A Phase III Open-Label, Prospective, Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human), IgPro20 in Subjects With Primary Immunodeficiency (PID)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- CSL Behring
- 入组人数
- 49
- 试验地点
- 14
- 主要终点
- Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs) (MITT Population)
研究概览
简要总结
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
详细描述
The entire study consists of a 12-week wash-in/wash-out period followed by a 12-month treatment period. Pharmacokinetic (PK) parameters were assessed in a sub-group of subjects.
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 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female aged 2 to 75 years
- •Subjects with primary humoral immunodeficiency, namely with a diagnosis of: CVID (Common Variable Immunodeficiency) as defined by PAGID (Pan-American Group for Immunodeficiency) and ESID (European Society for Immunodeficiencies) or XLA (X-linked Agammaglobulinemia)
- •Written informed consent
排除标准
- •Newly diagnosed PID
- •Evidence of an active serious infection at the time of screening (i.e., but not limited to: bacteremia/septicemia, pneumonia, fungal osteomyelitis)
- •Malignancies of lymphoid cells such as lymphocytic leukemia, Non-Hodgkin's lymphoma and immunodeficiency with thymoma
- •Known hyperprolinemia
- •Hypoalbuminemia, protein-losing enteropathies, and any proteinuria
- •Allergic reactions to immunoglobulins or other blood products
- •Known antibodies to Immunoglobulin A (IgA)
- •The subject is receiving steroids (oral and parenteral, daily ≥ 0.15 mg of prednisone equivalent/kg/day) or other systemic immunosuppressants
- •Female who is pregnant, breast feeding or planning a pregnancy during the course of the study
- •Participation in a study with an investigational product other than (IVIG) within 1 month prior to enrollment
- •A positive result at screening on any of the following viral markers: Human Immunodeficiency Virus (HIV), Hepatitis C virus (HCV) and Hepatitis B virus (HBV)
- •Aspartate aminotransferase (ASAT) or Alanine aminotransferase (ALAT) concentration > 2.5 times the upper normal limit (UNL)
- •Creatinine concentration > 1.5 times the UNL
- •Any condition that is likely to interfere with evaluation of the study drug or satisfactory conduct of the trial
结局指标
主要结局
Annualized Rate of Clinically Documented Serious Bacterial Infections (SBIs) (MITT Population)
时间窗: Efficacy period: up to 12 months (week 13 to the completion visit)
The annualized rate was based on the total number of SBIs and the total number of subject study days during the efficacy period for all subjects in the specified analysis population and adjusted to 365 days. Potential SBIs included pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, and visceral abscess. If an adverse event (AE) was identified as a potential SBI, the AE was adjudicated by a review committee to determine if the event fulfilled the predefined criteria for SBIs.
Area Under the Concentration-time Curve (AUC) of Total Serum Immunoglobulin G (IgG)
时间窗: Measured during a single dosing interval after at least 12 weeks of stable subcutaneous (SC) dosing with IgPro20 treatment
Evaluate non-inferiority of steady-state IgG area under the concentration-time curves standardized to a 7-day period (sAUCs) for subcutaneous immunoglobulin (SCIG) (IgPro20) versus the sAUC under intravenous immunoglobulin (IVIG) (Privigen) treatment. The sAUC under IVIG was taken from the same subjects in a preceding study (either ZLB03_002CR \[NCT00168025\] or ZLB05_006CR \[NCT00322556\]).
次要结局
- Annualized Rate of Clinically Documented SBIs (ITT Population)(For the duration of the study, up to 15 months)
- Annualized Rate of Clinically Documented SBIs (PPE Population)(Efficacy period: up to 12 months (week 13 to the completion visit))
- Annualized Rate of Infection Episodes(Efficacy period: up to 12 months (week 13 to completion visit))
- Number of Infection Episodes (Serious and Non-serious)(Efficacy period: up to 12 months (week 13 to the completion visit))
- Annualized Rate of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Daily Activities Due to Infections(Efficacy period: up to 12 months (week 13 to the completion visit))
- Number of Days Out of Work / School / Kindergarten / Day Care or Unable to Perform Normal Daily Activities Due to Infections(Efficacy period: up to 12 months (week 13 to the completion visit))
- Annualized Rate of Hospitalization Due to Infection(Efficacy period: up to 12 months (week 13 to the completion visit))
- Number of Days of Hospitalization Due to Infections(Efficacy period: up to 12 months (week 13 to the completion visit))
- Use of Antibiotics for Infection Prophylaxis and Treatment(Efficacy period: up to 12 months (week 13 to the completion visit))
- Total Serum IgG Trough Levels(Every 4 weeks, throughout the 12-month efficacy period)
- Maximum Concentration (Cmax) of Total Serum IgG at Steady State(Week 28 ± 1 week of the treatment period)
- Tmax at Steady State(Week 28 ± 1 week of the treatment period)