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

NCT ID: NCT05687903

Last Updated: 2025-01-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 112 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-09
• Study Completion Date: 2023-12-14

Brief Summary

The main aim of this study is to see how TAK-861 works on symptoms of narcolepsy, including excessive daytime sleepiness and cataplexy. Approximately 100 participants will take part in the study across North America, Europe and Asia Pacific.

The treatment (TAK-861 or placebo) will be administered for 8 or 12 weeks. After this treatment period the participant will have the option to participate in a separate, long- term extension study during which all participants will be treated with TAK-861.

Detailed Description

The drug being tested in this study is called TAK-861. This study will look at the effect of TAK-861 on improvement in narcolepsy symptoms, including excessive daytime sleepiness (EDS) and number of cataplexy episodes.

The study will enroll approximately 100 patients. Participants will be randomly assigned (by chance, like flipping a coin) to one of the five treatment groups which will remain undisclosed to the participant and study doctor during the study (unless there is an urgent medical need):

• TAK-861 Dose 1
• TAK-861 Dose 2
• TAK-861 Dose 3
• TAK-861 Dose 4
• Placebo (dummy inactive pill) - this is a tablet that looks like the study drug but has no active ingredient

This multi-center trial will be conducted worldwide. The overall time to participate in this study is up to 23 weeks. Participants will make multiple visits to the clinic during the treatment period and then will either enroll in a long-term extension study in which all participants will receive TAK-861 or have 2 final visits 7 and 28 days after last dose of study drug for follow-up assessments.

Conditions

Narcolepsy Type 1

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

Placebo

Participants received placebo tablets matching TAK-861, orally, twice daily (BID), from Days 1 to 56.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo oral tablets matching TAK-861

TAK-861 0.5 mg BID

Participants received TAK-861 0.5 milligrams (mg), orally, BID, from Days 1 to 56.

Group Type: EXPERIMENTAL

TAK-861

Intervention Type: DRUG

TAK-861 oral tablets

TAK-861 2 mg BID

Participants received TAK-861 2 mg, orally, BID, from Days 1 to 56.

Group Type: EXPERIMENTAL

TAK-861

Intervention Type: DRUG

TAK-861 oral tablets

TAK-861 2 mg and 5 mg

Participants received TAK-861 2 mg followed by 5 mg dose, orally, BID, from Days 1 to 56.

Group Type: EXPERIMENTAL

TAK-861

Intervention Type: DRUG

TAK-861 oral tablets

TAK-861 7 mg QD

Participants received TAK-861 7 mg, orally, once daily (QD), from Day 1 to 56. Placebo was given as the second dose.

Group Type: EXPERIMENTAL

TAK-861

Intervention Type: DRUG

TAK-861 oral tablets

Placebo

Intervention Type: DRUG

Placebo oral tablets matching TAK-861

Interventions

TAK-861

TAK-861 oral tablets

Intervention Type: DRUG

Placebo

Placebo oral tablets matching TAK-861

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. The participant is aged 18 to 70 years, inclusive, at the time of signing the informed consent form (ICF).

Note: In Japan, participants aged 16 to 70 years, inclusive, may be included.

2. The participant has body mass index (BMI) within the range 18 to 40 kilogram per square meter [kg/m^2] (inclusive).

3. The participant has an International Classification of Sleep Disorders, 3rd Edition (ICSD-3) diagnosis of narcolepsy type 1 (NT1) by polysomnography (PSG)/Multiple Sleep Latency Test (MSLT), performed within the past 10 years.

4. The participant is positive for the human leukocyte antigen (HLA) genotype HLA-DQB1*06:02 or results from cerebrospinal fluid (CSF) testing indicate the participant's 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).

Exclusion Criteria

1. The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS.

2. The participant has medically significant hepatic or thyroid disease.

3. The 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 cancer).

4. The participant has clinically significant coronary artery disease, a history of myocardial infarction, clinically significant angina, clinically significant cardiac rhythm abnormality, or heart failure.

5. The participant has a clinically significant history of head injury or head trauma.

6. The participant has history of epilepsy, seizure, or convulsion, or has a family history of inherited disorders associated with seizure (except for a single febrile seizure in childhood).

7. The participant has one or more of the following psychiatric disorders:

1. Any current unstable psychiatric disorder.

2. Current or history of manic or hypomanic episode, schizophrenia or any other psychotic disorder, including schizoaffective disorder, major depression with psychotic features, bipolar depression with psychotic features, obsessive compulsive disorder, intellectual disability, organic mental disorders, or mental disorders due to a general medical condition as defined in the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5).

3. Current diagnosis or history of substance use disorder as defined in the DSM-5.

4. Current active major depressive episode (MDE) or who have had an active MDE in the past 6 months.

8. The participant has a history of cerebral ischemia, transient ischemic attack (<5 years ago), intracranial aneurysm, or arteriovenous malformation.

9. The participant has a positive test result for hepatitis B surface antigen, hepatitis C virus antibody, or human immunodeficiency virus (HIV) antibody/antigen.

10. The participant's renal creatinine clearance (Cockcroft-Gault Equation) is ≤50 mL/minute.

11. The participant has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) values >1.5 times the upper limit of normal (ULN).

12. The participant is considered by the investigator to be at imminent risk of suicide or injury to self, others, or property, or the participant has attempted suicide within the past year.

• Minimum Eligible Age: 16 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Takeda

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Sleep Disorders Center of Alabama

Birmingham, Alabama, United States

Stanford Center for Sleep Sciences and Medicine

Redwood City, California, United States

SDS Clinical Trials, Inc.

