Idiopathic Hypersomnia, Narcolepsy Type 1, Narcolepsy Type 2
Conditions
Keywords
Drug Therapy
Brief summary
Central hypersomnia conditions are a group of sleeping disorders where the brain has trouble keeping a person awake during the day (called excessive daytime sleepiness or EDS). These conditions usually include narcolepsy (type 1 and 2) and idiopathic hypersomnia (IH). Narcolepsy type 1 (NT1) includes sudden muscle weakness while you stay awake, called cataplexy, often triggered by strong emotions. Narcolepsy type 2 (NT2) does not include cataplexy. People with narcolepsy typically feel refreshed by short naps. People with IH feel extremely sleepy during the day, and do not feel refreshed by sleep. Waking up from sleep is difficult. This is common in the morning and also when waking up from long naps. The study wants to learn about TAK-360 when taken over a long time period; this is called a long-term extension or LTE study. The main aim of this LTE study is to find out how well participants with NT1, NT2, and IH tolerate TAK-360 over a longer period (long-term tolerability) and to learn how safe TAK-360 is when given over a longer period of time (long-term safety). Participants who completed one of the TAK-360 parent studies can join this study if they meet the study rules. Parent studies include TAK-360-2001(NCT06952699), TAK-360-2002 (NCT06812078), or other TAK-360 studies that evaluate the TAK-360 medicine. All participants will receive TAK-360 in this study. They will either receive the same dose as they did in the parent study, or the closest dose available in this LTE study. Participants who received placebo (the placebo looks just like TAK-360 but does not have any medicine in it) in their parent study will receive one of the TAK-360 doses available in this study. Placebo will only be used to not reveal the dose of TAK-360 from parent studies to investigator, participants, and sponsor. Sponsor, investigators and participants will not know which TAK-360 dose was used in the LTE study as long as the parent study is ongoing. The participants will have to visit the clinic multiple times during this study.
Interventions
DRUGTAK-360
TAK-360 tablets
DRUGPlacebo
TAK-360-matching placebo tablets
Sponsors
Takeda
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 71 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Participant is willing and able to understand and fully comply with trial procedures and requirements. 2. Participant has a confirmed diagnosis of either NT1, NT2, or IH, and has completed the treatment period of a parent TAK-360 trial. 3. Participant has provided informed consent (that is, in writing, documented via a signed and dated informed consent form (ICF) and any required privacy authorization before the initiation of any trial procedures. Key
Exclusion criteria
1. Participant has a positive pregnancy test or is lactating/breastfeeding. 2. Participant has a risk of suicide according to endorsement of item 4 or 5 on the Columbia Suicide Severity Rating Scale (C-SSRS). 3. The participant has developed a new medical disorder associated with excessive daytime sleep (EDS). 4. Participant has developed (within the last 6 months) gastrointestinal disease that is expected to influence the absorption of drugs. 5. Participant has developed a new history of seizures. 6. Participant has experienced clinically significant head injury, per investigator opinion. 7. Participant has developed a history of cerebral ischemia, transient ischemic attack (less than \[\<\] 5 years ago), or cerebral haemorrhage. 8. Participant has developed a history of myocardial infarction, clinically significant coronary artery disease, clinically significant angina, clinically significant cardiac rhythm abnormality, or heart failure. 9. The participant has been diagnosed with medically significant thyroid disease, known functional hepatic impairment, or other severe chronic medical condition other than the central hypersomnolence disorder. 10. Participant has developed a history of cancer in the past 5 years.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With at Least 1 Treatment-Emergent Adverse Event (TEAE) | Up to approximately 5 years | An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product. A TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| All Participants: Change From Baseline in Epworth Sleepiness Scale (ESS) Total Score | Baseline through Year 5 | The ESS provides individuals with 8 different situations of daily life and asks them how likely they are to fall asleep in those situations (scored 0 to 3) and to try to imagine their likelihood of dozing even if they have not actually been in the identical situation; the scores are summed to give an overall score of 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are considered to be within the normal range. |
| All Participants: Change from Baseline in Mean Sleep Latency on the Maintenance of Wakefulness Test (MWT) | Baseline through Month 6 | The MWT evaluates a participant's ability to remain awake under soporific conditions for a defined period. Because there is no biological measure of wakefulness, wakefulness is measured indirectly by the inability or delayed tendency to fall asleep. This tendency to fall asleep is measured via electroencephalography-derived sleep latency in the MWT. One MWT, that includes four 40-minute wake trials, will be done. Sleep latency in each wake trial will be recorded. Participants will be required to stay awake in between the 4 MWT wake trials. |
| IH Participants: Change From Baseline in IH Severity Scale (IHSS) Total Score | Baseline through Year 5 | The IHSS is a 14-item self-assessment questionnaire, to measure the severity, frequency and functional impact of the 3 key IH symptoms (during a recall period of "past 7 days"). The IHSS includes 2 items (1 and 2) on nighttime sleep duration and quality, 3 items (3, 4, and 5) on sleep inertia and sleep drunkenness after nighttime sleep and 1 (8) after daytime nap, and 3 items (6, 7, and 9) on diurnal symptoms (nap occurrence, daytime sleepiness). Items 10 to 14 assess daytime functioning alterations due to hypersomnolence. Six items (1,2,3,5,7,8) are scored with 4 ranged response options, with score from 0 to 3, and 8 items (4,6,9,10,11,12,13,14) with 5 ranged response options, with score from 0 to 4, with 1 additional response option for item 14. The total IHSS score is the sum of all item scores (range: 0 to 50); higher scores indicate more severe symptoms. |
| NT1 Participants: Change From Baseline on the Narcolepsy Severity Scale for Clinical Trials (NSS-CT) | Baseline through Year 5 | The NSS-CT is a 15-item self-administered questionnaire that assesses the severity and consequences of the 5 major narcolepsy symptoms such as daytime sleepiness, cataplexy, hallucinations, sleep paralysis, and disrupted nighttime sleep (DNS) with a total score range of 0 to 57 (sum of 8 items that assess symptoms severity \[2 with 4 response options, 1 response with 6 options, and 5 items capturing event count\], and 7 items that describe the symptom effect on daily life with 4 response options). The presence of narcolepsy symptoms is defined by EDS (score greater than or equal to \[≥\] 1 on at least one of items 1, 2, 3, 4, 5, 6, or 7), cataplexy (score ≥1 on at least one of items 8, 9, or 10), hallucinations (score ≥1 on at least one of items 11 and 12), sleep paralysis (score ≥1 on at least one of the items 13 and 14), and disrupted nighttime sleep by a score ≥1 on item 15. Higher scores indicate greater symptom severity or degree of impact. |
| NT2 Participants: Change From Baseline on the Narcolepsy Severity Scale for Clinical Trials (NSS-2-CT) | Baseline, Month 1 through Year 5 | The NSS-2-CT is a 12-item self-administered questionnaire that assesses the severity and consequences of major narcolepsy symptoms such as daytime sleepiness, hallucinations, sleep paralysis, and disrupted nighttime sleep, with higher scores indicating more severe narcolepsy symptoms and impacts. The total score ranges from 0-44(sum of 6 items that assess event symptom severity \[including 2 items with 4 response options, 1 item with 6 response options, and 3 items capturing event count\], and 6 items that describe symptom effect on daily life, with 4 response options), and differs from the NSS-CT in its omission of cataplexy-related items. The presence of narcolepsy symptoms is defined by EDS (score ≥1 on at least one of items 1, 2, 3, 4, 5, 6, or 7), hallucinations (score ≥1 on at least one of items 8 and 9), sleep paralysis (score ≥1 on at least one of the items 10 and 11), and disrupted nighttime sleep by a score ≥1 on item 12. |
| Change from Baseline in Weekly Cataplexy Rate (WCR) Using the Participant-Reported Cataplexy eDiary | Baseline, Month 3 through Month 24 | Participants will complete a patient-reported sleep diary to record self-reported narcolepsy symptoms. Participants will record episodes of cataplexy attacks in the diary. The total number of events averaged for a week will be reported. |
Countries
China, Japan, United States
Contacts
CONTACTStudy Director
STUDY_DIRECTORTakeda Contact
Takeda
Outcome results
None listed