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

August 27, 2026 updated by: Takeda

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of TAK-861 for the Treatment of Narcolepsy With Cataplexy (Narcolepsy Type 1)

The main aim of this study is to learn how effective TAK-861 (oveporexton) is in improving excessive sleepiness during the day (called excessive daytime sleepiness or EDS) after 3 months of treatment. Other aims are to learn how effective TAK-861 (oveporexton) is in lowering the number of sudden, unexpected attacks of muscle weakness while staying conscious (cataplexy) in a week; to learn the effect TAK-861 (oveporexton) has on participants' ability to maintain attention, participant's overall quality of life, the spectrum of narcolepsy symptoms, and daily life functions; and to learn about the safety of TAK-861 (oveporexton).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The drug being tested in this study is called TAK-861 (oveporexton). TAK-861 (oveporexton) is being tested to evaluate its efficacy and safety in people with narcolepsy type 1 (NT1).

The study will enroll approximately 152 patients. Participants will be randomly assigned (by chance, like flipping a coin) to one of the three treatment groups:

  1. TAK-861 Dose 1
  2. TAK-861 Dose 2
  3. Placebo

The study drug will be administered for 12 weeks. This multi-center trial will be conducted globally.

Study Type

Interventional

Enrollment (Actual)

168

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Ottawa, Ontario, Canada, K2A 3Z3
        • Takeda Site 21
      • Toronto, Ontario, Canada, M5S 3A3
        • Takeda Site 11
    • Quebec
      • Montreal, Quebec, Canada, H4J 1C5
        • Takeda Site 50
  • France
      • Lyon, France, 69004
        • Takeda Site 40
      • Paris, France, 75013
        • Takeda Site 29
    • Herault
      • Montpellier, Herault, France, 34090
        • Takeda Site 25
    • Midi-Pyrenees
      • Toulouse, Midi-Pyrenees, France, 31059
        • Takeda Site 43
  • Germany
      • Berlin, Germany, 10117
        • Takeda Site 34
      • Schwerin, Germany, 19053
        • Takeda Site 26
    • Bavaria
      • Regensberg, Bavaria, Germany, 93053
        • Takeda Site 47
  • Italy
      • Bologna, Italy, 40139
        • Takeda Site 30
    • Lazio
      • Rome, Lazio, Italy, OO133
        • Takeda Site 32
  • Japan
    • Fukuka-Ken
      • Kurume-shi, Fukuka-Ken, Japan, 830-0011
        • Takeda Site 16
    • Fukuoka
      • Kitakyushu-shi, Fukuoka, Japan, 802-0084
        • Takeda Site 13
    • Fukuoka-Shi Hakata-Ku
      • Fukuoka, Fukuoka-Shi Hakata-Ku, Japan, 812-0025
        • Takeda Site 14
    • Kanagawa
      • Kohoku, Kanagawa, Japan, 222-0033
        • Takeda Site 9
    • Kumamoto
      • Kumamoto, Kumamoto, Japan, 861-0954
        • Takeda Site 7
    • Nagasaki
      • Isahaya-shi, Nagasaki, Japan, 854-0081
        • Takeda Site 17
    • Okinawa
      • Urasoe-Shi, Okinawa, Japan, 901-2132
        • Takeda Site 15
    • Osaka
      • Yodogawa, Osaka, Japan, 532-0003
        • Takeda Site 12
    • Tokyo
      • Bunkyo-ku, Tokyo, Japan, 112-0012
        • Takeda Site 8
      • Kodaira-shi, Tokyo, Japan, 187-8551
        • Takeda Site 19
      • Shibuya-ku, Tokyo, Japan, 151-0053
        • Takeda Site 20
  • Netherlands
      • Zwolle, Netherlands
        • Takeda Site 28
  • Norway
      • Oslo, Norway, 450
        • Takeda Site 33
    • Hordaland
      • Bergen, Hordaland, Norway, 5021
        • Takeda Site 27
  • Spain
      • Barcelona, Spain, 8035
        • Takeda Site 35
      • Barcelona, Spain, O8036
        • Takeda Site 36
      • Madrid, Spain, 28046
        • Takeda Site 24
      • Valencia, Spain, 46600
        • Takeda Site 41
    • Alava
      • Vitoria-Gasteiz, Alava, Spain, 1004
        • Takeda Site 37
    • Castellon
      • Castellon, Castellon, Spain, 12004
        • Takeda Site 38
  • Switzerland
      • Bern, Switzerland, 3010
        • Takeda Site 23
      • Lugano, Switzerland, CH-6900
        • Takeda Site 51
    • Canton of Aargau
      • Barmelweid, Canton of Aargau, Switzerland, 5017
        • Takeda Site 22
  • United Kingdom
      • Cambridge, United Kingdom, CB2 OAY
        • Takeda Site 46
      • London, United Kingdom, SE1 9RT
        • Takeda Site 52
      • London, United Kingdom, W1G 9ST
        • Takeda Site 53
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Takeda Site 48
    • California
      • Redwood City, California, United States, 94063
        • Takeda Site 39
      • Santa Ana, California, United States, 92705
        • Takeda Site 31
      • Upland, California, United States, 91786
        • Takeda Site 18
    • Florida
      • Orlando, Florida, United States, 32803
        • Takeda Site 4
    • Georgia
      • Atlanta, Georgia, United States, 30328
        • Takeda Site 3
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Takeda Site 45
    • Missouri
      • St Louis, Missouri, United States, 63123
        • Takeda Site 5
    • North Carolina
      • Denver, North Carolina, United States, 28037
        • Takeda Site 2
    • Ohio
      • Cincinnati, Ohio, United States, 45227
        • Takeda Site 6
      • Cleveland, Ohio, United States, 44195
        • Takeda Site 42
    • Pennsylvania
      • Danville, Pennsylvania, United States, 17821
        • Takeda Site 49
    • South Carolina
      • Columbia, South Carolina, United States, 29201
        • Takeda Site 1
    • Texas
      • Dallas, Texas, United States, 75231
        • Takeda Site 44
    • Virginia
      • Norfolk, Virginia, United States, 23510
        • Takeda Site 10

