Drug resistant epilepsy in children MedDRA version: 21.0 Level: PT Classification code 10015037 Term: Epilepsy System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: Clioquinol
Pharmaceutical Form: Oral solution
INN or Proposed INN: CLIOQUINOL
CAS Number: 130-26-7
Other descriptive name: PBT-1
Concentration unit: mg/ml milligram(s)/millilitre
Concent
Sponsors
University Hospital Leuven
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Voluntary written informed consent of the participant or their legally authorized representative has been obtained prior to any screening procedures 2. In females with child bearing potential: negative pregnancy test or use of highly effective methods of birth control; defined as those that, alone or in combination, result in low failure rate (i.e., less than 1% per year) when used consistently and correctly; such as implants, injectables, combined oral contraceptives, some intra-uterine devices, true sexual abstinence (i.e. refraining from heterosexual intercourse during the entire period of risk associated with the Trial treatment(s)) or commitment to a vasectomized partner 3. Age = 4 years and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Participant has a history of liver or kidney disease. Children with a co-existing active neuropathy (such as neuritis optica, transverse myelitis) 2. Asian etnicity 3. Abnormal low blood level of vitamin B12 or Zn 4. Patients with hypothyroidism 5. Any disorder, which in the Investigator’s opinion might jeopardize the participant’s safety or compliance with the protocol 6. Any prior or concomitant treatment(s) that might jeopardize the participant’s safety or that would compromise the integrity of the Trial 7. Exposure to clioquinol before the trial 8. Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing potential and not using an adequate, highly effective contraceptive 9. Participation in an interventional Trial with an IMP or device
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To study whether add-on clioquinol decreases the seizure frequency and severity in children with drug-resistant epilepsy.;Secondary Objective: a. Safety during trial (systematic recording of adverse events) b. Assessment of seizure severity with the National Hospital Seizure Severity Scale (NHS3 scale) c. Assessment of overall impact of seizures, medication side effects, comorbidities, and overall Quality of Life (QoL) using the Personal Impact of Epilepsy Scale (PIES);Primary end point(s): Efficacy of Clioquinol will be measured: a. percentage of 50% responders after 2 weeks and 10 weeks exposure to low (1mg/kg BID) and higher dose (4mg/kg BID) of clioquinol respectively (visit 3 and 5) b. median % reduction of the seizure frequency at visit 5;Timepoint(s) of evaluation of this end point: A and B is evaluated on visit 3 and 5. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): (Cardiac) Safety, QoL and seizure severity;Timepoint(s) of evaluation of this end point: ECG: Screening and visit 5 AE + Concomitant medication: every visit QoL and seizure severity: Visit 2, 3, 5 and 6 | — |
Countries
Belgium
Contacts
Public ContactClinical Trial Assistant
University Hospital Leuven
Outcome results
None listed