EUCTR2014-000484-41-GB进行中（未招募）1 期
A Non-Randomised, Open Label, Pilot Trial of Sirolimus Therapy for Segmental Overgrowth Due to PIK3CA Related Overgrowth - Sirolimus Treatment for PIK3CA Related Overgrowth (PROMISE)
Cambridge University Hospitals NHS Foundation Trust and the University of Cambridge0 个研究点目标入组 11 人开始时间: 2016年3月31日最近更新:
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 11
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Have given, or their guardian has given written informed consent to participate
- •Aged: 3-65 years inclusive
- •Male or female
- •Post-zygotic PIK3CA mutation
- •Clinically stable in the opinion of the investigator
- •Measurably progressive overgrowth, in current progression or with clinical history of overgrowth progression
- •Have undergone an MRI and/or DXA scan in the previous 6 months as part of the Investigation of Segmental Overgrowth Disorders study
- •All women of childbearing potential and all sexually active male participants must agree to use effective contraception
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 5
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 5
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •The participant may not enter the study if ANY of the following apply:
- •Aged 2 years or younger or aged 66 years or older
- •Pregnant or breastfeeding
- •Male and Female participants of childbearing potential who are not using an effective method of contraception (during treatment and up to 12 weeks after sirolimus discontinuation)
- •Hypersensitivity to sirolimus or any of the excipients
- •Any current medical disorder or medication likely to impair ability to follow the study protocol safely and effectively
- •Incapacity to give informed consent
- •Sirolimus treatment in the prior 4 weeks
- •Personal history of malignancy or ongoing investigations for malignancy
- •Active skin infections requiring antibiotics or anti-viral medication
- •HCV/HBV/HIV seropositivity
- •Previous/ active MTB infection
- •Active Pneumonitis
- •Inability to attend study visits
- •If less than 3 months post-surgery
- •Inadequate bone marrow function defined as:
- •- Peripheral absolute neutrophil count (ANC) < 1.0 x10^9/l
- •- Platelet count < 100 x10^9/l
- •- Haemoglobin <10.0 g/dL
- •Inadequate renal function defined as:
- •A serum creatinine based on age as follows:
- •Age (years)Maximum Serum Creatinine (µmol/l)
- •6 11 >16132
- •OR a creatinine clearance or radioisotope GFR < 70ml/min/1.73 m2
- •Inadequate liver function defined as:
- •- Bilirubin (sum of conjugated + unconjugated) > 1.5 x upper limit of normal
- •(ULN) for age, and
- •- SGPT (ALT) > 5 x upper limit of normal (ULN) for age, and
- •- Serum albumin > 30 g/dL.
- •Inadequate Fasting LDL cholesterol > 4.2 mmol/l
研究者
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