Nadolol for Proliferating Infantile Hemangiomas: A Prospective Open Label Study With a Historical Control
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Proportion of subjects with at least 75% improvement in the extent of the hemangioma
研究概览
简要总结
The purpose of this study is to explore the efficacy and safety of Nadolol in hemangiomas of infancy.
The secondary objective is to assess the feasibility of conducting a randomized controlled trial comparing nadolol with corticosteroids and propranolol.
详细描述
Systemic corticosteroids are currently the most frequent used medication for treatment of problematic infantile hemangiomas (IH's). Since June 2008, systemic propranolol has been an important addition to the therapeutic options for problematic IH, allowing decreased dependence on the systemic corticosteroids. So far, we have found excellent response with propranolol with minimal short-term side effects. Studies, which compared nadolol and propranolol in children with other conditions, suggest that nadolol is safer and more efficacious than propranolol. In addition, it has better dosing schedules and less central nervous system (CNS) penetration, making it suitable even for patients with suspected or proven PHACES syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Month 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Intervention Group
- •Infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement.
- •Historical Control Group
- •Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years
- •Angiogenesis Marker Control Group
- •Infants aged 1 month to 1 year attending dermatology clinic
排除标准
- •Intervention Group
- •Patients with PHACES syndrome (proven) or suspected PHACES (plaque like hemangioma awaiting imaging).
- •Children with history of hypersensitivity to beta blockers
- •Children with personal history or family history of a first degree relative with asthma
- •Children with known renal impairment
- •Children with known cardiac conditions which may predispose to heart blocks
- •Personal history of hypoglycemia
- •Children on medications that may interact with beta blockers
- •Historical Control Group:
- •No digital photography available documenting IHs progression
- •Angiogenesis Marker Control Group:
- •Children with IH
- •Children on beta blocker or systemic corticosteroids
研究组 & 干预措施
Intervention Group:
The patients in this study are infants aged 1 month to 1 year of age with head and neck hemangiomas currently causing /or with impending function loss (e.g. vision, airway obstruction, feeding, etc), or hemangiomas currently causing/or with potential for facial disfigurement
Infants aged 1 month to 1 year of age with head and neck hemangiomas that received treatment with systemic propranolol in the past 2 years as a control group
干预措施: Nadolol (Drug)
结局指标
主要结局
Proportion of subjects with at least 75% improvement in the extent of the hemangioma
时间窗: Baseline, 6months
次要结局
- The correlation between the changes in the levels of angiogenesis markers and clinical response to treatment.(6 months)
- The proportion of subjects with at least 50% improvement in the extent of the hemangiomas(Baseline and 6 months)
- The percentage of patients with >75% improvement in the Nadolol group compared to a historical cohort of patients receiving propranolol.(6 months)
研究者
Elena Pope
Staff Physician
The Hospital for Sick Children