A Pilot Study Using Topical Statins to Treat Children With Infantile Hemangioma
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- Joyce Teng
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Number of Participants with Treatment-Related Adverse Events
研究概览
简要总结
This is a 48-week, open-label pilot study to evaluate the safety, including systemic exposure and skin tolerability, and preliminary efficacy of 5% simvastatin ointment in the treatment of 15 children with newly diagnosed superficial proliferating IH.
The primary objective:
To evaluate the safety, including systemic exposure and skin tolerability of topical treatment with 5% simvastatin ointment for superficial proliferating IH over 48 weeks.
The secondary objective:
1.1 To evaluate the efficacy and durability of 5% simvastatin ointment when topical treatment is administered twice daily for 24 weeks, followed by a 24-week post-treatment follow-up period. Evaluation is performed at each clinic visit via investigator global assessment (IGA) based on standardized 3D digital photography and hemangioma activity score (HAS).
1.2 To evaluate the impact of 5% simvastatin ointment on quality of life using the IH-QoL questionnaire.
The exploratory objective:
To explore whether clinical response and regrowth following topical 5% simvastatin treatment are associated with SOX18-mevalonate pathway-axis activation in infantile hemangioma tissue.
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
- 3 Months 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants may be included in the study only if they meet all of the following criteria:
- •Healthy children aged between 3-6 months without medical disorders that contradict topical statin treatment.
- •Infants with newly diagnosed superficial IH. [3] Participants must possess at least one IH lesion with the longest diameter equal to or greater than 1 cm but less than 1% BSA, located on any part of the body except the lips.
- •[4] Written informed consent from the parent(s)/guardian(s) must be obtained before any study procedure is performed.
- •[5] Parent(s)/guardian(s) are willing to comply with the study protocol
排除标准
- •Participants meeting any of the following criteria will not be eligible to participate in the study:
- •IH is primarily characterized as subcutaneous, and deep, with minimal cutaneous involvement for evaluation.
- •IH with active ulceration at screening visit.
- •IH to be treated involving the lips mainly.
- •IH with high-risk criteria that need systemic propranolol treatment to avoid the delay with standard treatment
- •Participants with concurrent skin conditions that may impede accurate clinical assessment of the IH.
- •Participants with hereditary or metabolic disorders requiring systemic statin therapy.
- •Participants who are allergic to statins, or other ingredients present in the topical medication.
- •Participants who have received any of the following treatments for their IH:
- •i) Topical medical therapy, i.e. imiquimod, sirolimus, timolol, intermediate or high strength steroids, etc. within the past 4 weeks ii) Systemic medical therapy, i.e. beta blockers, steroids, sirolimus for IH within the past 3 months iii) Surgical intervention including laser treatment within the past 6 weeks
研究组 & 干预措施
5% simvastatin ointment
Participants will be applied 5% simvastatin ointment on IH lesion
干预措施: 5% simvastatin ointment (Drug)
结局指标
主要结局
Number of Participants with Treatment-Related Adverse Events
时间窗: Baseline through week 24
Evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks
时间窗: Baseline through week 48
The primary endpoint is the evaluation of the safety and tolerability of the 5% simvastatin ointment over 48 weeks, defined by the following three combined components: * Adverse Events: The incidence, severity, and proportion of participants experiencing adverse events (AEs) from Week 0 through Week 48. * Systemic Exposure: The proportion of participants with detectable serum simvastatin levels, alongside descriptive statistics of measured concentrations, at Week 24 (End of Treatment). * Skin Tolerability: The incidence and maximum severity score of local skin reactions at the treatment site at Week 24 (End of Treatment).
次要结局
- Percentage of participants achieved a 75% HAS reduction from baseline by the end of the study (week 24) compared to baseline.(Baseline through week 24)
- Percentage of participants with a significant improvement in quality-of-life, defined as > 50% reduction in the IH-QoL questionnaire score from baseline to week 24.(Baseline through week 24)
- Percentage of participants achieved a 50% HAS reduction at week 12 compared to baseline(Baseline through week 12)
- Percentage of targeted hemangiomas with complete or nearly complete resolution (IGA 0 or 1), defined as a minimal degree of telangiectasia, skin thickening, and no definitive palpable cutaneous texture changes.(Baseline through week 24)
- Percentage of targeted hemangiomas that are stabilized (no noticeable change to baseline) or continue to progress.(Baseline through week 24)
- To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Key Efficacy Endpoints (Week 24)(Baseline through week 24)
- To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Treatment Failure and Durability Endpoints(Baseline through week 48)
- To evaluation of the efficacy and durability of the 5% simvastatin ointment over 48 weeks: Time-to-Event Assessments(Baseline through week 48)
研究者
Joyce Teng
Professor of Dermatology and Pediatric
Stanford University