Pharmacokinetics and Safety of Treatment With Paracetamol in Children and Adults With Spinal Muscular Atrophy and Cerebral Palsy
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 48
- 试验地点
- 1
- 主要终点
- Clearance paracetamol
研究概览
简要总结
To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.
详细描述
-
Aim
-
Objectives
Primary objective:
The aim of the project is to study the pharmacokinetics of therapeutic doses of paracetamol with specific emphasis on the contributions of the metabolites (glucuronide, sulphate, cysteine and mercapturate), in patients with spinal muscular atrophy(SMA), patients with cerebral palsy(CP), and patients with SMA or CP admitted to the intensive care unit (ICU), in comparison with healthy controls.
Secondary objective:
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 45 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •- Patients: Men, women and children diagnosed with/biochemically verified SMA and CP
- •Patients admitted to the ICU: Men, women, children diagnosed with/biochemically verified SMA and CP
- •Healthy controls: Need to be healthy, evaluated by the investigator.
- •Children: 6-18 years
- •Adult patients: 18-45 years
- •Healthy controls: 18-45 years
- •ICU-admitted patients: 6-45 years
- •Signed informed consent to participation in the trial
排除标准
- •- Inability to understand the purpose of the trial or cooperate in the conduction of the experiments.
- •o For the children this will concern of course the parents or the guardians of the child.
- •Competing conditions at risk for compromising the results of the study.
- •Participation in other trials that may interfere with the results.
- •Intake of medications that may interfere with the results, evaluated by investigator.
- •Pregnancy and breastfeeding.
- •BMI >30*
- •In morbidly obese patients, the median area under the plasma concentration-time curve from 0 to 8 h. (AUC0-8h) of paracetamol is significantly smaller (p = 0.009), while the AUC0-8h ratios of the glucuronide, sulphate and cysteine metabolites to paracetamol are significantly higher (p = 0.043, 0.004 and 0.010, respectively). In this model, paracetamol CYP2E1-mediated clearance (cysteine and mercapturate) increased with lean body weight.
研究组 & 干预措施
Paracetamol 15mg/kg
干预措施: Paracetamol 120Mg/5mL Oral Suspension (Drug)
结局指标
主要结局
Clearance paracetamol
时间窗: Three days
- Clearance (unchanged paracetamol) of paracetamol in patients with SMA, CP and ICU-admitted patients with either SMA or CP
Volume of distribution of paracetamol
时间窗: Three days
- Volume of distribution of paracetamol in patients with SMA, CP and ICU-admitted patients with SMA or CP, in comparison with healthy controls.
次要结局
- Liver function tests: ALT, AST, LDH, Alkaline Phosphatase, Bilirubin (U/L)(Three days)
- Concentration-time data on liver function and paracetamolparametres(Three days)
研究者
Mette Cathrine Oerngreen
Principal Investigator
Rigshospitalet, Denmark