jRCT2041220051进行中（未招募）不适用
Placebo-controlled comparative study of NPC-12Y oily gel in patients with skin lesions associated with tuberous sclerosis complex
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 40
- 主要终点
- -
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized Controlled Trial
- 干预模型
- Parallel Assignment
- 主要目的
- Treatment Purpose
- 盲法
- Double Blind
入排标准
- 年龄范围
- 3age old over 至 No limit（—）
- 性别
- All
入选标准
- •(1) Male or female patients 3 years old or greater at the time of informed consent
- •(2) Patients who are diagnosed as definite diagnosis according to diagnostic criteria for tuberous sclerosis complex (International Tuberous Sclerosis Complex Consensus Conference 2012)
- •(3) Patients with three or more reddish papules of angiofibroma ( >= 2 mm in diameter) on the face at screening tests
- •(4) Patients who are not suitable for therapy with laser or surgery (including liquid nitrogen therapy and phototherapy) for angiofibroma, or who do not want therapy with laser or surgery
- •(5) Patients who are being treated with Rapalimus gel 0.2% (NPC-12G Gel 0.2%) and who are able to and agree to a withdrawal of at least 4 weeks prior to enrollment in this study.
- •(6) Patients who (or whose guardian) give a written informed consent in understanding and willingness after having received enough explanation regarding the study participation.
排除标准
- •(1) Patients who (or whose guardian) are hard to apply the investigational drug topically with keeping compliance
- •(2) Patients who are hard to be taken pictures of their lesions adequately in such cases that they may not follow instruction of stillness
- •(3) Patients with clinical findings such as erosion, ulcer and eruption on or around the lesion of angiofibroma, which may affect assessment of safety or efficacy
- •(4) Patients with a history or complication of allergy to the component of the investigational drug (sirolimus)
- •(5) Patients who have any infectious disease, cardiac disease, hepatic disease, pulmonary disease, renal disease, hematological disease, or malignant tumor that is considered inappropriate for participation in this clinical study.
- •(6) Patients who have complications such as diseases unsuitable for the trial participation, for examples, uncontrolled diabetes, dyslipidemia, etc.
- •(7) Patients who have participated in other clinical trial or clinical study, and have taken an investigational or clinical study drug within 6 months before the initial registration
- •(8) Patients who used mTOR inhibitors (oral or injectable) other than everolimus within 4 weeks before the initial registration
- •(9) Female patients who are pregnant, may be pregnant, or are lactating
- •(10) Patients who cannot agree to use appropriate contraception after the date of consent to participate in the clinical study and for the duration of the clinical study (including male patients with a partner of childbearing potential)
- •(11) Patients who have received therapy with laser or surgery (including liquid nitrogen therapy and phototherapy) to the lesion of angiofibroma within 6 months before the initial registration
- •(12) Other patients who are considered by the investigator as unsuitable for participation in the clinical study
结局指标
主要结局
-
Improvements in angiofibroma assessed by IRC at 12 weeks after the start of administration
次要结局
- Improvements and improvement rate in angiofibroma assessed by IRC (except the primary outcome)
- Improvements and improvement rate in the color (reddishness) of angiofibroma assessed by IRC
- Improvements and improvement rate in the size of angiofibroma assessed by IRC
- Changes in Index of Facial Angiofibromas (IFA) score over time by investigators
- Changes in shagreen Patch surface and volume over time
- Changes in Ungual fibromas surface over time
- Patient satisfaction
- Incidence of adverse events and adverse drug reactions
- Serious adverse events and adverse drug reactions
- Significant adverse events and adverse drug reactions
- Laboratory findings and vital signs
- Sirolimus / everolimus blood concentration
研究者
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