| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
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| E.1.1.1 | Medical condition in easily understood language |
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| E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
| MedDRA Classification |
| E.1.2 Medical condition or disease under investigation |
| E.1.2 | Version | 21.1 |
| E.1.2 | Level | PT |
| E.1.2 | Classification code | 10070530 |
| E.1.2 | Term | Benign rolandic epilepsy |
| E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
| E.1.3 | Condition being studied is a rare disease | No |
| E.2 Objective of the trial |
| E.2.1 | Main objective of the trial |
The primary objective of the trial is to test two hypotheses:
A) To determine if Carbamazepine or Levetiracetam are superior to no AED with respect to time to 6-month seizure remission.
B) To determine if a Parent-Based Sleep (PBS) intervention is superior to standard care with respect to 3-month sleep problem frequency measured by Children’s Sleep Habits Questionnaire (CSHQ).
Primary (economic):
To estimate the cost-utility of carbamazepine, levetiracetam and PBS |
|
| E.2.2 | Secondary objectives of the trial |
| 1)To compare how long it takes for treatment to fail due to uncontrolled seizures or unacceptable reactions 2)To compare how long it takes for treatment to fail due to uncontrolled seizures 3)To compare how long it takes for treatment to fail due to unacceptable reactions 4)To compare time to 1st seizure 5)To compare time to 12-month remission from seizures 6)To determine if the sleep training plan is better than standard care 7)To compare measures of cognition across treatment groups 8)To compare health related&quality of life across treatment groups 9)To compare measures of children’s behaviour across treatment groups 10)To detect any adverse reactions&their rate 11)To estimate child health utilities&QALYs across treatment groups 12)To estimate health utilities&QALYs across treatment groups 13) To compare parenting self-efficacy across the different treatment groups 14)To compare sickness related school absences across treatment groups 15)To determine the costs to the NHS&PSS |
|
| E.2.3 | Trial contains a sub-study | Yes |
| E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Title: Qualitative Interview Study
Version: N/A (same as the protocol)
Objective: To explore and capture qualitative information relating to children’s and their parents’ experiences of the decisions and choices they make regarding the treatment and management of RE. |
|
| E.3 | Principal inclusion criteria |
a. Children diagnosed with RE
b. EEG showing focal sharp waves with normal background
c. Aged ≥5 years and <13 years at the time of randomisation
d. Currently untreated with antiepileptic drugs
e. Written informed consent received from person with parental responsibility/legal representative.
f. Family have a valid email address and regular internet access (for online sleep intervention)
g. Parent and child are to have a good understanding of the English language.
Parent-child dyads meeting the trial criteria and the following characteristics will be eligible for inclusion in the qualitative sub-study:
h. Ability of parent and child to speak conversational English
i. Additional consent to main trial: Consent of parent to participate and for their child to participate. |
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| E.4 | Principal exclusion criteria |
a. Known contraindication to any of the trial drugs
b. Previously treated for epilepsy with antiepileptic drugs |
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| E.5 End points |
| E.5.1 | Primary end point(s) |
A) Time to six month seizure remission based on seizure report.
B) Total sleep problem score as measured by the CSHQ.
Primary (economic):
Total costs based on resource use questionnaires, and routine hospital data; and quality-adjusted life years (QALYs) based on health utilities
|
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
A) 3, 6,12 months and annually thereafter - ‘when was last seizure’ question in clinic
B) At baseline and 3 months
Primary (economic)
Resource use and utility questionnaires at baseline, 3, 12 months and annually thereafter; routine hospital data at the end of the trial |
|
| E.5.2 | Secondary end point(s) |
1. Time taken from randomisation to decision by child, parent or treating physician to be removed from treatment due to inadequate seizure control or unacceptable adverse reactions
2. Time taken from randomisation to decision by child, parent or treating physician to be removed from treatment due to inadequate seizure control
3. Time taken from recruitment to decision by child, parent or treating physician to be removed from trial due to unacceptable adverse reactions
4. Time to first seizure based on seizure report
5. Time to 12-month seizure remission based on seizure report
6. Total sleep problem score as measured by the CSHQ
7. Total score in three chosen assessments delivered by CANTAB iPad Neuropsychological battery
8. CHEQOL score change
9. Total score on strengths and difficulties questionnaire (SDQ)
10. Records of adverse reactions
11. Score changes in CHU9D or EQ-5D-Y
12. EQ-5D-5L score change
13. Score changes in: 1. PSAM
14. Total sickness related school absences (days)
15. Results from 1) Resource use questionnaire, 2)Patient Level Information and Costing System (PLICS) data and 3) Hospital Episode Statistics (HES)
16. Summary of actigraphy variables (total sleep time/sleep latency/sleep efficiency) averaged over a 2-week period
Sub-study outcome measure:
16. Face-to-face/telephone or skype interviews (audio recorded and transcribed).
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|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
1., 2. and 3.timepoints are 3,6,12months and annually thereafter
4.and 5.timepoints are 3,6,12months and annually thereafter ‘when was last seizure’ question in clinic
6.At 12 months and annually thereafter
7.At baseline,3 and 12months and annually thereafter
8.and 9.timepoints are at baseline and 1months and annually thereafter
10.Adverse reactions at 3,6,12,24 months and annually thereafter in clinic
11.12. and 13. timpoints are at baseline, 3,12 months and annually thereafter
14. 3,6,12 months and annually thereafter 'sickness related school absences' question in clinic
14.RUQ at 3,12 months and annually thereafter. PLICS and HES at end of study
15.2 weeks actigraphy(arranged centrally via Oxford unit)at baseline,3 and 12 months
16.2-4 weeks, 6months and 12months after randomisation |
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| E.6 and E.7 Scope of the trial |
| E.6 | Scope of the trial |
| E.6.1 | Diagnosis | No |
| E.6.2 | Prophylaxis | No |
| E.6.3 | Therapy | Yes |
| E.6.4 | Safety | No |
| E.6.5 | Efficacy | Yes |
| E.6.6 | Pharmacokinetic | No |
| E.6.7 | Pharmacodynamic | No |
| E.6.8 | Bioequivalence | No |
| E.6.9 | Dose response | No |
| E.6.10 | Pharmacogenetic | No |
| E.6.11 | Pharmacogenomic | No |
| E.6.12 | Pharmacoeconomic | No |
| E.6.13 | Others | No |
| E.7 | Trial type and phase |
| E.7.1 | Human pharmacology (Phase I) | No |
| E.7.1.1 | First administration to humans | No |
| E.7.1.2 | Bioequivalence study | No |
| E.7.1.3 | Other | No |
| E.7.1.3.1 | Other trial type description | |
| E.7.2 | Therapeutic exploratory (Phase II) | No |
| E.7.3 | Therapeutic confirmatory (Phase III) | No |
| E.7.4 | Therapeutic use (Phase IV) | Yes |
| E.8 Design of the trial |
| E.8.1 | Controlled | Yes |
| E.8.1.1 | Randomised | Yes |
| E.8.1.2 | Open | Yes |
| E.8.1.3 | Single blind | No |
| E.8.1.4 | Double blind | No |
| E.8.1.5 | Parallel group | No |
| E.8.1.6 | Cross over | No |
| E.8.1.7 | Other | No |
| E.8.2 | Comparator of controlled trial |
| E.8.2.1 | Other medicinal product(s) | Yes |
| E.8.2.2 | Placebo | No |
| E.8.2.3 | Other | Yes |
| E.8.2.3.1 | Comparator description |
|
| E.8.2.4 | Number of treatment arms in the trial | 3 |
| E.8.3 |
The trial involves single site in the Member State concerned
| No |
| E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
| E.8.4.1 | Number of sites anticipated in Member State concerned | 90 |
| E.8.5 | The trial involves multiple Member States | No |
| E.8.6 Trial involving sites outside the EEA |
| E.8.6.1 | Trial being conducted both within and outside the EEA | No |
| E.8.6.2 | Trial being conducted completely outside of the EEA | No |
| E.8.7 | Trial has a data monitoring committee | Yes |
| E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
| The end of the trial is defined to be the date on which data for all participants is locked and data entry privileges are withdrawn from the trial database. |
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| E.8.9 Initial estimate of the duration of the trial |
| E.8.9.1 | In the Member State concerned years | 4 |
| E.8.9.1 | In the Member State concerned months | 0 |
| E.8.9.1 | In the Member State concerned days | 0 |
| E.8.9.2 | In all countries concerned by the trial years | 4 |
| E.8.9.2 | In all countries concerned by the trial months | 0 |
| E.8.9.2 | In all countries concerned by the trial days | 0 |
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