Efficacy, Safety, and Pharmacokinetics of Timolol in Infants With Infantile Hemangioma (IH)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 105
- 试验地点
- 12
- 主要终点
- Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm and Compared With Untreated Controls
研究概览
简要总结
The purpose of this study is to assess the safety and efficacy of Timolol 0.25% and 0.5% doses.
详细描述
Primary: Describe the efficacy of 0.25% and 0.5% topical timolol maleate Gel-forming solution (GFS) as assessed through Infantile Hemangioma (IH) changes in volume.
Secondary: Describe the safety of topical timolol maleate GFS for treatment of IH.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- — 至 84 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
0.25% Timolol Treatment
Subjects assigned to this arm will be randomized to 0.25% timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.25% timolol they will be changed to 0.5% timolol.
干预措施: 0.25% Timolol Maleate Gel Forming Solution (Drug)
0.5% Timolol Treatment
Subjects assigned to this arm will be randomized to 0.5% timolol for 180 days. If during the 180 days the subject is considered a treatment failure, the subject will be unblinded. If the subject is on 0.5% timolol the treating physician will decide to either continue 0.5% timolol or withdraw the subject and begin an alternative treatment.
干预措施: 0.5% Timolol Maleate Gel Forming Solution (Drug)
结局指标
主要结局
Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm and Compared With Untreated Controls
时间窗: 180 days
The VAS-volume is a 100 mm scale used to independently grade hemangioma volume. -100 indicates hemangioma has doubled in size, 0 indicates no change, and +100 indicates complete shrinkage. Partial response is defined as \>20% and up to 80% reduction in volumetric size of hemangioma.
次要结局
- Number of Participants Who Reach Partial Response, Assessed by Hemangioma Color(180 days)
- Number of Participants With Partial Response in Hemangioma Color as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm and Compared With Untreated Controls(180 days)
- Number of Participants Who Reach Partial Response, Assessed by Volume(30 days, 60 days, 120 days, 180 days)
- Number of Participants With Partial Response of Hemangioma Volume as Measured by VAS (Visual Analog Scale) Within Each Treatment Arm(180 days)
- Comparison of Partial Response of Hemangioma Color From Baseline to 180 Days, Within Each Treatment Arm(180 days)
- Change in Hemangioma Dynamic Complication Scale (HDCS)(baseline, day 180)
- Number of Serious Adverse Events and Adverse Events of Special Interest in Infants Treated With Topical Timolol Maleate(up to 270 days)
- Change in Hemangioma Quality of Life (IH-QoL) Assessment for Infants(baseline, day 180)
研究者
Kanecia Obie Zimmerman
Principal Investigator
Duke University