E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Lennox-Gastaut Syndrome (LSG)
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|
E.1.1.1 | Medical condition in easily understood language |
LSG
Childhood-Onset Epilepsy |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
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 evaluate that the efficacy of E2080 in percent change in
tonic-atonic seizure frequency in participants with LGS relative to placebo. |
|
E.2.2 | Secondary objectives of the trial |
To compare the efficacy of E2080 in terms of 50% responder
rate in tonic-atonic seizure frequency, percent change in the
total seizure frequency, percent change in the frequency of
seizures other than tonic-atonic seizures, and clinical global
impression of change (CGIC) in participants with LGS relative to
placebo, to compare the safety and tolerability of E2080 relative to
placebo and to evaluate the pharmacokinetics of E2080. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Participants who are diagnosed as LSG with tonic/atonic seizures and atypical absence seizures (A history of atypical absence seizures will also be incorporated).
2. Participants who had a slow spike-and-wave pattern in an electroencephalogram within 6 months prior to the enrollment for the Observation Period.
3. Participants who had at least a total of 90 seizures in the 28 days prior to the enrollment for the Observation Period.
4. Participants who have been on 1 - 3 anti-epileptic drugs from 28 days prior to the enrollment for the Observation Period and have not changed the type of the anti-epileptic drugs.
5. Participants who have not changed the type nor the dose or administration of the anti-epileptic drugs they are taking in the Observation Period. |
|
E.4 | Principal exclusion criteria |
1. Participants who had a history of generalized tonic-clonic status epilepticus within baseline.
2. Participants who received drug therapy at least 4 times to be rescued from status epilepticus within baseline.
3. Participants who had a history of hypoxia which needed emergency resuscitation within 12 months prior to the Treatment Period.
4. Participants who were on a ketogenic diet or have received adrenocorticotropic hormone (ACTH) therapy or Vitamin B6 therapy within 6 months prior to the Treatment Period.
5. Participants who had a history of suicide attempt within the 1 year prior to the Treatment Period.
6. Participants who had a history of or has an allergy to triazole compound.
7. Participants who have clinically significant electrocardiogram abnormalities at baseline.
8. Participants who are pregnant, who may be pregnant, who are lactating or who wish to be pregnant. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Percent Change in Tonic-Atonic Seizure Frequency From Baseline (Per 28 Days)
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|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline (28 day observational period) and End of Treatment (28 day treatment period) |
|
E.5.2 | Secondary end point(s) |
1. Number of Participants Achieving a 50% Reduction in Tonic-atonic Seizure Frequency
2. Percent Change in Total Seizure Frequency (Per 28 Days)
3. Percentage Change in the Frequency of Seizures Other Than Tonic-atonic Seizures (Per 28 Days)
4. CGIC |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. 12 weeks
2. Baseline (28 day observational period) and End of Treatment (28 day treatment period)
3. Baseline (28 day observational period) and End of Treatment (28 day treatment period)
4. Up to Week 12 of the treatment period
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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 | 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 | 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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
|
E.8.7 | Trial has a data monitoring committee | No |
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial months | 14 |