An Open-Label, Single-Arm, Historically Controlled, Prospective, Multi-Center Phase III Study to Evaluate the Pharmacokinetics and Safety of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
- 试验地点
- 8
- 主要终点
- The Pharmacokinetic (PK) Volume of distribution of total IgG
研究概览
简要总结
The purpose of this study is to evaluate the pharmacokinetics and safety of Immune Globulin Intravenous (Human) GC5107 in pediatric subjects with Primary Humoral Immunodeficiency (PHID).
详细描述
This is a prospective, open-label, single-arm, historically controlled, multi-center Phase III study to assess the pharmacokinetics and safety of Immune Globulin Intravenous (Human) GC5107 in pediatric subjects aged ≥ 2 years and < 17 years with PHID.
Subjects will receive intravenous infusions of the investigational product at the same dose and interval as used for their previous Immunoglobulin intravenous (IGIV) maintenance therapy. GC5107 will be infused every 21 or 28 days for a period of 12 months.
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
- 2 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must be ≥ 2 to < 17 years of age, at the time of signing the informed consent
- •Pediatric subject has a confirmed and documented clinical diagnosis of Primary Humoral Immunodeficiency, including hypogammaglobulinemia or agammaglobulinemia
- •Subject who has received 300 - 900 mg/kg of IGIV therapy at 21 or 28 day intervals for at least 3 months prior to this study
- •Subject who has at least 2 documented plasma IgG trough level of ≥ 500 mg/dL at two infusion cycles (21 or 28 days) within 12 months prior to enrollment
- •Subject who is willing to comply with all requirements of the protocol
排除标准
- •Subject who has a history of clinically significant reactions or hypersensitivity to IGIV or other injectable forms of IgG
- •Subject who has IgA deficiency and is known to have antibodies to IgA
- •Subject who has secondary immunodeficiency
- •Subject who has participated in another clinical study (other than an IGIV study) within 3 weeks prior to screening
- •Subject who has been diagnosed with dysgammaglobulinemia or isolated IgG subclass deficiency or isolated IgA deficiency, or who has clinically significant impairment of cellular or innate immunity at the discretion of the Investigator
- •Subject who has received blood products other than human albumin or human immune globulin within 6 months prior to enrollment
研究组 & 干预措施
GC5107
Immune Globulin Intravenous (Human), 10% Liquid
干预措施: GC5107 (Biological)
结局指标
主要结局
The Pharmacokinetic (PK) Volume of distribution of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Minimum concentration of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Area under the curve of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Plasma concentration-time curve of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Half-life of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Time of maximum concentration of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The Pharmacokinetic (PK) Clearance of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
The proportion of infusions with temporally associated adverse events (AEs) that occur during or within 1 hour, 24 hours, and 72 hours following an infusion of investigational product
时间窗: 12 months
AEs that occur during or within 1 hour, 24 hours, and 72 hours following each infusion during 12 months of the study period
The Pharmacokinetic (PK) Maximum concentration of total IgG
时间窗: before and after 5th infusion (12 or 16 weeks)
Trough serum total IgG levels before each infusion of GC5107 in all subjects and the interval between infusions
时间窗: 12 months
次要结局
- Number and proportion of subjects who failed to meet the target IgG trough level (500 mg/dL) at any time point equal to or subsequent to 5th infusion (estimated 5 half-lives)(12 months)
- The Pharmacokinetic (PK) Minimum concentration of IgG subclasses(before and after 5th infusion (12 or 16 weeks))
- Trough serum level of IgG subclasses and specific IgG antibodies before Infusion 1 and 13 (for subjects on 28-day infusion schedule) or Infusion 1 and 17 (for subjects on 21-day infusion schedule)(12 months)
- Viral safety (freedom from transmission of blood-borne viral diseases): the human immunodeficiency virus (HIV) type 1 & 2, hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), and parvovirus B19(13 months (12 months of treatment + 1 month of follow-up))
- The overall incidence of all AEs that occur during or within 1 hour, 24 hours, and 72 hours following an infusion of investigational product(12 months)
- The frequency of all AEs that occur during the study regardless of the investigator's assessment of their relationship to investigational product(13 months (12 months of treatment + 1 month of follow-up))
- The Pharmacokinetic (PK) Half-life of IgG subclasses(before and after 5th infusion (12 or 16 weeks))
- The Pharmacokinetic (PK) Maximum concentration of IgG subclasses(before and after 5th infusion (12 or 16 weeks))
- The frequency of suspected adverse reactions as defined by all AEs either classified as at least possibly related to GC5107(13 months (12 months of treatment + 1 month of follow-up))
- The proportion of AEs considered by the investigator to be investigational product related(13 months (12 months of treatment + 1 month of follow-up))
- The number and proportion of GC5107 infusions for which the infusion rate was decreased due to AEs(12 months)