Nadolol Versus Propranolol in Children With Infantile Hemangiomas: a Randomized, Controlled, Double-blinded Trial
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 74
- 试验地点
- 1
- 主要终点
- The change in the bulk (size/extent) and color of the infantile hemangioma (IH)at Week 24 compared to baseline using Visual Analog Scale (VAS).
研究概览
简要总结
The purpose of this study is to assess the efficacy and safety of oral propranolol versus nadolol in patients with Infantile Hemangiomas (IH) in a randomized, controlled, double-blinded study.
详细描述
The study objective is to compare the efficacy and safety of oral propranolol in comparison with oral nadolol in patients with IH. Patients will be randomly assigned to either propranolol or dose equivalent nadolol. The duration of the study will be 24 weeks, however, patient will be monitored for up to 1 year post study enrolment. Both efficacy and safety will be closely monitored and captured.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 1 Month 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •1-6 months corrected age
- •Written parental informed consent
- •At least one of the following:
- •Size: hemangioma >1.5 cm on the face or >3 cm on other body parts
- •Causing or with potential for functional impairment (e.g. amblyogenic IH, ulcerated hemangioma)
- •Causing or with potential for cosmetic disfigurement (e.g. nasal tip, glabella location)
排除标准
- •Contraindications to beta-blockers
- •Hypotension
- •Bradycardia
- •Hypoglycemia
- •Cardiac disease associated with decreased ejection fraction and/or > second degree heart block
- •Bronchospasm (including bronchial asthma)
- •Allergic rhinitis
- •Corrected gestational age less than 1 month at screening
- •Patients with PHACES cerebral arteriopathy at risk of stroke
- •Patients and/or breastfeeding mothers receiving treatment with anti-arrhythmic agents, calcium channel blockers, ACE inhibitors, inotropic agents, vasodilators, hypoglycemic agents, neuroleptics, antiacids, benzodiazepines, thyroxine, warfarin
- •Patients treated with an oral beta-blocker or other agent (e.g. systemic steroids, vincristine) within 2 weeks from randomization
- •Patients treated with topical timolol within 1 week from randomization
- •Vascular tumors other than infantile hemangioma (e.g. pyogenic granuloma, hemangioendothelioma)
研究组 & 干预措施
Nadolol group
40 study participants will take Nadolol (oral liquid suspension)
干预措施: Nadolol (Drug)
Propranolol group
40 study paticipants will take Propranolol (oral liquid suspension)
干预措施: Propranolol (Drug)
结局指标
主要结局
The change in the bulk (size/extent) and color of the infantile hemangioma (IH)at Week 24 compared to baseline using Visual Analog Scale (VAS).
时间窗: 24 weeks
A 100 mm visual analog scale (VAS) will be used to quantify changes in the visible bulk (size/extent) and color of the lesion by comparing clinical photographs at 24 weeks versus baseline
次要结局
- Frequency of observed and reported adverse events(52 weeks)
- Time and dose to reach the 50%, 75% and 100% tumor shrinkage(52 weeks)
- Percent change in the volumetric changes of hemangioma(24 and 52 weeks)
- Percentage of patients with residual changes (telangiectasias, discoloration, fibro-fatty changes, anetoderma)(52 weeks)
- Percent change in IH bulk using VAS at 4, 12, 52 weeks(4, 12, 52 weeks)
- Inter-rater reliability of the VAS scores(52 weeks)
- Percentage of patients achieving functional correction at Week 4, 12, 24, 52(4,12,24,52 weeks)
研究者
Elena Pope
Section Head, Dermatology
The Hospital for Sick Children