Phase II Study of Topical Rapamycin to Erase Angiofibromas in TSC-Multicenter Evaluation of a Novel Therapy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 179
- 试验地点
- 10
- 主要终点
- Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
研究概览
简要总结
The study is a multi-center prospective, randomized, double-blind, placebo-controlled evaluation of the safety and efficacy of a topically applied formulation of rapamycin to cutaneous angiofibromas in subjects with Tuberous Sclerosis Complex (TSC). Subjects will apply either the topical vehicle containing rapamycin or the topical vehicle alone nightly to their angiofibromas for six months.
The primary goal of this study is to evaluate the efficacy of the topical medication for reduction of cutaneous angiofibromas in patients with TSC. The secondary goal of this study is to confirm the safety of the topical medication.
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
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects must be willing and able to comply with all trial requirements.
- •Subject has a diagnosis of TSC and has visible facial angiofibromas.
- •Female subjects of child bearing potential must not be pregnant and must agree to use appropriate contraceptive methods .
排除标准
- •Subject is currently receiving therapy with Rapamycin.
- •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 a clinical trial involving an investigational drug.
- •Subject has a known hypersensitivity to either the vehicle or Rapamycin.
- •Subject is a pregnant or nursing female.
- •Subject has other dermatologic conditions that would preclude or prevent adequate assessment of changes to their facial angiofibromas.
- •Subject has had laser surgery, cryotherapy, or other dermatologic treatment to their facial angiofibromas within the previous 6 months.
研究组 & 干预措施
Placebo
Cream only
干预措施: Placebo (Drug)
0.1 % Rapamycin
0.1% Rapamycin cream
干预措施: Rapamycin (Drug)
1% Rapamycin
1% Rapamycin cream
干预措施: Rapamycin (Drug)
结局指标
主要结局
Change From Baseline in the Facial Angiofibroma Grading Scale (AGS) Score
时间窗: baseline, 6 months
Subject's lesion/s will be digitally photographed before initial application (visit 1), at each study visit (visits 2 through 6), and immediately prior to study termination (visit 7). Following completion of the study, all photographs will be evaluated by two independent dermatologists blinded to both the treatment arm and the stage of treatment. The dermatologists will assess each photograph's appearance using the facial Angiofibroma Grading Scale (AGS), which assesses the forehead, nose, cheeks, and chin for erythema, average lesion size, lesion density, and percent involvement, as well as presence of any pedunculated angiofibromas. A total score is reported with a range of 0 to 202, with higher scores representing a worse outcome.
次要结局
- Photo Readers' Rating (Better, Same, or Worse) of Paired Baseline and End-of-trial (EOT) Photographs for Each Patient(baseline, 6 months)
- Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Dermatology Life Quality Index (DLQI)(baseline, 6 months)
- Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Children's Dermatology Life Quality Index (CDLQI)(baseline, 6 months)
- Change in Quality of Life (QOL) Questionnaire Scores as Assessed by the Family Dermatology Life Quality Index (FDLQI)(baseline, 6 months)
研究者
Mary Kay Koenig
Associate Professor
The University of Texas Health Science Center, Houston