NCT03243019招募中2 期
Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis
University Hospital, Lille4 个研究点 分布在 1 个国家目标入组 28 人开始时间: 2018年6月25日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 28
- 试验地点
- 4
- 主要终点
- Response rate to rapamycin
研究概览
简要总结
To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period.
The success rate is determined by volume reduction superior to 1/5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case of mucosal involvement.
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
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient from 0 to 18 years of age, presenting with poly-cystic suprahyoid or mediastinal lymphatic.
- •with chronic pain or functional respiratory or swallowing impairment with a CDS score < 8
- •Curative treatment is not possible or associated with a high risk of morbidity ,mortality and functional and cosmetic impairment
- •Karnofsky Score (> 10 years of age) or Lansky score (≤10 years of age) > 50%
- •Neutrophils count≥1.0 x 109/L
- •Platelets count ≥ 100 x 109/L
- •Hemoglobin ≥ 8 g/dL
- •Bilirubin ≤ 1,5 ULN
- •Transaminases < 2,5 ULN
- •Serum albumin ≥ 2 g/dL.
- •LDL cholesterol <160 mg/dL
- •Triglycerides < 150 mg/dL
- •Negative test of pregnancy if relevant
- •Social security affiliation
- •At least 2 months after a previous procedure on the malformation
排除标准
- •Non-respect of inclusion criteria
- •Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period
- •renal failure
- •Liver failure
- •Digestive disease leading to rapamycin malabsorption
- •uncontrolled or severe infectious disease
- •Patients requiring treatment interfering with CYP3A4 isoenzyme (rifampicin, rifabutin, carbamazepine, phenobarbital, phenytoin) or inhibiting CYP3A4 isoenzyme's activity (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin, Diltiazem, Verapamil, nicardipine, clotrimazole, fluconazole , troleandomycin, bromocriptine, cimetidine, danazol, protease inhibitors) -patients requiring treatment by cisapride and metoclopramide
- •Concomitant administration of mTOR inhibitor
- •Peanuts or soya allergy
- •Impossibility to receive informed consent
- •Absence of social security affiliation
- •refusal to sign consent
- •Ongoing pregnancy or breastfeeding
- •refusal to participate
研究组 & 干预措施
SIROLIMUS
Experimental
干预措施: rapamycin (Drug)
SIROLIMUS
Experimental
干预措施: MRI (Device)
SIROLIMUS
Experimental
干预措施: Rapamycin dosage (Biological)
结局指标
主要结局
Response rate to rapamycin
时间窗: At 3 months
Volumetric assessment by MRI. A response is considered as positive if volume decrease is superior to 1/5th of the initial volume.
次要结局
- Kinetic of rapamycin response(At 3, 6 and 12 months)
- Rapamycin side effects(Monthly during 1 years)
- Efficacy of rapamycin on clinical symptoms(At 3, 6 and 12 months)
- Pediatric Quality of Life Inventory (PedsQL 4) Scales(Baseline, at 3, 6 and 12 months)
- Biological response to rapamycin(Baseline and at 6 months)
研究者
研究点 (4)
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