A Study of TAK-861 in People With Narcolepsy Type 1

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 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 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 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.

Study Overview

• Status: Completed
• Conditions: Narcolepsy Type 1
• Intervention / Treatment: Drug: TAK-861; Drug: Placebo

Detailed Description

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

The study will enroll approximately 93 participants. Participants will be randomly assigned (by chance, like flipping a coin) to one of the two treatment groups:

1. TAK-861
2. Placebo

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

• Study Type: Interventional
• Enrollment (Actual): 105
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Glebe, New South Wales, Australia
      • Takeda Site 1
• Austria
  • Linz, Austria, 4020
    • Takeda Site 22
• Belgium
  • Alken, Belgium
    • Takeda Site 2
  • Erpent, Belgium, 5101
    • Takeda Site 3
  • Ghent, Belgium, 9000
    • Takeda Site 25
  • Leuven, Belgium, 3000
    • Takeda Site 24
  • Liège, Belgium, 4000
    • Takeda Site 23
• China
  • Guangdong, China
    • Takeda Site 26
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Takeda Site 4
  • Henan
    • Zhengzhou, Henan, China
      • Takeda Site 27
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Takeda Site 28
• Finland
  • Helsinki, Finland, 380
    • Takeda Site 20
  • Tampere, Finland, 33520
    • Takeda Site 21
• France
  • Bron, France
    • Takeda Site 6
  • Marseille, France, 13385
    • Takeda Site 14
  • Montpellier, France
    • Takeda Site 5
  • Nantes, France, 44093
    • Takeda Site 15
  • Bordeaux
    • Léon, Bordeaux, France, 33076
      • Takeda Site 16
• Italy
  • Bologna, Italy, 40139
    • Takeda Site 7
  • Pozzilli, Italy, 86077
    • Takeda Site 17
  • Roma, Italy
    • Takeda Site 8
• Poland
  • Krakow, Poland
    • Takeda Site 9
• South Korea
  • Seoul, South Korea, 3080
    • Takeda Site 12
  • Daegu
    • Seoul, Daegu, South Korea, 41931
      • Takeda Site 11
  • Gyeonggi-do
    • Suwon, Gyeonggi-do, South Korea, 16247
      • Takeda Site 10
• Spain
  • Barcelona, Spain, 08035
    • 28003
  • Madrid, Spain
    • Takeda Site 13
• Sweden
  • Uppsala, Sweden
    • Takeda Site 19

Participation Criteria

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 any current unstable psychiatric disorder or current active major depressive episode (MDE) or an active MDE in the past 6 months.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Participants will receive TAK-861-matching placebo tablets, orally, for 12 weeks.	Drug: Placebo; TAK-861-matching placebo tablet.
Experimental: TAK-861; Participants will receive TAK-861 tablets, orally, for 12 weeks.	Drug: TAK-861; Oral tablet.

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	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 will be recorded. Participants will be required to stay awake in between the 4 sessions.	Baseline, Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Weekly Cataplexy Rate (WCR) at Week 12		Week 12
Patient Global Impression of Change (PGI-C) Score at 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.	Week 12
Number of Participants with At Least one Treatment-Emergent Adverse Event (TEAE)	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 ESS Total Score	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.	Baseline, Week 12
Change From Baseline to Week 12 in Mean Number of Lapses on the 3 Psychomotor Vigilance Test (PVT)	The PVT is a simple reaction performance task that aims to measure sustained attention. The change from baseline in the mean number of lapses (delayed responses to a visual cue from intraday sessions) during the 10 minute test will be used as a measure of objective sustained attention.	Baseline, Week 12
Change From Baseline to Week 12 in Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score	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.	Baseline, Week 12
Change From Baseline to Week 12 in Functional Impacts of Narcolepsy Instrument (FINI) Domain Scores	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.	Baseline, Week 12
Change From Baseline to Week 12 in Short Form-36 Survey (SF-36) Mental and Physical Component Scores	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.	Baseline, Week 12

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link.

Study record dates

Study Major Dates

• Study Start (Actual): October 8, 2024
• Primary Completion (Actual): June 4, 2025
• Study Completion (Actual): June 4, 2025

Study Registration Dates

• First Submitted: July 11, 2024
• First Submitted That Met QC Criteria: July 11, 2024
• First Posted (Actual): July 17, 2024

Study Record Updates

• Last Update Posted (Estimated): September 15, 2025
• Last Update Submitted That Met QC Criteria: September 12, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Nervous System Diseases; Mental Disorders; Sleep Wake Disorders; Sleep Disorders, Intrinsic; Dyssomnias; Disorders of Excessive Somnolence; Narcolepsy; Substandard Drugs; Pharmaceutical Preparations; Counterfeit Drugs
• Other Study ID Numbers: TAK-861-3002; 2024-511998-30-00 (Ctis: EU CTIS)

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