Efficacy and Safety of Topical Timolol Maleate 0.5% Solution for Superficial Infantile Hemangioma in Early Proliferative Phase. A Randomized Clinical Trial
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 70
- 试验地点
- 2
- 主要终点
- Complete or almost complete resolution of the lesions
研究概览
简要总结
Infantile hemangioma (IH) is the most common benign vascular tumor in pediatric population. Oral propranolol is the treatment of choice for complicated hemangiomas. Topical timolol, a non-selective beta-blocker, is an emerging treatment which has been reported to be effective and safe for the treatment of IH, especially for superficial hemangiomas. Investigators hypothesize that treatment with topical timolol in the first two months of life, before the proliferative phase or in early proliferative phase, may prevent from further growing and the need to treat with oral propranolol.
详细描述
A multicentric, randomized, double-blind, placebo-controlled, phase II clinical trial to evaluate the efficacy and safety of 0.5% timolol maleate solution for the early treatment of infantile hemangioma.
Patients with less of 60 days of life with focal or segmental hemangiomas, superficial, mixed or abortive will be treated with topical timolol 0.5% twice a day for 24 weeks.
Changes in lesion size, color and thickness will be evaluated from photographs taken at 2,4,8,12,24 and 36 weeks. Vital signs and side effects will be recorded at each visit. Response to treatment will be evaluated by a blinded investigator in a semiquantitative scale (complete resolution, improve, stabilization, worsening).
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
- 10 Days 至 60 Days（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent for study participation and the use of the patient's photographs from the patient's parent(s) or guardian(s),
- •The patient is between 10 and 60 days of age at the time of enrollment,
- •The patient should have:
- •at least one focal or segmental hemangioma, mixed or superficial, present anywhere on the body of at least 0.3 cm of minimum diameter and not greater of 5 cm.
- •abortive or minimal growth hemangioma
- •Infantile hemangioma precursors
排除标准
- •Patients > 60 days of age
- •Complicated hemangiomas (life-threatening, function-threatening, or ulcerated)
- •Children who have previously received systemic, intra-lesional or topical corticosteroids, imiquimod, vincristine, alpha-interferon, propranolol or other beta blockers, PDL laser.
- •Patients with PHACES, LUMBAR, SACRAL or PELVIS syndrome.
- •Internal involvement hemangiomas (liver, GI tract, larynx)
- •Children with a personal history of asthma or cardiac conditions that may predispose to heart block.
- •Children with congenital birth defects
- •Children with Cancer (leukemia, sarcoma, neuroblastoma, retinoblastoma)
- •Children with a history of hypersensitivity to beta blockers or excipients.
研究组 & 干预措施
Timolol
Timolol maleate 0.5% topical solution; 50% of the patients treated (randomized)
干预措施: Timolol (Drug)
Placebo
Saline topical solution; 50% of the patients treated (randomized)
干预措施: Placebo (Drug)
结局指标
主要结局
Complete or almost complete resolution of the lesions
时间窗: 24 weeks
Efficacy to treatment at 24 weeks, categorized as complete or almost complete resolution of the lesions (almost complete resolution defined as faint macular erythema, telangiectasia and no palpable component). This will be generated by independent blind evaluations by comparing photographs at baseline and 24 weeks. The investigators will score this improvement into one of the following categories (complete resolution, improve, stabilization, worsening)
次要结局
- Adverse reactions(24 weeks)