Is treatment with the painkiller paracetamol safe in patients with spinal muscular atrophy and cerebral palsy?
- Conditions
- Spinal muscular atrophy type II (SMA II)Cerebral palsy (CP)Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2018-002295-40-DK
- Lead Sponsor
- Copenhagen Neuromuscular Center
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 24
-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.
-Age:
oChildren: 6-18 years
oAdult patients: 18-45 years
oHealthy controls: 18-45 years
oICU-admitted patients: 6-45 years
-Signed informed consent to participation in the trial
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
-Inability to understand the purpose of the trial or cooperate in the conduction of the experiments.
oFor 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.<br><br>;Secondary Objective: Not applicable.;Primary end point(s): -Clearance (total, glucuronidation, sulphation, CYP2E1 oxidation and unchanged) of paracetamol in patients with SMA, CP and ICU-admitted patients with either SMA or CP. <br>-Volume of distribution of paracetamol in patients with SMA, CP and ICU-admitted patients with SMA or CP, in comparison with healthy controls. <br>;Timepoint(s) of evaluation of this end point: Before, during and after treatment of paracetamol in three consecutive days.
- Secondary Outcome Measures
Name Time Method Secondary end point(s): -Liver function tests in patients with SMA, CP and ICU-admitted patients with SMA or CP. <br>-Concentration-time data on plasma paracetamol, paracetamol-sulphate, paracetamol-glucuronide, paracetamol-cysteine and paracetamol-mercapturate (oxidative metabolites), plasma-glutathione and liver biomarkers (ALAT, PP, bilirubin, MicroRNA-122 (miR-122)). <br>;Timepoint(s) of evaluation of this end point: Before, during and after treatment of paracetamol in three consecutive days.