A Phase IV, Randomized, Double-Blind, Placebo-Controlled Exploratory Study of Xolair (Omalizumab) for Treatment of Idiopathic Angioedema in Patients Who Remain Symptomatic Despite Current Therapy
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Presence of a 7-day Angioedema Activity Score (AAS7) Greater Than 0 Across All Treatment Visits
研究概览
简要总结
The overall hospitalizations for a diagnosis of angioedema doubled from the year 2000 to 2009. Although some of the cases represented hereditary angioedema or ace-inhibitor induced angioedema, the majority of episodes were idiopathic. Idiopathic Angioedema (IAE) can be life- threatening especially when affecting tissues within the respiratory tract. No clear guidelines exist for management of this important condition for clinicians. Current therapies typically include avoidance of potential triggers and use of medications either for prophylaxis or for acute events, such as antihistamines, corticosteroids, and epinephrine. There remains a critical need for therapeutic options to provide more effective prophylaxis.
详细描述
This study is a randomized, double-blind, placebo-controlled, parallel group trial which will study the effects of omalizumab on patients with 2 or more episodes of Idiopathic Angioedema (IAE) in the past 6 months, despite current therapy. This study has three periods; screening, treatment, and follow-up. Subjects in the screening period will be consented and screened for eligibility criteria. 40 qualified individuals will enter the treatment period. Individuals will be randomized to either monthly subcutaneous administration of omalizumab 300mg (20 subjects) versus monthly placebo injection (20 subjects) in addition to their previously prescribed management plan for a total of 6 months. Individuals will then enter a follow-up period of 4 months. Study visits will occur monthly during the treatment period for update of clinical status and administration of omalizumab/placebo injection. After, the treatment period individuals will be seen twice for follow-up period. The entire study will consist of 10 study visits and will last approx. 10 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Care Provider)
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 or adolescents who are 18 years or older at the time of screening with physician diagnosis of idiopathic angioedema
- •Minimum of two episodes of idiopathic angioedema in the past 6 months at the time of screening
- •Management of idiopathic angioedema with a stable controller treatment plan for the prior 6 months
- •Complement profile (C1 Esterase inhibitor panel) within normal reference values
- •If a woman is of child-bearing potential, she must agree to a reliable form of birth control including: abstinence, oral contraceptives (birth control pills), Depo-provera, an intrauterine device (IUD), or double-barrier contraception (partner using condom and participant using diaphragm, contraceptive sponge or cervical cap, and spermicidal)
排除标准
- •Diagnosis of Hereditary Angioedema (HAE), Acquired Angioedema, or Ace-inhibitor associated angioedema, which are forms of angioedema with known mechanisms and alternate treatment options
- •Chronic Urticaria (itching and/or hives) with or without Angioedema which are known mast cell mediated processes previously shown to be responsive to the use of omalizumab
- •Previous usage of omalizumab in the last 3 months which can affect the patient-related outcomes and biomarker assessments if not "washed out" of the system
- •Patients, who in the judgment of the investigator, have a history or condition that might compromise patient safety or compliance, interfere with evaluations, or preclude completion of the study
研究组 & 干预措施
Placebo
Placebo contains sodium chloride 0.9% and will be provided by Novartis. Placebo will be administered as a subcutaneous injection.
干预措施: Placebos (Drug)
Omalizumab
Omalizumab is a sterile, white, preservative-free, lyophilized powder, contained in a single-use vial that will be reconstituted with sterile water for injection (SWFI), USP, and administered as a subcutaneous injection. Each omalizumab vial contains 202.5 mg of omalizumab, 145.5 mg sucrose, 2.8 mg L-histidine hydrochloride monohydrate, 1.8 mg L-histidine, and 0.5 mg polysorbate 20. Each vial is designed to deliver 150 mg of omalizumab in 1.2 mL after reconstitution with 1.4 mL SWFI, USP.
干预措施: Omalizumab (Drug)
结局指标
主要结局
Presence of a 7-day Angioedema Activity Score (AAS7) Greater Than 0 Across All Treatment Visits
时间窗: across all visits during treatment period (up to 6 months)
The AAS7 Score is a measure of the frequency and intensity of angioedema episodes. The total possible range of scores over 7 days is 0-15 (mean day sum score) where higher scores indicate increased angioedema activity.
Mean 7-day Angioedema Activity Score (AAS7)
时间窗: baseline to end of treatment period at 6 months
The AAS7 Score is a measure of the frequency and intensity of angioedema episodes. The total possible range of scores over 7 days is 0-15 (mean day sum score) where higher scores indicate increased angioedema activity.
次要结局
- Number of Participants Who Used Rescue Medication or Corticosteroids(baseline, end of treatment period at 6 months, follow up at 9 months)
- Presence of Visual Analog Scale Less Than 100 Across All Treatment Visits(across all visits during treatment period (up to 6 months))
- Number of Participants Who Visited Urgent Care or Emergency Room(baseline, end of treatment period at 6 months, follow up at 9 months)
- Mean Angioedema Quality of Life (AE-QoL) Questionnaire(baseline to end of treatment period at 6 months)
- Mean Angioedema Quality of Life (AE-QoL) Questionnaire Across All Treatment Visits(across all visits during treatment period (up to 6 months))
- Mean Visual Analog Scale(baseline to end of treatment period at 6 months)
- Change in Duration of IAE Episodes(baseline to end of treatment period at 6 months)
- Number of Times Rescue Medications Were Used During the Treatment Period(up to 26 weeks)
- Mean Number of IAE Episodes 2 Weeks Prior to Randomization(baseline to end of treatment period at 6 months)
- Number of IAE Events Across All Treatment Visits(across all visits during treatment period (up to 6 months))