A Phase IV, Multicenter, Open-label Study to Evaluate the Safety and Pharmacokinetics of BIVIGAM® in Primary Immune Deficiency Disorders in Subjects Aged 2 to 16
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 16
- 主要终点
- Infusion Site Reactions
研究概览
简要总结
This study is part of the BIVIGAM® post marketing requirement (PMR). It is being conducted in subjects aged 2-16 with primary immune deficiency disorders associated with defects in humoral immunity to generate additional data on these populations, and more specifically safety and pharmacokinetic (PK) assessments.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 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
- •Written informed consent/Assent
- •Male or female between 2 and 16 years, inclusive, at time of Signing Informed Consent/Assent
- •Have a confirmed and documented clinical diagnosis of Primary Immune Deficiency Disorder, including hypogammaglobulinemia or agammaglobulinemia.
- •Have received IGIV therapy which was maintained at a steady dose (± 25% of the mean dose) for at least 3 months prior to study entry, and have maintained a trough IgG level at least 500mg/dL prior to receiving BIVIGAM®.
- •Subjects and/or parents/legal guardians must be able to understand and adhere to the study visit schedule and all other protocol requirements.
排除标准
- •Known intolerance to immunoglobulins or comparable substances (e.g. vaccination reaction).
- •Known intolerance to proteins of human origin or known allergic reactions to components of the study product(s).
- •Any previous randomization/participation in this clinical study must be discussed with and approved by the medical director (or designee).
- •Inability or lacking motivation to participate in the study.
- •Medical condition, laboratory finding, or physical exam finding (specify, e.g., vital signs outside of specific range that precludes participation. Per lab results at the Screening visit through Baseline.
- •Confirmed Screening visit laboratory results ˃2.5 X ULN as defined for pediatric populations for any of the following: ALT (alanine aminotransferase/SGPT), AST (aspartate aminotransferase/SGOT), LDH (lactate dehydrogenase), BUN (blood urea nitrogen), Serum creatinine
- •Has selective IgA deficiency or demonstrated antibodies to IgA.
- •History of thrombotic complications of IGIV therapy or history of (deep vein thrombosis)DVT.
- •Current use of daily corticosteroids (>10 mg of prednisone equivalent/day),immunosuppressants or immunomodulators are not allowed unless approved in advance by the medical monitor. Intermittent use of corticosteroids during the study is allowed if medically necessary.
- •Positive diagnosis of hepatitis B or hepatitis C.
- •Positive human immunodeficiency virus (HIV) test.
- •Subject has had a serious bacterial infection (SBI) within the last 3 months.
- •Subject has an active infection and is receiving antibiotic therapy for the treatment of this infection at the time of Screening. Note: if the subject is deemed a Screen Failure due to a nonserious active infection requiring antibiotic therapy, the subject may be rescreened 3 or 4 weeks (depending on drug administration schedule) after the initial screening.
- •Subject has a history of thrombotic events (including deep vein thrombosis, myocardial infarction, cerebrovascular accident and pulmonary embolism) within 6 months before 1st IGIV dose or has preexisting risk factors for thrombotic events.
- •Acquired medical condition known to cause secondary immune deficiency such as chronic lymphacitic leukemia, lymphoma or multiple lymphoma.
- •Subjects with protein-losing enteropathies, hypoalbuminaemia.
- •Females taking oral contraceptives.
- •Pregnancy or unreliable contraceptive measures or lactation period (females of childbearing potential (female capable of becoming pregnant) only. Males capable of reproduction must agree to a double barrier method of contraception during their study participation.
结局指标
主要结局
Infusion Site Reactions
时间窗: Up to approximately 7 months
Incidence reactions occuring at the infusion site
Serious Adverse Events
时间窗: Up to approximately 7 months
Incidence of serious adverse events
Temporally Associated Adverse Events
时间窗: During each infusion (During or within 1 hour, 24 hours and 72 hours of completion of an infusion)
Incidence of adverse events (During or within 1 hour, 24 hours and 72 hours of completion of an infusion)
Related Serious Adverse Events
时间窗: Up to approximately 7 months
Incidence of related serious adverse events
Treatment Emergent Adverse Events
时间窗: Up to approximately 7 months
Incidence of treatment emergent adverse events
Temporally Associated Infusion Adverse Events
时间窗: Up to approximately 7 months
Incidence of adverse events which have a causal relationship with infusion treatment
Non-treatment Emergent Adverse Events
时间窗: Up to approximately 7 months
Incidence of adverse events which do not have a causal relationship with study treatment
Adverse Reactions
时间窗: Up to approximately 7 months
Number and incidence of adverse reactions plus suspected adverse reactions combined
Related Adverse Reactions
时间窗: Up to approximately 7 months
Incidence of adverse infusion related reactions
Temporally Associated Adverse Events Following Infusions
时间窗: Up to 72 hours after each infusion through study completion, up tp approximately 7 months
Incidence of adverse events
Number of Temporally Associated Adverse Events
时间窗: Up to 72 hours of completion of an infusion
Mean number of temporally associated per infusion
Related Treatment Emergent Averse Events
时间窗: Within 72 hours of infusion
Incidence of adverse events that first appear, or that worsen relative to the pre-treatment state, which occur during and within 72 hours of treatment administration
Vital Signs
时间窗: Before and after each administration of study drug through study completion, up to approximately 7 months
Change in vital signs
次要结局
- Serious Bacterial Infections(Up to approximately 7 months)
- IgG subclasses(Prior to first and last infusion, up tp approximately 7 months)
- Cmax(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months)
- Other Infections(Up to approximately 7 months)
- Resolution of Infections(Up to approximately 7 months)
- Missed Days(Up to approximately 7 months)
- Hospitalizations(Up to approximately 7 months)
- Total IgG Trough(At each visit through study completion, up tp approximately 7 months)
- Fever(Up to approximately 7 months)
- Tmax(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months)
- AUC(0-∞)(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after infusion)
- Infections(Up to approximately 7 months)
- Total IgG Post(At each infusion through study completion, up tp approximately 7 months)
- AUC(0-ʈ)(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months)
- Terminal phase elimination half-life (ʈ½)(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months)
- Antibodies(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months)
- First Serious Bacterial Infection(Up to approximately 7 months)
- Terminal phase elimination rate (λZ)(At prior to, at end of infusion, and 6 hours, 24 hours, 7 days, and 4 days, 21 days and 28 days (if still enrolled) after final infusion, up tp approximately 7 months)