2024-515955-39-01已完成2 期
TOPical sirolimus in linGUal microkystic lymphatic malformation
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- The primary outcome will consist in the evaluation of global severity of the LMLM using PGA (Physical Global Assessment) 0 to 5 score, by three independent blinded experts, on monthly standardized photographs. A 1-point improvement versus baseline in PGA scale would already have a clinical relevance. Our primary analysis will focus on change in PGA after topical application of Sirolimus for 12 weeks
研究概览
简要总结
To evaluate the efficacy and safety of a 1mg/mL sirolimus solution applied once daily on the anterior part of lingual microcystic lymphatic malformation of any stage in children and adults after 4, 8, 12, 16, 20 and 24 weeks of treatment as compared to usual care (no treatment).
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •≥ 5 years of age
- •Lingual microcystic lymphatic malformation of any stage (Wiegand 2009) assessed by clinical examination and head-and-neck MRI imaging prior to study enrolment, with or without underlying syndromic malformation (CLAPO for instance)
- •Participants covered by or having the rights to social security
- •Written informed consent obtained from participant and participant’s legal representative if participant is under 18
- •Ability for participant to comply with the requirements of the study
- •Compliance with the French mandatory immunization program
排除标准
- •Patients with a lymphatic malformation requiring a continued background therapy (involving deep organs)
- •Known allergy to one of the components of the sirolimus solution
- •Soy bean or Peanut allergy
- •Pregnant or breastfeeding women
- •Women of child-bearing potential (including teenagers) not using a reliable contraceptive method until the end of the study and three month after the end of the study or sirolimus discontinuation
- •Already involved in another therapeutic trial
- •Secondary lymphatic malformations (lymphangiectasia post-radiotherapy, etc)
- •Previous treatment with systemic or topical mTOR inhibitors within 6 months before inclusion (half-life of oral sirolimus is 60 hours in adults according to Rapamune ® Summary of Product Characteristics)
- •Previous treatment with oral or topical steroids within 10 days before inclusion (half-life of corticosteroids is 12-36 hours)
- •Immunosuppression (immunosuppressive disease or immunosuppressive treatment)
- •Ongoing neoplasia
- •Active chronic infectious disease (HBV, HCV, HIV, etc)
- •Local necrosis
- •Local fungal, viral (HSV, VZV, etc) or bacterial infection on the site of the LMLM (based on clinical examination)
结局指标
主要结局
The primary outcome will consist in the evaluation of global severity of the LMLM using PGA (Physical Global Assessment) 0 to 5 score, by three independent blinded experts, on monthly standardized photographs. A 1-point improvement versus baseline in PGA scale would already have a clinical relevance. Our primary analysis will focus on change in PGA after topical application of Sirolimus for 12 weeks
The primary outcome will consist in the evaluation of global severity of the LMLM using PGA (Physical Global Assessment) 0 to 5 score, by three independent blinded experts, on monthly standardized photographs. A 1-point improvement versus baseline in PGA scale would already have a clinical relevance. Our primary analysis will focus on change in PGA after topical application of Sirolimus for 12 weeks
次要结局
- Time to obtain optimal results
- Global Quality of life assessment (DLQI or children’s DLQI for minors aged 5 to 16), at baseline, time of switch to treatment and W24
- Investigator-assessed PGA at weeks 0, 4, 8, 12, 16, 20 and 24
- Assessment by the patient regarding severity of oozing, bleeding, sialorrhea, eating impairment, taste modification, aesthetic impairment, pain and global discomfort, each using a numeric scale from 0 to 10 (0: clear, 10: very severe), at weeks 0, 4, 8, 12, 16, 20 and 24
- Global evolution compared to baseline, assessed by the patient from -10 to 10 (-10 = severe worsening, 0 = no change, 10 = complete recovery), at weeks 4, 8, 12, 16, 20 and 24
- Measurement of the lesion (length, width, thickness) by the investigator, at baseline, time of switch to treatment and W24
- Safety: Assessment of tolerance of topical sirolimus: record of local adverse events at each visit, before and after the patient has crossed over to the intervention
- Safety : record of general adverse events at each visit, before and after the patient has crossed over to the intervention, physical examination, systolic and diastolic blood pressure measurement
- Safety: Assessment of sirolimus blood passage by measuring residual sirolimus blood concentration: after 4 weeks of treatment, then 8 weeks, then every 8 weeks until W24
- Safety: Evaluation of biological safety at weeks 8, 16 and 24 of exposure compared to baseline (we will perform biological measurements that are required for assessing safety of oral sirolimus: complete blood count, liver (ASAT, ALAT, GGT) and renal (serum creatinine) functions, lipids [cholesterol i.e. total cholesterol, HDL and LDL calculation according to Friedwald's formula and triglycerides] and glycaemia)
研究者
Pr Annabel MARUANI
Scientific
Centre Hospitalier Regional Universitaire De Tours
研究点 (3)
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