Observational Study of the Use of KRYSTEXXA® (Pegloticase) in Adult Hyperuricemic Patients With Gout Refractory to Conventional Therapy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 188
- 试验地点
- 67
- 主要终点
- Number of Participants With Infusion Reactions
研究概览
简要总结
The primary purpose of this study is to observe patients being treated with pegloticase in a standard healthcare setting in order to evaluate the frequency and severity of infusion reactions, anaphylaxis and immune complex related events. Additionally, serious adverse events associated with pegloticase therapy will be identified.
详细描述
This was a Phase 4, multicenter, open-label, single-arm observational study of pegloticase 8 mg administered intravenously every 2 weeks in adult hyperuricemic patients with gout refractory to conventional therapy. Study duration is approximately 63 weeks, including 51 weeks of treatment and 12 weeks of follow-up.
The design of this study follows the FDA-approved Full Prescribing Information for the use of pegloticase and allows for capturing additional data related to the safety and efficacy of pegloticase within the standard healthcare setting.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults (age 18 years or more) with chronic gout refractory to conventional therapy, defined as patients who have failed to normalize SUA and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose, or for whom these drugs are contraindicated.
- •Patients who have made the decision, along with their treating physician, to begin treatment with KRYSTEXXA.
- •Patients who are willing and able to give informed consent and adhere to visit/protocol schedules.
排除标准
- •Glucose-6-phosphate dehydrogenase (G6PD) deficiency
- •Non-compensated congestive heart failure
- •Pregnancy or breast feeding
- •Prior treatment with pegloticase or another recombinant uricase
- •Known allergy to urate oxidase
- •Prior treatment or concomitant therapy with a polyethylene glycol (PEG)-conjugated drug
- •Recipient of an investigational drug within 4 weeks prior to study drug administration or plans to take an investigational agent during the study
结局指标
主要结局
Number of Participants With Infusion Reactions
时间窗: 52 weeks
Infusion reactions were defined as adverse events (AEs) or clusters of events, not attributable to another cause that occurred during or within 2 hours after the infusion of pegloticase. Any other case that occurred outside of the 2-hour window was categorized per Investigator discretion.
Number of Participants With Anaphylaxis
时间窗: 52 weeks
Anaphylaxis was defined using the National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network (NIAID/FAAN) criteria: Acute onset of an illness (minutes to several hours) with involvement of the skin, mucosal tissue, or both (e.g., generalized hives; pruritus or flushing; swollen lips, tongue, or uvula), and at least 1 of the following: 1. Respiratory compromise (e.g., dyspnea, wheeze-bronchospasm, stridor, reduced peak expiratory flow, hypoxemia). 2. Reduced blood pressure (i.e., systolic blood pressure \< 90 mm Hg or greater than 30% decrease from that patient's baseline) or associated symptoms of end-organ failure (e.g., hypotonia \[collapse\], syncope, incontinence).
Number of Participants With Immune Complex-related Events
时间窗: From first dose of study drug to the end of the 12-week follow-up period (63 weeks).
Immune complex-related events were defined as any presumptive immune complex-related disorders that were confirmed by an appropriate investigation of the event and of complement markers (C3 and C4 levels). Clinical manifestations could have included skin rash, arthralgia, arthritis, proteinuria, serum sickness, and cryoglobulinemia.
次要结局
- Number of Tender Joints Over Time(Baseline and weeks 24 and 52)
- Number of Swollen Joints Over Time(Baseline and weeks 24 and 52)
- Percentage of Participants With Normalization of Serum Uric Acid at Week 24 and Week 52(Week 24 and week 52)
- Number of Palpable Tophi Over Time(Baseline and weeks 24 and 52)
- Change From Baseline in Number of Gout Flares(Baseline, week 24 and week 48)