Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex and Neurofibromatosis 1
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 52
- 试验地点
- 1
- 主要终点
- Rapamycin level
研究概览
简要总结
This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1). Subjects will apply either a Polyvinylidene fluoride (PVDF) coating (Skincerity) containing rapamycin or the PVDF coating alone nightly to fibromatous lesions for a duration of six months.
The primary goal of this study is to evaluate the safety of the topical product in patients with TSC and NF1. The secondary goal of this study is to evaluate the effectiveness of the topical product for treatment of cutaneous fibromatous lesions.
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
- 13 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Subject is willing and able to comply with all trial requirements
- •Subject is male or female and over 13 years of age
- •Subject has a diagnosis of either TSC or NF1 and has visible fibromatous lesions (angiofibromas or neurofibromas)
- •Female subjects of child-bearing potential must not be pregnant and must agree to use appropriate contraceptive methods for the duration of the trial
排除标准
- •Subject is currently receiving therapy with rapamycin or sirolimus
- •Subject is receiving any form of immunosuppression or has previously experienced immune dysfunction
- •Subject is currently participating in or has participated within the last 30 days in any clinical trial involving an investigational drug
- •Subject has a known hypersensitivity to either the PVDF coating (Skincerity®) or rapamycin
- •Subject is a pregnant or nursing female
研究组 & 干预措施
TSC 1% Arm
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to facial angiofibromas
干预措施: Skincerity plus sirolimus/rapamycin (Drug)
TSC Placebo Arm
TSC subjects will apply a study product containing polyvinylidene fluoride coating alone to facial angiofibromas
干预措施: Skincerity (Drug)
TSC 5% Arm
TSC subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to facial angiofibromas
干预措施: Skinercity plus sirolimus/rapamycin (Drug)
NF1 Placebo Arm
NF1 subjects will apply a study product containing polyvinylidene fluoride coating alone to cutaneous neurofibromas
干预措施: Skincerity (Drug)
NF1 1% Arm
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 1 mg of sirolimus/rapamycin to cutaneous neurofibromas
干预措施: Skincerity plus sirolimus/rapamycin (Drug)
NF1 5% Arm
NF1 subjects will apply a study product containing polyvinylidene fluoride coating plus 5 mg of sirolimus/rapamycin to cutaneous neurofibromas
干预措施: Skinercity plus sirolimus/rapamycin (Drug)
结局指标
主要结局
Rapamycin level
时间窗: 6 months
Complete blood count
时间窗: 6 months
Total cholesterol
时间窗: 6 months
Dermatologic sensitivity at site of application (pain, erythema, edema, pruritis)
时间窗: 6 months
次要结局
- Reduction in lesion size and appearance(6 months)
研究者
Mary Kay Koenig
Assistant Professor, Pediatrics-Neurology
The University of Texas Health Science Center, Houston