A Study of TAK-861 for the Treatment of Narcolepsy Type 1

JRCT ID: jRCT2051240083

Registered date:03/07/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Narcolepsy Type 1
• Date of first enrollment: 02/07/2024
• Target sample size: 152
• Countries of recruitment: Canada,Japan,France,Japan,Germany,Japan,Italy,Japan,Netherlands,Japan,Norway,Japan,Spain,Japan,Switzerland,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

TAK-861 Dose 1:
Participants will receive TAK-861 tablets at dose 1, orally, for 12 weeks.

TAK-861 Dose 2:
Participants will receive TAK-861 tablets at dose 2, orally, for 12 weeks.

Placebo:
Participants will receive TAK-861-matching placebo tablets, orally, for 12 weeks.

Outcome(s)

Primary Outcome

1.Mean Sleep Latency from the MWT at Week 12
Time Frame: Week 12
The MWT evaluates a person's ability to remain awake under soporific conditions for a defined period of time. 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. The MWT consists of four 40-minute sessions done 2 hours apart. Sleep latency in each session will be recorded. Participants will be required to stay awake in between the 4 sessions.

Secondary Outcome

1.Epworth Sleepiness Scale (ESS) Total Score at Week 12
Time Frame: Week 12
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.

2.Weekly Cataplexy Rate (WCR) at Week 12
Time Frame: Week 12

3.Number of Lapses on the 3 Post Meridiem (PM) Psychomotor Vigilance Test (PVT) Session at Week 12
Time Frame: Week 12
The PVT is a simple reaction performance task that aims to measure sustained attention. The number of lapses (delayed responses to a visual cue) will be recorded.

4.Patient Global Impression of Change (PGI-C) Score at Week 12
Time Frame: Week 12
The PGI-C is a patient self-rated scale to assess improvement in daytime sleepiness and overall narcolepsy symptoms. The PGI-C includes 7 items being scored from 1 (best outcome) to 7 (worst outcome) with 4 being no change.

5.Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score at Week 12
Time Frame: Week 12
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 disturbed nighttime sleep (DNS) with a total score range of 0 to 57 (sum of 6 items that assess symptoms severity are rated using a six-point Likert scale [0-5] and 9 items that describe the symptom effect on daily life are rated using a four-point Likert scale [0-3]). Higher total scores mean a worse outcome.

6.Functional Impacts of Narcolepsy Instrument (FINI) Domain Scores at Week 12
Time Frame: Week 12
The FINI measures the functional impacts of narcolepsy across 6 domains. Each domain is scored from 0 to 4, where 0 indicates the best health and 4 the worst.

7.Short Form-36 Survey (SF-36) Mental and Physical Component Scores at Week 12
Time Frame: Week 12
The SF-36 is participant-reported survey of participant health that assesses the quality of life and includes both physical and mental components. The scores for each component range from 0 to 100. Higher scores represent better health-related quality of life.

8.Number of Participants with At Least one Treatment-Emergent Adverse Event (TEAE)
Time Frame: Up to 16 weeks
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.

Key inclusion & exclusion criteria

• Age minimum: >= 16age old
• Age maximum: <= 70age old
• Gender: Both

Include criteria

1.The participant has a body mass index (BMI) within the range 18 to 40 kilograms per meter square (kg/m^2).
2.The participant has an International Classification of Sleep Disorders, Third Edition (ICSD-3) or International Classification of Sleep Disorders, Third Edition, Text Revision (ICSD-3-TR) diagnosis of NT1.
3.The participant has >=4 partial or complete episodes of cataplexy/week (WCR).
4.The participant is positive for the human leukocyte antigen (HLA) genotype HLA-DQB1*06:02 or results from radioimmunoassay indicate the participant's cerebrospinal fluid (CSF) orexin (OX)/hypocretin-1 concentration is =<110 picograms per milliliter (pg/mL) [or less than one-third of the mean values obtained in normal participants within the same standardized assay].

Exclude criteria

1.The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS.
2.The participant: (a) has a history of myocardial infarction; (b) has a history of clinically significant hepatic disease, thyroid disease, coronary artery disease, cardiac rhythm abnormality or heart failure; or (c) has any medical condition (such as unstable cardiovascular, pulmonary, renal or gastrointestinal disease).
3.The participant has current or recent (within 6 months) gastrointestinal disease that is expected to influence the absorption of drugs.
4.The participant has a history of cancer in the past 5 years.
5.The participant has a clinically significant history of head injury or head trauma.
6.The participant has a history of epilepsy, seizure, or convulsion.
7.The participant has a history of cerebral ischemia, transient ischemic attack (<5 years from screening), intracranial aneurysm, or arteriovenous malformation.

Related Information

• Primary Sponsor: Nonomura Hidenori
• Secondary ID(s): 2023-508465-32,NCT06470828