Propranolol vs Prednisolone for Infant Hemangiomas-A Clinical and Molecular Study
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 发起方
- 入组人数
- 19
- 试验地点
- 1
- 主要终点
- Decrease in Size of Hemangioma (Length x Width) in Square mm
研究概览
简要总结
Hemangiomas are relatively common lesions in infants. Most go away spontaneously after one year of life and do not need treatment. Others require treatment because they cause significant symptoms such as pain, or difficulty with breathing, eating or ambulating. Steroids have classically been used to treat hemangiomas and help to shrink them in 1/3 - 2/3 of patients. Unfortunately, steroids have many side effects in babies so physicians have sought other ways to treat them. Recently, the use of propranolol, a heart medication, was serendipitously found to reduce the size of hemangiomas. It appears to have many fewer side effects than steroids but it is not yet known if it works as well as steroids. This study seeks to compare the effect and the side effects of propranolol versus steroids for treating hemangiomas that cause symptoms in infants.
详细描述
Infants with symptomatic hemangiomas will be enrolled. Magnetic resonance imaging will be completed before starting medication if the extent of the hemangioma is not evident on clinical examination alone. Infants will be randomized to receive either propranolol or steroids for 4-6 months. Hemangioma response will be measured and compared monthly as will tolerability of the medications. Additionally, urine specimens will be collected at each visit to determine if markers are present that can predict response to therapy.
Additionally, any hemangiomas that are excised will be examined for genetic markers to aid in predicting response to therapy.
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 2 Weeks 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •infants with symptomatic hemangiomas
排除标准
- •hypertension
- •hypotension
- •hypoglycemia
- •liver failure
- •previous treatment for hemangiomas
研究组 & 干预措施
propranolol for treatment of hemangiomas
Assessing efficacy and tolerability of propranolol in management of symptomatic hemangiomas
干预措施: propranolol (Drug)
Prednisolone
Assessing efficacy and tolerability of prednisolone in management of symptomatic hemangiomas and comparing to propranolol.
干预措施: Prednisolone (Drug)
结局指标
主要结局
Decrease in Size of Hemangioma (Length x Width) in Square mm
时间窗: 4-5 months after initiating therapy
A priori primary outcome was proportional change in the total surface area as measured by lesion's outer margin length x width at baseline minus the same measure at 4 months with surrogate data used at 5 months if 4 months not available.
次要结局
- Tolerability of Medication(enrollment until study close out or withdrawal up to 9 months)
- Number of Serious Adverse Events (SAEs)(enrollment until study close out or withdrawal up to 9 months)
- Growth and Development Adverse Events(enrollment to study withdrawal or close out up to 9 months)
- Pulmonary/Respiratory Adverse Events(enrollment through study close out or withdrawal, up to 9 months)
- Allergy/Immunology Adverse Events(enrollment through study closeout or study withdrawal up to 9 months)
- Dermatologic Adverse Events(enrollment to study close out or withdrawal up to 9 months)
- Endocrinologic Adverse Events(enrollment to close out or study withdrawal up to 9 months)
- Gastrointestinal Adverse Events(enrollment to study withdrawal or study close out up to 9 months)
- Infectious Adverse Events(enrollment to study withdrawal or close out up to 9 months)
- Metabolic or Laboratory AEs(enrollment to study withdrawal or close out up to 9 months)
- Vascular Adverse Events(enrollment to study withdrawal or close out up to 9 months)
- Constitutional Adverse Events(enrollment to study close out or withdrawal up to 9 months)
研究者
Nancy Bauman
Professor, George Washington University, Attending Children;s National
Children's National Research Institute