E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8ADEE) |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10077380 |
E.1.2 | Term | Epileptic encephalopathy |
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 evaluate the long-term safety and tolerability of NBI-921352 when administered for up to 106 weeks. |
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E.2.2 | Secondary objectives of the trial |
To investigate the effect of NBI-921352 on long-term seizure control. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Written or oral pediatric assent from the subject and written informed consent from the subject’s parent(s) or legal guardian(s) for subjects<18 years of age and for subjects ≥18 years of age. 2. Completed 16 weeks of treatment in Study NBI-921352-DEE2012. 3. Continue to use a nocturnal alerting system or practice consistent with standards of care for the duration of the study. Acceptable nocturnal alerting systems or practices 4. Have an adequate rescue medication regimen per the investigator's judgment in place for the duration of the study. 5. Female subjects of childbearing potential must agree to use contraception consistently from Day 1 until the final study visit or 30 days after the last dose of study treatment, whichever is longer. 6. A male subject of childbearing potential is defined as a subject who has reached spermarche and has not been vasectomized for at least 3 months prior to Day 1. Male subjects must agree to use contraception consistently from Day 1 until 30 days after the last dose of study treatment
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E.4 | Principal exclusion criteria |
1.Females who are pregnant or currently breastfeeding. 2.Have developed any other disorder for which the treatment takes priority over treatment ofSCN8A-DEE or is likely to interfere with study treatment or impair treatment compliance. 3.The subject or subject’s parent/caregiver is, in the investigator’s opinion, unlikely to comply with the protocol, including the requirement to travel to the study sites for study visits, or is unsuitable for any reason. 4.Have received any other investigational drug within 30 days or 5 half-lives (if known),whichever is longer, of Day -1 or plan to use an investigational drug (other than the study treatment) during the study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
•The subject incidence of serious TEAEs, TEAEs leading to discontinuation of studytreatment, and fatal TEAEs. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Treatment period : Day 1 to Week 106. |
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E.5.2 | Secondary end point(s) |
Percentage change from baseline in 28-day seizure frequency for countable motorseizures during the Treatment Period of the study at Weeks 5, 30, 54, 78, and 104.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
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) | Yes |
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 | 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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Canada |
United States |
Belgium |
Czechia |
Denmark |
France |
Germany |
Italy |
Netherlands |
Poland |
Portugal |
Spain |
United Kingdom |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 5 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |