatment with an investigational drug other than somavaratan within 30 days prior to Screening
- •A significant abnormality in Screening laboratory results
研究组 & 干预措施
Somavaratan
Long-acting recombinant human growth hormone therapy administered subcutaneously twice-monthly in adult participants with GHD
干预措施: somavaratan (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs)
时间窗: From first dose of study drug up to approximately 2 years
An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
次要结局
- Number of Participants With Dose Adjustments(Months 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, and 12)
- Number of Participants With Positive Neutralizing Antibodies (NABs)(Up to approximately 2 years)
- Number of Participants Who Were Anti-drug Antibody (ADA) Positive(Up to approximately 2 years)
- Average Dose Level During Titration/Maintenance(Up to Month 12)
- Change From Baseline in Mean Insulin-like Growth Factor 1 (IGF-I) Standard Deviation Score (SDS) at Specified Timepoints During Maintenance Period(Baseline, Month 2 Day 1, Month 2 Day 8, Month 3 Day 1, Month 3 Day 8, Month 4 Day 1, Month 4 Day 8, Month 5 Day 1, Month 5 Day 8, Month 6 Day 1, Month 6 Day 8, Month 7 Day 1, Month 7 Day 8, Month 8 Day 1, Month 8 Day 8, Month 10 Day 1, and Month 10 Day 8)
- Mean Insulin-like Growth Factor-binding Protein 3 (IGFBP-3) SDS During Maintenance Period(Baseline up to Month 12)