NCT03988426已完成3 期
Clinical Phase 3 Study to Evaluate the Efficacy, Tolerability and Safety of Subcutaneous Human Immunoglobulin (Octanorm) in Patients With Primary Immunodeficiency Diseases.
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Octapharma
- 入组人数
- 25
- 试验地点
- 10
- 主要终点
- Number of Serious Bacterial Infections Per Person-Year on Treatment
研究概览
简要总结
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
详细描述
Octanorm (cutaquig®) is a 16.5% human normal immunoglobulin solution developed by Octapharma for subcutaneous administration (SCIG). It is supplied as a liquid formulation ready to use. One important therapeutic use of immunoglobulins is to provide antibodies to prevent viral and bacterial diseases (replacement therapy). Children and adults with a Primary Immunodeficiency Disease (PID) have an increased risk of recurrent bacterial and viral infections. These 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 SCIGs have been shown to decrease the total number of severe infections and the duration of hospitalization. This study evaluated the efficacy, safety and tolerability of octanorm in adult PID patients in an open-label, multi center, phase 3 study with an 8-week wash-in/wash-out period followed by a 6-month efficacy period
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 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age of ≥18 years and ≤70 years.
- •Confirmed diagnosis of PI requiring immunoglobulin replacement therapy due to hypogammaglobulinaemia or agammaglobulinaemia. The type of PI should be recorded.
- •Patients with at least 4 infusions on regular treatment with any Intravenous Immunoglobulin (IVIG) prior to entering the study. Constant IVIG dose between 200 and 800 mg/kg body weight (the individual doses of the last 4 infusions should not vary by more than ±25% of the mean dose for the last 4 infusions).
- •Availability of at least 2 IgG trough levels with an IgG level of ≥5.0 g/L from the period of the last 4 IVIG infusions.
- •Negative result on a pregnancy test (Human Chorionic Gonadotrophin [HCG]-based assay in urine) for women of childbearing potential and use of a reliable method of contraception for the duration of the study. Women of non-childbearing potential must be post-menopausal (amenorrhoeic for at least 12 months) or surgically sterile.
- •Examples for medically acceptable methods of birth control for this study include:
- •Oral, implantable, transdermal or injectable contraceptives
- •Intrauterine device
- •Condoms; diaphragm or vaginal ring with spermicidal jellies or cream
- •Sexual abstinence
- •Vasectomised partner
- •Patient must freely give written informed consent.
- •Willingness to comply with all aspects of the protocol, including blood sampling, for the duration of the study.
排除标准
- •Acute infection requiring intravenous (IV) antibiotic treatment within 2 weeks prior to and during the screening period.
- •Known history of adverse reactions to Immunoglobulin A in other products.
- •Patients with body mass index >40 kg/m2
- •Exposure to blood or any blood product or plasma derivatives, other than IVIG treatment of PI, within the past 3 months prior to first infusion of octanorm.
- •Ongoing history of hypersensitivity or persistent reactions to blood or plasma derived products, or any component of the investigational medicinal product (IMP) (such as Polysorbate 80).
- •History of malignancies of lymphoid cells and immunodeficiency with lymphoma.
- •Severe liver function impairment (ALAT 3 times above upper limit of normal).
- •Known protein-losing enteropathies or proteinuria.
- •Presence of renal function impairment (creatinine >120 µM/L or creatinine >1.35 mg/dL), or predisposition for acute renal failure (e.g., any degree of pre-existing renal insufficiency or routine treatment with known nephritic drugs).
- •Treatment with enteral or parenteral steroids for ≥30 days or when given intermittently or as bolus, at daily doses ≥0.15 mg/kg. Inhaled corticosteroids are allowed.
- •Patients with chronic obstructive pulmonary disease (COPD) stage Global Initiative for Chronic Obstructive Lung Disease (GOLD) III or IV.
- •Treatment with immunosuppressive drugs.
- •Live viral vaccination (such as measles, rubella, mumps and varicella) within the last 2 months prior to first infusion of octanorm.
- •Treatment with any IMP within 3 months prior to first infusion of octanorm.
- •Presence of any condition that is likely to interfere with the evaluation of study medication or satisfactory conduct of the trial.
- •Known or suspected to abuse alcohol, drugs, psychotropic agents or other chemicals within the past 12 months prior to first infusion of octanorm.
- •Known or suspected human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV) infection.
- •Pregnant or nursing women; planned pregnancy during course of the study
结局指标
主要结局
Number of Serious Bacterial Infections Per Person-Year on Treatment
时间窗: Primary Treatment Period (24 Weeks)
Serious Bacterial Infections defined as bacteraemia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess
次要结局
- Hospitalizations Due to Infection(Primary Treatment Period (24 Weeks))
- Annual Rate of Antibiotic Use(Primary Treatment Period (24 Weeks))
- Patients With Days Missed From Work/Study Due to Infections and Treatment(Primary Treatment Period (24 Weeks))
- Episodes of Fever(Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks))
- Rate of Episodes of Fever(Primary Treatment Period (24 Weeks))
- Number of Patients With Other Infections(Primary Treatment Period (24 Weeks))
- Number of Other Infections(Primary Treatment Period (24 Weeks))
- Time to Resolution of Infections(Primary Treatment Period (24 Weeks) and Whole Treatment Period (up to 36 Weeks))
- Number of Participants Using Antibiotics From 0 to > 20 Days(Primary Treatment Period (24 Weeks))
- Rate of Hospitalizations Due to Infection(Primary Treatment Period (24 Weeks))
- Changes in the Subscales of the Form-36 Health Survey Scores From Baseline to the End of the Study(Baseline to the end of study (up to 36 weeks))
- Trough Levels of Serum Total IgG(At baseline and at last infusion (week 33))
- Number of Participants Experiencing Treatment-Emergent AEs(Up to 36 weeks)
- Proportion of Infusions With at Least 1 Temporally Associated AE(Up to 36 weeks)
- Number of Related Adverse Events(Up to 36 weeks)
- Total Number of Adverse Events Regardless of Causality(Up to 36 weeks)
- Number of Infusions With Infusion Site Reaction(Up to 36 weeks)
- Annual Rate of Infections(Up to 36 weeks)
研究者
研究点 (10)
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