NCT04461886终止3 期
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
Nobelpharma4 个研究点 分布在 1 个国家目标入组 100 人开始时间: 2020年7月8日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 100
- 试验地点
- 4
- 主要终点
- Discontinuation rate associated with adverse events (Kaplan-Meier method)
研究概览
简要总结
The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel to patients with neurofibromatosis type I.
详细描述
A Phase 3, open-label, uncontrolled, multicenter study to evaluate the safety and efficacy of long-term treatment with NPC-12G gel for patients with neurofibromatosis type I. Patients who meet all entry criteria for this trial apply NPC-12G gel twice a day for 52 weeks or more. Approximately 100 eligible patients will be enrolled.
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 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed as neurofibromatosis type 1 based on the clinical diagnostic criteria in guideline of Japanese Dermatological Association
- •Patients participated in a Phase II/III investigator-initiated clinical trial for neurofibromatosis type I (OSD-001-004) who wish to continue treatment of the investigational drug
- •At the time of enrollment, patients who are able to choose ten evaluable target lesions for efficacy (at least 5)..
- •Patients who have evaluable skin neurofibromas at baseline.
- •Males and females who are 3 years old or elder at the time of informed consent.
- •Patients who (or whose guardian) give a written informed consent in understanding and willingness after having received enough explanation regarding the study participation.
- •Patients whose use or continued use of the investigational drug is judged to be reasonable by the principal investigator or sub-investigator.
排除标准
- •Patients who occurred serious adverse effects, who discontinued the sudy due to adverse effects, or who discontinued the trial due to withdrew of their consent in the Phase II/III investigator-initiated study of neurofibromatosis type I (OSD-001-004)
- •Patients with abnormal findings (e.g., pneumonia) in the results of chest x-ray at the time of screening
- •Patients with creatinine clearance of less than 50 mL/min
- •Patients with poorly controlled dyslipidemia (serum triglycerides >500 mg/dL or LDL cholesterol >190 mg/dL even with treatment)
- •Patients who have complications such as infection, cardiac, hepatic, pulmonary, renal, or hematological diseases and malignant tumors that are considered unsuitable for participation in this trial.
- •Patients with alcohol sensitivity or allergy to an ingredient of the study drug (Sirolimus)
- •Female patients who are pregnant, may be pregnant, or are lactating
- •Patients (including male patients with a fertile partner) who cannot consent to use adequate contraception from the date of consent to 12 weeks after the end of treatment
- •Patients who participated in any other clinical trial or clinical study, other than the OSD-001-004 study, and have taken an investigational or investigational drug within 6 months prior to the date of the consent
- •Patients who are participating in an observational study during this trial
- •Patients who are considered by the investigator as unsuitable for participation in this trial
研究组 & 干预措施
NPC-12G gel
Experimental
NPC-12G gel is containing 0.2% Sirolimus
干预措施: NPC-12G gel (Drug)
结局指标
主要结局
Discontinuation rate associated with adverse events (Kaplan-Meier method)
时间窗: 52 weeks
Number of Adverse events leading to discontinuation
次要结局
未报告次要终点
研究者
研究点 (4)
Loading locations...
相似试验
进行中（未招募）
3 期
A long-term study of NPC-12G gel in neurofibromatosis type IJPRN-jRCT2051200032Kaneda Mari100
已完成
3 期
Phase III Trial of Topical Formulation of Sirolimus to Skin Lesions in Patients With Tuberous Sclerosis Complex (TSC)Tuberous SclerosisHypomelanotic MaculePlaqueAngiofibromaNCT02635789Nobelpharma62
已完成
3 期
Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis ComplexTuberous SclerosisHypomelanotic MaculePlaqueAngiofibromaNCT02634931Nobelpharma94
已完成
3 期
Efficacy and Safety, Long-Term Study of NPC-01 to Treat Dysmenorrhea Associated With EndometriosisDysmenorrheaNCT00902746Nobelpharma149
已完成
3 期
Clinical Study of NPC-12Y Gel in Patients With Skin Lesions Associated With TSCTuberous Sclerosis ComplexNCT05495425Nobelpharma43