Is treatment with the painkiller paracetamol safe in patients with spinal muscular atrophy and cerebral palsy?

• Register: EUCTR
• Last refreshed on: 29 November 2021
• Main ID: EUCTR2018-002295-40-DK
• Date of registration: 11/07/2018
• Prospective Registration: Yes
• Primary sponsor: Copenhagen Neuromuscular Center
• Public title: Is treatment with the painkiller paracetamol safe in patients with spinal muscular atrophy and cerebral palsy?
• Scientific title: Pharmacokinetics and safety of treatment with paracetamol in children and adults with spinal muscular atrophy and cerebral palsy - Paracetamol study in patients with low muscle mass
• Date of first enrolment: 11/09/2018
• Target sample size: 24
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-002295-40
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Matched healthy controls for adult patients and data from litterature for child patients Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Denmark

Contacts

• Name: Copenhagen Neuromuscular Center
• Affiliation: Rigshospitalet

• Name: Copenhagen Neuromuscular Center
• Affiliation: Rigshospitalet

Key inclusion & exclusion 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:
- 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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Spinal muscular atrophy type II (SMA II) Cerebral palsy (CP)

Intervention(s)

Pharmaceutical Form: Oral suspension
INN or Proposed INN: Paracetamol
Other descriptive name: PARACETAMOL
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 24-

Primary Outcome(s)

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.

Main Objective: To investigate the safety and toxicity related to paracetamol treatment in children and adults with respectively SMA and CP.

Secondary Outcome(s)

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.

Secondary ID(s)

08-06-2018-paracet

Source(s) of Monetary Support

Elsass Foundation

Ethics review

Status: Approved
Approval date: 11/09/2018
Contact: