E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Paediatric non-convulsive status epilepticus |
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E.1.1.1 | Medical condition in easily understood language |
Epilepsy WITHOUT convulsion. |
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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 | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10057769 |
E.1.2 | Term | Nonconvulsive status epilepticus |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To assess the feasibility of the protocol and of data collection (blood gases, respiratory rate, breathlessness) and to confirm recruitment rates to inform the design of a definitive randomised controlled trial. |
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E.2.2 | Secondary objectives of the trial |
To assess the safety and tolerability of 5% carbogen inhalation in the context of non-convulsive status epilepticus (NCSE)in children. To seek preliminary evidence of efficacy in the form of short-term normalisation of EEG and clinical improvement during and after the period of carbogen administration. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Child under sixteen with known epilepsy (i.e. established tendency to recurrent seizures)
• Aetiology of epilepsy known, or investigation deemed sufficient to infer a cryptogenic (presumed symptomatic) epilepsy
• No clinical suspicion of current episode being associated with acutely raised intracranial pressure
• Confirmed non-convulsive status epilepticus on EEG defined as the presence of o (i) bihemispheric (ii) continuous or near-continuous (iii) spike-wave or other features indicative of seizure activity and o history from parent, or other experienced carer familiar with the patient, of lower level of awareness than normal.
• Note that the presence of subtle continuing clinical seizure activity is not an exclusion criterion so long as the treating physician is satisfied that urgent treatment is not required.
• Informed consent provided by parent/legal guardian
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E.4 | Principal exclusion criteria |
• Age > 16 years
• Absence of informed consent from parent/legal guardian
• Evidence of prior chronic respiratory disease and/or type 2 respiratory failure as indicated by a baseline (pre-inhalation) capillary blood gas pCO2 > 8kPa
• Any clinical suspicion of a condition associated with acutely raised intracranial pressure
• Currently involved in any other clinical trial of an investigative medical product.
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E.5 End points |
E.5.1 | Primary end point(s) |
Duration of tolerated period of carbogen inhalation
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Initial inhalation will be for 120 seconds. If interim analyses suggest acceptable tolerability but limited efficacy, inhalation duration may be increased to 360s or 600s at the DMECs discretion. |
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E.5.2 | Secondary end point(s) |
Incidence of adverse effects
Recruitment rates to trial
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
AE data will be collected on an ongoing basis. Recruitment rates will be assessed at the end of this pilot study. |
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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 | No |
E.6.4 | Safety | Yes |
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 | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 2 |
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
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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 | 2 |
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 | 2 |
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 |