E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Narcolepsy with Cataplexy (Type 1) and Narcolepsy without Cataplexy (Type 2) |
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E.1.1.1 | Medical condition in easily understood language |
Narcolepsy with Cataplexy (Type 1) and Narcolepsy without Cataplexy (Type 2) |
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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 | 10028713 |
E.1.2 | Term | Narcolepsy |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
• To evaluate the long-term safety and tolerability of TAK-861. |
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E.2.2 | Secondary objectives of the trial |
• To assess the effect of TAK-861 on excessive daytime sleepiness (EDS) as assessed by the mean sleep latency from the Maintenance of Wakefulness Test(MWT) • To assess the effect of TAK-861 on EDS as measured by the Epworth Sleepiness Scale (ESS) total score. • To assess the effect of TAK-861 on cataplexy as assessed by the weekly cataplexy rate (WCR) (participants with narcolepsy type 1 [NT1] only). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Participant with a diagnosis of narcolepsy who has completed a controlled study with TAK-861 (including participants diagnosed with NT1 or NT2) and for whom the investigator has no clinical objection to their enrollment. |
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E.4 | Principal exclusion criteria |
1. Participant has a moderate or severe ongoing treatment emergent adverse event (TEAE) related to the study drug from the parent study or discontinued because of TEAEs in the parent study. 2. Participant has a positive urine screen for drugs of abuse (findings confirmed) and/or positive alcohol test during any visit in their prior TAK-861 study, or during the screening period for participants with a dosing gap. 3. Participant has a risk of suicide according to endorsement of item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS) on any visit in the parent TAK-861 study, or has positive answers on item 4 or 5 on the Screening/Baseline C-SSRS Lifetime (based on the past year) during the screening assessment for participants with a dosing gap. 4. Participant has alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >1.5 times the upper limit of normal (ULN) at multiple visits in the parent study and the findings are of clinical significance, per investigator or sponsor opinion, or ALT/AST >1.5 times ULN during the screening period for participants with a dosing gap. 5. Participant has a current medical disorder, other than narcolepsy with or without cataplexy, associated with excessive daytime sleepiness (EDS). 6. Participant has current active major depressive episode (MDE) or has had an active MDE in the past 6 months. 7. Participant has developed (within the last 6 months) gastrointestinal disease that is expected to influence the absorption of drugs (i.e., a history of malabsorption, esophageal reflux, peptic ulcer disease, erosive esophagitis, frequent [more than once per week] occurrence of heartburn, or any surgical intervention). 8. Participant has epilepsy or history of seizure. 9. Participant has any other medical condition, such as anxiety, depression, heart disease, or significant hepatic, pulmonary, or renal disease, that requires them to take excluded medications. 10. Participant has a history of cerebral ischemia, transient ischemic attack (<5 years ago), or cerebral hemorrhage. 11. Participant has a history of myocardial infarction, clinically significant coronary artery disease, clinically significant angina, clinically significant cardiac rhythm abnormality, or heart failure. 12. Participant has a history of cancer in the past 5 years (does not apply to participants with carcinoma in situ that has been resolved without further treatment, or basal cell skin cancer. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Occurrence of at least 1 treatment-emergent adverse event (TEAE). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Day 395, 425, 485, 515, 575, 605, 665, 695 |
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E.5.2 | Secondary end point(s) |
• Change from baseline in the parent study in MWT mean sleep latency. • Change from baseline in the parent study in ESS total score • Change from baseline in the parent study in WCR using the patient-reported Cataplexy Diary (participants with NT1 only) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Day 182 - Day 14, 28, 56, 84, 182, 266, 364, 448, 546, 630. 728 - Day 70 to 84; 167 to 181; 252 to 266; 350 to 364; 532 to 546; 714 to 728; 729 to 736 |
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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 | No |
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 | 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) | 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) | 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 | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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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 | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 4 |
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 | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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 |
Switzerland |
Australia |
Japan |
United States |
Finland |
France |
Germany |
Italy |
Netherlands |
Norway |
Spain |
Sweden |
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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
|
The end of study is defined as the final date on which data were or are expected to be collected, ie, the last visit of the last participant in the study |
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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 | 4 |
E.8.9.1 | In the Member State concerned days | 4 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 4 |