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion 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 greater than or equal to (≥)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 less than or equal to (≤)110 picograms per milliliter (pg/mL) [or less than one-third of the mean values obtained in normal participants within the same standardized assay].

Exclusion 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Participants received oveporexton (OVE)-matching placebo tablets, orally, for 12 weeks.
Drug: Placebo
OVE-matching placebo tablet.
Experimental: OVE 1 mg BID
Participants received OVE tablets, 1 milligram (mg), orally, for 12 weeks.
Drug: Oveporexton
Oral tablet.
Other Names:
  • TAK-861
Experimental: OVE 2 mg BID
Participants received OVE tablets, 2 mg, orally, for 12 weeks.
Drug: Oveporexton
Oral tablet.
Other Names:
  • TAK-861

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to Week 12 in Mean Sleep Latency From the 4 Maintenance of Wakefulness Test (MWT) Wake Trials
Time Frame: Baseline, 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 (trials) done 2 hours apart. Sleep latency in each session was recorded. Participants were required to stay awake in between the four sessions. A positive change from baseline indicates an improvement. The linear mixed effects model for repeated measures (MMRM) was used for analysis.
Baseline, Week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline to Week 12 in Mean Number of Lapses on the Psychomotor Vigilance Test (PVT)
Time Frame: Baseline, Week 12
The PVT is a simple reaction performance task that aims to measure sustained attention. The mean number of lapses (delayed responses to a visual cue from intraday sessions) from the two 10-minute intraday sessions was used as a measure of objective sustained attention. A negative change from baseline indicates an improvement. A constrained Longitudinal Data Analysis (cLDA) model was used to analyze the mean number of lapses and to estimate change from baseline at each visit.
Baseline, Week 12
Change From Baseline to Week 12 in Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score
Time Frame: Baseline, 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. A negative change from baseline indicates an improvement. The linear MMRM was used for analysis.
Baseline, Week 12
Change From Baseline to Week 12 in Functional Impacts of Narcolepsy Instrument (FINI) Domain Scores
Time Frame: Baseline, Week 12
The FINI measures the functional impacts of narcolepsy across 6 domains Tiredness (items 1-7), Cognitive Functioning (items 8-12), Cataplexy (items 13-17), Social Activities (items 18-21), Everyday Activities (items 22-25), and Everyday Responsibilities (items 26-28). Each item asks about the impact that narcolepsy has had on their daily functioning during the past 7 days, and is scored from 0 to 4, where 0 indicates the best health and 4 the worst. An average score is calculated for each domain and then standardized to a 0-100 scale, where 0 indicates the best health and 100 the worst health. Standardized score = average score/4 × 100. A negative change from baseline indicates and improvement. The linear MMRM was used for analysis.
Baseline, Week 12
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.
Up to 16 weeks
Change From Baseline to Week 12 in Epworth Sleepiness Scale (ESS) Total Score
Time Frame: Baseline, 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. A negative change from baseline indicates an improvement. The linear MMRM was used for analysis.
Baseline, Week 12
Weekly Cataplexy Rate (WCR) at Week 12
Time Frame: Week 12
Participants completed a daily participant-reported cataplexy diary to record self-reported episodes of cataplexy attacks. WCR = (total number of cataplexy attacks over a number of non-missing diary days for a given period/number of non-missing diary days in that period)*7. The generalized estimating equations (GEE) model was used for analysis. Reported here is the estimated mean of incidence rate with a 95 percent (%) confidence interval (CI).
Week 12
Responder Rate on Patient Global Impression of Change (PGI-C) Score at Week 12
Time Frame: Week 12
The PGI-C is a participant self-rated scale to assess change 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. Responders were the participants reporting "1=very much improved" or "2=much improved" on PGI-C. Responder rate (proportion of participants who were responders) was estimated using a GEE model.
Week 12
Change From Baseline to Week 12 in Short Form-36 Survey (SF-36) Mental and Physical Component Scores
Time Frame: Baseline, 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. A positive change from baseline indicates an improvement. The linear MMRM was used for analysis.
Baseline, Week 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Takeda

Investigators

  • Study Director: Medical Director, Takeda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 2, 2024

Primary Completion (Actual)

June 3, 2025

Study Completion (Actual)

June 3, 2025

Study Registration Dates

First Submitted

June 18, 2024

First Submitted That Met QC Criteria

June 18, 2024

First Posted (Actual)

June 24, 2024

Study Record Updates

Last Update Posted (Actual)

August 31, 2026

Last Update Submitted That Met QC Criteria

August 27, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TAK-861-3001
  • 2023 (U.S. NIH Grant/Contract: GRAMMY Museum Foundation)
  • 2023-508465-32-00 (Ctis)
  • jRCT2051240083 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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