An Investigator Driven Observational Study to Determine the Benefit of Octagam 5% for Treatment of Patients Diagnosed With Primary Immunodeficiency Disorders (PID) on Intravenous Immunoglobulin (IVIG) Therapy That Experience Adverse Events (AEs) on Any 10% IVIG Preparation
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 15
- 主要终点
- The change in the number of AEs post-infusion between any 10% IVIG product and octagam 5%
研究概览
简要总结
Patients with primary immunodeficiency disorders (PID) on intravenous immunoglobulin (IVIG) treatment may experience adverse events (AEs). Patients who experience AEs on any 10% IVIG solution will be changed to octagam 5% for six infusions to evaluate the potential benefit for reduction of AEs on a lower concentration IVIG product.
详细描述
Patients with PID require life long immunoglobulin (Ig) replacement therapy with IVIG being the most common form. As more 10% IVIG products are FDA approved, the older and well characterized 5% IVIG products are becoming less used. Currently, the standard of care for patients who experience AEs on IVIG is to move to a subcutaneous (SCIG) delivery and product. This study will evaluate the AEs on a 10% product and octagam 5%. The study will enroll 15 patients after an AE on any 10% product who will then be infused with octagam 5% for six infusions. AEs will be documented and compared to the 10% product along with changes in biomarkers. The study data may document another therapeutic option for patients who experience AEs - SCIG and octagam 5%.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 10 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants, or legal guardians with assent by underage children, will sign informed consent/assent and are willing to comply with all aspects of the study
- •Diagnosis of CVID according IUIS Expert Committee
- •Participants on a 10% product who experience AEs
- •Ages between 10 and 75 years of age
- •Participants on 10% IVIG therapy every 21±3 days or 28±3 days between 300 - 800 mg/Kg body weight
排除标准
- •Acute infection requiring antibiotic therapy within 7 days prior to visit 1
- •Presence of any condition that is likely to interfere with the evaluation of the study medication or satisfactory conduct of the trial
- •History of anaphylactic or severe systemic reactions to human immunoglobulin
- •IgA deficient patients with antibodies against IgA and a history of hypersensitivity
- •Females who are pregnant or lactating
结局指标
主要结局
The change in the number of AEs post-infusion between any 10% IVIG product and octagam 5%
时间窗: AEs will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks
次要结局
- Safety Evaluations (complete blood count [CBC])(Screening and prior to each infusion (six infusions total) up to 24 weeks)
- Safety evaluations (Complete Metabolic profile[CMP])(Screening and prior to each infusion (six infusions total) up to 24 weeks)
- The change in levels of inflammatory biomarkers associated with AEs between any 10% IVIG and octagam 5%(Levels will be documented at screening and up to 72 hours post-infusion for six infusions up to 24 weeks)
- Safety evaluations (IgG trough level)(Screening and prior to last infusion up to 24 weeks)