Onreltea (Brimonidine) Gel In Pediatric Patients With Capillary Malformations: A Prospective, Open-label, Cohort Study
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- The change in the color of the capillary malformation using Chroma meter values (Δa, ΔE) at 12 weeks.
研究概览
简要总结
Capillary Malformations (CM) affect a significant proportion of otherwise healthy children and may lead to psychological discomfort if left untreated. A significant proportion of untreated lesions undergo soft tissue thickening and darker discoloration later in life due to progressive ectasia of the affected vessels. While laser treatment is available, its use may be limited due to need for repeated sedation/general anesthetic use, partial response and cost.
The investigators propose to conduct an open-label, prospective, cohort study using Onreltea ( Brimonidine) gel for treatment of facial capillary malformations in children. The study medication will be applied topically on affected area of the skin daily for 12 weeks. Follow up visits will occur at at Week 1,4,8,12, and 16 to assess the efficacy and safety of the proposed treatment.
The study second aim is to explore the feasibility of conducting a multicenter placebo controlled study.
详细描述
The investigators are planning to enroll in the study 20 participants at SickKids.
It is a prospective, open label, cohort study. Patients enrolled in the study will be followed at the Hospital For Sick Children for 16 weeks. They will come for the study visits 6 times: in 1 week, 4,8,12, and 16 weeks after the treatment has been started. During each study visit the study investigators will assess any changes in the characteristics of CM lesion(s) captured by a Chromometer *, Analogue Scale and Erythema Assessment tools. Participants or their parents will assess the changes at the final study visit (VAS and EA tools).
Patients will be provided with study medication for all duration of the study treatment (12 weeks).
The results of the treatment will be compared with the baseline data to evaluate the efficacy and safety of Onreltea (Brimonidine) gel in children with facial capillary malformations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •diagnosis of facial capillary malformation (port-wine stain, PWS) made by a dermatologist
- •age: 12-17 years of age
- •weight > 45 kg
- •lesions with a surface area < 100 cm2
- •signed consent and assent for study participation
排除标准
- •skin breakdown overlying the malformation due to other dermatological conditions (e.g. eczema, psoriasis)
- •current or treatment with laser the past 3 months
- •other topical treatments used within the past 4 weeks (e.g. rapamycin, corticosteroids, calcineurin inhibitors, etc)
- •known chronic renal or hepatic disorders
- •known cardiovascular disorders
- •other systemic medications that potentially interact with Brimonidine (opiates, chlorpromazine, methyphenidate, reserpine, etc)
- •mixed capillary/ venous or lymphatic malformations
- •known allergy to one of the constituents of Onreltea
- •pregnancy, or sexually active subjects of child-bearing potential (CBP), unwilling to use contraception during the study (such as barrier method, or oral contraceptives).
研究组 & 干预措施
Onreltea ( Brimonidine)
All the patients enrolled in the study will apply Onreltea ( Brimonidine 0.33%) gel on the affected skin area for 12 weeks (from Day 0 to Week 12 visit).
干预措施: Brimonidine 0.33% gel (Drug)
结局指标
主要结局
The change in the color of the capillary malformation using Chroma meter values (Δa, ΔE) at 12 weeks.
时间窗: 12 weeks
Measurement of erythema will be performed using Chroma Meter, CR-400, Konica, Minolta, Osaka, Japan. The meter readings will result in 3 values: L- refers to the relative light intensity, ranging from 0 (black) to +100 (white); a-captures color saturation, ranging from +60 (green) to -60 (red) and b- captures color spectrum from +60 (blue) to -60 (yellow). In most studies both, changes in a (Δa) and overall changes in the composite score (ΔE calculated as √((ΔL\*before-ΔL\*after)\^2+(Δa\*before-Δa\*after)\^2+(Δb\*before-ΔL\*after)\^2 ) are obtained.
次要结局
- Changes in CEA scores at 12, 16 weeks compared to baseline(12 and 16 weeks)
- Correlation between iVAS, pVAS, CEA, pEA and Chroma Meter values(1,4,8,12,16 weeks)
- Percentage of patients achieving 75% and 100% resolution of the lesion(12 weeks)
- Frequency of observed and reported adverse events (AE)(16 weeks)
- Changes in the color of the lesion (Δa, ΔE) at each follow up visit including the last visit at 16 weeks compared to baseline(1,4,8,16 weeks)
- Changes in the iVAS at 12 and 16 weeks compared to baseline(12 and 16 weeks)
研究者
Elena Pope
Professor of Paediatrics, University of Toronto Fellowship Director and Section Head, Paediatric Dermatology.
The Hospital for Sick Children