Santa Ana, California, United States

Delta Waves LLC - Hunt - PPDS, Sleep Disorder and Research Center

Colorado Springs, Colorado, United States

Florida Pediatric Research Institute

Orlando, Florida, United States

Neurotrials Research

Atlanta, Georgia, United States

Georgia Neuro Center

Gainesville, Georgia, United States

University of Kansas Medical Center Research Institute, Inc.

Kansas City, Kansas, United States

Neurocare Inc

Newton, Massachusetts, United States

Henry Ford Medical Center - Columbus

Novi, Michigan, United States

St. Lukes Sleep Medicine and Research Center

Chesterfield, Missouri, United States

Research Carolina Elite

Denver, North Carolina, United States

ARSM Research, LLC

Huntersville, North Carolina, United States

Intrepid Research

Cincinnati, Ohio, United States

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Ohio Sleep Medicine Institute

Dublin, Ohio, United States

Medical University of South Carolina - PPDS

Charleston, South Carolina, United States

Bogan Sleep Consultants, LLC

Columbia, South Carolina, United States

Comprehensive Sleep Medicine Associates - Sugar Land

Houston, Texas, United States

Sleep Therapy and Research Center

San Antonio, Texas, United States

Children's Specialty Group

Norfolk, Virginia, United States

Woolcock Institute of Medical Research, Sleep and Circadian Research Group

Glebe, New South Wales, Australia

Terveystalo Helsinki Sleep Clinic

Helsinki, Uusimaa, Finland

Hopital Pierre-Paul Riquet

Toulouse, Haute-Garonne, France

CHU Gui De Chauliac

Montpellier, Herault, France

CHU de Grenoble

La Tronche, Isere, France

Hopital de la Pitie Salpetriere

Paris, , France

Universitaet Regensburg am Bezirksklinikum

Regensburg, Bavaria, Germany

Somni Bene Institut fur Medizinische Forschung und Schlafmedizin Schwerin GmbH

Schwerin, Mecklenburg-Vorpommern, Germany

Charite - Universitatsmedizin Berlin

Berlin, , Germany

Klinische Forschung Hamburg

Hamburg, , Germany

IRCCS Istituto delle Scienze Neurologiche di Bologna, Dipartimento di Scienze Biomediche e Neuromotorie

Bellaria, Bologna, Italy

Fondazione PTV Policlinico Tor Vergata, Centro del Sonno e del Trattamento Neurologico delle Fragilta

Rome, Lazio, Italy

Istituto Neurologico Mediterraneo Neuromed, Centro Per La Diagnosi E la Cura Del Sonno

Pozzilli, Molise, Italy

Akita University Hospital

Akita, Akita, Japan

Kurume University Hospital

Kurume-Shi, Hukuoka, Japan

Howakai Kuwamizu Hospital, Chuo-Ku

Kumamoto, Kumamoto, Japan

Gokeikai Osaka Kaisei Hospital, Yodogawa-Ku

Osaka, Osaka, Japan

Koishikawa Tokyo Hospital

Bunkyo-Ku, Tokyo, Japan

National Center of Neurology and Psychiatry

Kodaira-Shi, Tokyo, Japan

Yoyogi Sleep Disorder Center

Shibuya-Ku, Tokyo, Japan

Aichi Medical University Hospital

Nagakute, , Japan

RESM respiratory and sleep medical-care clinic, Yokoharna Building, 4Floor

Yokohama, , Japan

Kempenhaeghe - PPDS

Heeze, North Brabant, Netherlands

Slaap-Waakcentrum SEIN Heemstede

Heemstede, North Holland, Netherlands

University of Oslo

Oslo, , Norway

Hospital Universitario Araba Santiago, Unidad Funcional de Sueno

Vitoria-Gasteiz, Alava, Spain

Hospital General de Castello, Sleep Unit

Castellon, Castellon, Spain

Hospital de La Ribera, Unidad de Sueno

Alzira, Valencia, Spain

Hospital Universitario Vall d'Hebron - PPDS

Barcelona, , Spain

Hospital Clinic de Barcelona, Neurology Service, scale 8-4th floor

Barcelona, , Spain

Instituto de Investigaciones del Sueno

Madrid, , Spain

Hospital Vithas Madrid Arturo Soria

Madrid, , Spain

Sahlgrenska Universitetssjukhuset

Gothenburg, Västra Götaland County, Sweden

Klinik Barmelweid AG

Barmelweid, Aargau (de), Switzerland

Neurocenter of Southern Switzerland

Lugano, Ticino (it), Switzerland

Universitaetsspital Bern, Department of Neurology

Bern, , Switzerland

Countries

United States; Australia; Finland; France; Germany; Italy; Japan; Netherlands; Norway; Spain; Sweden; Switzerland

References

Dauvilliers Y, Plazzi G, Mignot E, Lammers GJ, Del Rio Villegas R, Khatami R, Taniguchi M, Abraham A, Hang Y, Kadali H, Lamberton M, Sheikh S, Stukalin E, Neuwirth R, Swick TJ, Tanaka S, von Hehn C, von Rosenstiel P, Wang H, Cai A, Naylor M, Olsson T. Oveporexton, an Oral Orexin Receptor 2-Selective Agonist, in Narcolepsy Type 1. N Engl J Med. 2025 May 15;392(19):1905-1916. doi: 10.1056/NEJMoa2405847.

Reference Type: DERIVED

PMID: 40367374 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://clinicaltrials.takeda.com/study-detail/4c5a5e765b46449e?idFilter=%5B%22TAK-861-2001%22%5D

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

Other Identifiers

2022-001654-38

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

U1111-1277-4261

Identifier Type: OTHER

Identifier Source: secondary_id

jRCT2031220644

Identifier Type: REGISTRY

Identifier Source: secondary_id

TAK-861-2001

Identifier Type: -

Identifier Source: org_study_id