A Phase II, Randomized, Double-Blind Comparison of Corticosteroid and Corticosteroids With Propranolol Treatment of Infantile Hemangiomas (IH)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Compare Changes in IH Size and Vascularity for the Two Treatment Groups
研究概览
简要总结
This is a prospective randomized, double-blind study to compare the clinical efficacy of infantile hemangioma treatment using propranolol with corticosteroids as compared to therapy with corticosteroids and placebo. We hypothesize that a two-month treatment period with propranolol plus corticosteroids is more effective at reducing infantile hemangioma size and vascularity when compared to corticosteroids used without propranolol for the same time period.
详细描述
Infantile hemangiomas (IH) are the most common head and neck pediatric tumors. Presence of these tumors can lead to complications of vision and airway compromise, bleeding and disfigurement. Medical treatment of these lesions has traditionally been focused on stopping new blood vessel growth with corticosteroids. Recent reports and our own experience have demonstrated that significant reduction in IH size and vascularity can also occur through the use of propranolol. Our initial experience with propranolol has demonstrated significant efficacy with fewer side effects than corticosteroids. Despite this experience, the standard of care for initial IH medical therapy remains corticosteroids.
This Trial is a direct comparison of traditional IH therapy with corticosteroids to newer therapy with propranolol and corticosteroids.
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, Investigator)
入排标准
- 年龄范围
- — 至 9 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 0 to < 9months
- •Patients with clinical, radiographic or histologic diagnosis of infantile hemangioma (IH) requiring medical treatment
- •IH patients whose parents desire medical treatment for the IH
- •Stable cardiac function
排除标准
- •IH patients over 9 months of age.
- •Hypersensitivity to propranolol
- •Untreated heart failure: If the patient has heart failure associated with the hemangioma, propranolol would be initiated after anti-congestive therapy and under observation as an in-patient.
- •Atrioventricular (AV) block
- •Resting heart < 2 SD of normal*(below)
- •Resting blood pressure < 2 SD of normal**(below)
- •Wolff-Parkinson-White syndrome (WPW)
- •History of unexplained syncope
- •Bronchial asthma
- •History of impaired renal or liver function
- •Diabetes mellitus
研究组 & 干预措施
Group A: Corticosteroid with Placebo
Group A will receive oral, liquid Prednisolone, which is the standard corticosteroid that we use here at Seattle Children's, and oral liquid placebo. The dose of prednisolone that Group A will receive will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. This is a standard dose for IH treatment. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone. This treatment will be given for two months, as is our standard practice.
干预措施: Prednisolone (Corticosteroid) (Drug)
Group A: Corticosteroid with Placebo
Group A will receive oral, liquid Prednisolone, which is the standard corticosteroid that we use here at Seattle Children's, and oral liquid placebo. The dose of prednisolone that Group A will receive will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. This is a standard dose for IH treatment. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone. This treatment will be given for two months, as is our standard practice.
干预措施: Placebo (Drug)
Group B: Corticosteroid with Propranolol
Group B will receive oral liquid prednisolone, and oral propranolol. As in Group A, the dose of prednisolone will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. Oral liquid propranolol will be dosed at 2 mg/kg/day, following initiation in the Cardiology Clinic. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone.
干预措施: Prednisolone (Corticosteroid) (Drug)
Group B: Corticosteroid with Propranolol
Group B will receive oral liquid prednisolone, and oral propranolol. As in Group A, the dose of prednisolone will be 1-2mg/kg/day for 7 days and then the dose will be slowly reduced and then stopped after 3 weeks. Oral liquid propranolol will be dosed at 2 mg/kg/day, following initiation in the Cardiology Clinic. Gastric prophylaxis (Zantac) will be given to help prevent any stomach problems associated with prednisolone.
干预措施: Propranolol (Drug)
结局指标
主要结局
Compare Changes in IH Size and Vascularity for the Two Treatment Groups
时间窗: 1, 2, and 6 months after treatment initiation
This outcome cannot be evaluated. Trial limited by lack of patient family to have randomization, which limited the number of participants and made the project nonviable.
Lesion Regression
时间窗: 12 months
measure of change in lesion area or volume
次要结局
- Determine Therapeutic Response of IH to Propranolol Among Patients Who Switch to Corticosteroids Plus Propranolol Therapy After Failing to Respond to Corticosteroids Alone.(1, 2, and 6 months after treatment initiation)
- Assess the Safety of Propranolol With Corticosteroids and Corticosteroids Alone in the Treatment of IH.(1, 2 and 6 months after treatment initiation)
- Demonstrate How Duplex Scanning to Assess Blood Vessel Density and Qualitative Color Ratings of Cutaneous Lesions From Photographs Can be Used to Measure and Quantify Changes in IH Size and Vascularity in a Clinically Relevant Manner.(1, 2 and 6 months after treatment initiation)
研究者
Jonathan Perkins
Principal Investigator
Seattle Children's Hospital