Clinical Study to Evaluate the Efficacy, Pharmacokinetics and Safety of Immunoglobulin Intravenous (Human) 10% (NewGam) in Patients With Primary Immunodeficiency Diseases
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 51
- 试验地点
- 8
- 主要终点
- Number of Serious Bacterial Infections Per Person-year of Treatment
研究概览
简要总结
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam. The safety of NewGam and its effect on quality of life were also evaluated.
详细描述
NewGam is a new 10% human normal immunoglobulin (IVIG) solution developed by Octapharma for intravenous administration. It is supplied as a liquid formulation ready to use. The primary therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). IVIG has proved to be useful in a variety of clinical conditions other than for replacement of immunoglobulins; IVIG exhibits an immunomodulatory effect. Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections that typically attack the respiratory tract (sinusitis, bronchitis, pneumonia) but can also affect the gastrointestinal tract (gastroenteritis). Theses diseases can be severe and can lead to substantial morbidity. Responses to antibacterial therapy are often poor. At present, most primary immune deficiencies are not curable, but IVIGs have been shown to decrease the total number of severe infections and the duration of hospitalization.
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
- •Age of ≥ 2 years and ≤ 75 years.
- •Confirmed diagnosis of common variable immunodeficiency (CVID) or X-linked agammaglobulinemia (XLA).
- •Previously treated with a commercial immune globulin intravenous (human) every 21-28 days for at least 6 infusion intervals at a constant dose between 200 and 800 mg/kg body weight.
排除标准
- •Acute infection requiring intravenous antibiotic treatment within 2 weeks prior to and during the screening period.
- •Exposure to blood or any blood product or derivative, other than commercially available intravenous immunoglobulin (IVIG), within the past 3 months prior to enrollment.
- •Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational product.
- •Requirement of any routine pre-medication for IVIG infusion.
- •Severe liver function impairment (alanine aminotransferase [ALAT] 3x > upper limit of normal).
- •Presence of renal function impairment (creatinine > 120 μmol/L), or predisposition for acute renal failure (eg, any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
- •History of autoimmune hemolytic anemia.
- •History of diabetes mellitus.
- •Congestive heart failure New York Heart Association (NYHA) class III or IV.
- •Non-controlled arterial hypertension (systolic blood pressure > 160 mmHg or diastolic blood pressure > 90 mmHg).
- •History of deep vein thrombosis or thrombotic complications of IVIG therapy.
- •A positive result at screening on any of the following viral markers: human immunodeficiency virus (HIV), hepatitis C virus (HCV), hepatitis B virus (HBV).
- •Treatment with steroids (oral or parenteral, long-term, ie, 30 days or more, not intermittent or burst, daily, ≥ 0.15 mg of prednisone or equivalent/kg/day), immunosuppressive or immunomodulatory drugs.
- •Planned vaccination during the study period.
- •Treatment with any investigational agent within 3 months prior to enrollment.
- •Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to enrollment.
- •Pregnant or nursing women.
结局指标
主要结局
Number of Serious Bacterial Infections Per Person-year of Treatment
时间窗: Baseline to end of the study (up to 12 months)
The number of serious bacterial infections per person-year of treatment was calculated by the following formula: Total number of serious bacterial infections / patient-years on NewGam treatment. Serious bacterial infections were defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess.
次要结局
- Percentage of Participants With at Least 1 Episode of Fever(Baseline to end of the study (up to 12 months))
- Trough Level Concentration of Antibodies Against Varicella-zoster Virus(Baseline to end of the study (up to 12 months))
- Total Number of Infections(Baseline to end of the study (up to 12 months))
- IgG Trough Level Concentration(Baseline to end of the study (up to 12 months))
- Trough Level Concentration of Antibodies Against Haemophilus Influenzae(Baseline to end of the study (up to 12 months))
- Trough Level Concentration of Antibodies Against Streptococcus Pneumoniae(Baseline to end of the study (up to 12 months))
- Trough Level Concentration of Antibodies Against Cytomegalovirus(Baseline to end of the study (up to 12 months))
- Number of Non-serious Infections(Baseline to end of the study (up to 12 months))
- Number of Antibiotic Treatment Episodes Per Person-year of Treatment(Baseline to end of the study (up to 12 months))
- Number of Antibiotic Treatment Days Per Person-year of Treatment(Baseline to end of the study (up to 12 months))
- Percentage of Participants That Missed School or Work Due to an Infection(Baseline to end of the study (up to 12 months))
- Time to Resolution of Serious and Other Infections(Baseline to end of the study (up to 12 months))
- Percentage of Participants Treated With Antibiotics(Baseline to end of the study (up to 12 months))
- Number of Participants Hospitalized Due to an Infection(Baseline to end of the study (up to 12 months))
- Trough Level Concentration of Antibodies Against Measles(Baseline to end of the study (up to 12 months))
- Trough Level Concentration of Antibodies Against Tetanus(Baseline to end of the study (up to 12 months))
- Changes in the Physical and Psychosocial Child Health Questionnaire-Parent Form Scores From Baseline to the End of the Study(Baseline to end of the study (up to 12 months))
- Changes in the Physical and Mental Short Form-36 Health Survey Scores From Baseline to the End of the Study(Baseline to end of the study (up to 12 months))