Spinal muscular atrophy type II (SMA II) Cerebral palsy (CP)
Conditions
Interventions
Pharmaceutical Form: Oral suspension
INN or Proposed INN: Paracetamol
Other descriptive name: PARACETAMOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration numb
Sponsors
Copenhagen Neuromuscular Center
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - 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: o Children: 6-18 years o Adult patients: 18-45 years o Healthy controls: 18-45 years o ICU-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
Exclusion criteria
Exclusion criteria: - 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP. ;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. -Volume of distribution of paracetamol in patients with SMA, CP and ICU-admitted patients with SMA or CP, in comparison with healthy controls. ;Timepoint(s) of evaluation of this end point: Before, during and after treatment of paracetamol in three consecutive days. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Liver function tests in patients with SMA, CP and ICU-admitted patients with SMA or CP. - 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)). ;Timepoint(s) of evaluation of this end point: Before, during and after treatment of paracetamol in three consecutive days. | — |
Countries
Denmark
Contacts
Public ContactCopenhagen Neuromuscular Center
Rigshospitalet
Outcome results
None listed