A Phase 2/3, Multi-Center, Double-Blind, Placebo-Controlled, Randomized, Parallel-Group, Dose-Response Comparison of the Efficacy and Safety of a Topical Rapamycin Cream for the Treatment of Facial Angiofibromas (FA) Associated With Tuberous Sclerosis Complex (TSC) in Patients 6 Years of Age and Over
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 107
- 试验地点
- 17
- 主要终点
- Percentage of Participants Obtaining Successful Treatment
研究概览
简要总结
The study aims to compare the safety and efficacy of two different strengths of Rapamycin cream, topical and placebo over 26 weeks in the treatment of facial angiofibroma (FA) associated with Tuberous Sclerosis Complex (TSC).
详细描述
Topical rapamycin has previously been used to treat FA associated with TSC, reducing erythema, papule size, while flattening lesions and improving skin texture. Topical rapamycin has been reported to be well tolerated.
The efficacy and safety of a topical rapamycin cream at two strengths (0.5% and 1.0%) will be assessed during a 26 week double-blind treatment phase with assessments made at clinical visits at baseline, 2, 8, 14, 20 and 26 weeks, and at follow-up (4 weeks after the last dose of study drug).
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
- 6 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients aged ≥ 6 years and ≤ 65 years on the day informed consent is obtained
- •Patients diagnosed with TSC based on the clinical diagnostic criteria of International Tuberous Sclerosis Complex Consensus Conference 2012 and presenting visible facial angiofibroma
- •An FA severity score of 2 or 3 on the IGA scale
- •Patients or their legal representatives capable of understanding the explanation of the clinical trial and who give written informed consent for participation
- •Patients or their legal representatives able to maintain patient diaries following the instructions of the investigator or sub-investigator
排除标准
- •Patients who cannot carry out the treatment plan or follow-up assessment
- •Patients with serious skin lesions such as erosions or ulcers
- •Patients with known hypersensitivity to any component of the study product
- •Patients who have received rapamycin/sirolimus, everolimus, or temsirolimus within 3 months of enrolment
- •Patients who received laser therapy or surgical therapy within 6 months prior to trial enrolment
- •Patients who participated in any other clinical trial within 3 months prior to the day of enrolment
- •Patients judged unsuitable for this clinical trial by the investigator or sub-investigator
- •Pregnant or lactating females
- •Sexually active females of childbearing potential not using adequate contraception and sexually active males not using adequate contraception
- •Patients with immune dysfunction or receiving any form of immunosuppression
- •Patients with severe FA, with a score of 4 on the IGA scale
- •Patients with an FA severity score of less than 2 on the IGA scale
研究组 & 干预措施
0.5% Rapamycin cream, topical
Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
干预措施: rapamycin (Drug)
1.0% Rapamycin cream, topical
Rapamycin cream topical, 0.5% w/w, applied once daily before bed on affected area for 26 weeks
干预措施: rapamycin (Drug)
Placebo
Placebo cream topical, applied once daily before bed on affected area for 26 weeks
干预措施: placebo (Drug)
结局指标
主要结局
Percentage of Participants Obtaining Successful Treatment
时间窗: After 26 weeks treatment
Success on the Investigator Global Assessment (IGA) scale is defined as clear or almost clear with an improvement of at least two grades from baseline. IGA scores range from 0-4: 0=Clear 1. Almost Clear 2. Mild 3. Moderate 4. Severe
次要结局
- Subjective (Participant or Parent/Caregiver) Percentage Change Rating Scale(After 26 weeks treatment)
- Time to Treatment Success(From first dose to 26 weeks (± 2 weeks))
- Change From Baseline in Investigator's Global Assessment(At baseline and after 26 weeks treatment)
- Change From Baseline in Facial Angiofibroma Severity Index (FASI)(At baseline and after 26 weeks treatment)
- Objective (Clinician) Percentage Change Rating Scale(After 26 weeks treatment)
- Categorical Change in Facial Angiofibroma(After 26 weeks treatment)