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

Narcolepsy Type 1 Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05687903
• Other study ID #: TAK-861-2001
• Secondary ID: 2022-001654-38U1
• Status: Completed
• Phase: Phase 2
• Start date: January 9, 2023
• Est. completion date: December 14, 2023

Study information

• Verified date: January 2024
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 112
• Est. completion date: December 14, 2023
• Est. primary completion date: December 14, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 16 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Narcolepsy
• Narcolepsy Type 1

Intervention

Drug:
• TAK-861
TAK-861 tablets
• Placebo
TAK-861 placebo matching tablets

Locations

Country	Name	City	State
Australia	Woolcock Institute of Medical Research, Sleep and Circadian Research Group	Glebe	New South Wales
Finland	Terveystalo Helsinki Sleep Clinic	Helsinki	Uusimaa
France	CHU de Grenoble	La Tronche	Isere
France	CHU Gui De Chauliac	Montpellier	Herault
France	Hopital de la Pitie Salpetriere	Paris
France	Hopital Pierre-Paul Riquet	Toulouse	Haute-Garonne
Germany	Charite - Universitatsmedizin Berlin	Berlin
Germany	Klinische Forschung Hamburg	Hamburg
Germany	Universitaet Regensburg am Bezirksklinikum	Regensburg	Bayern
Germany	Somni Bene Institut fur Medizinische Forschung und Schlafmedizin Schwerin GmbH	Schwerin	Mecklenburg-Vorpommern
Italy	IRCCS Istituto delle Scienze Neurologiche di Bologna, Dipartimento di Scienze Biomediche e Neuromotorie	Bellaria	Bologna
Italy	Istituto Neurologico Mediterraneo Neuromed, Centro Per La Diagnosi E la Cura Del Sonno	Pozzilli	Molise
Italy	Fondazione PTV Policlinico Tor Vergata, Centro del Sonno e del Trattamento Neurologico delle Fragilta	Roma	Lazio
Japan	Akita University Hospital	Akita-Shi	Akita
Japan	Koishikawa Tokyo Hospital	Bunkyo-Ku	Tokyo
Japan	National Center of Neurology and Psychiatry	Kodaira-Shi	Tokyo
Japan	Howakai Kuwamizu Hospital, Chuo-Ku	Kumamoto-Shi	Kumamoto
Japan	Kurume University Hospital	Kurume-Shi	Hukuoka
Japan	Aichi Medical University Hospital	Nagakute
Japan	Gokeikai Osaka Kaisei Hospital, Yodogawa-Ku	Osaka-Shi	Osaka
Japan	Yoyogi Sleep Disorder Center	Shibuya-Ku	Tokyo
Japan	RESM respiratory and sleep medical-care clinic, Yokoharna Building, 4Floor	Yokohama
Netherlands	Slaap-Waakcentrum SEIN Heemstede	Heemstede	Noord-Holland
Netherlands	Kempenhaeghe - PPDS	Heeze	Noord-Brabant
Norway	University of Oslo	Oslo
Spain	Hospital de La Ribera, Unidad de Sueno	Alzira	Valencia
Spain	Hospital Clinic de Barcelona, Neurology Service, scale 8-4th floor	Barcelona
Spain	Hospital Universitario Vall d'Hebron - PPDS	Barcelona
Spain	Hospital General de Castello, Sleep Unit	Castellón De La Plana	Castellon
Spain	Hospital Vithas Madrid Arturo Soria	Madrid
Spain	Instituto de Investigaciones del Sueno	Madrid
Spain	Hospital Universitario Araba Santiago, Unidad Funcional de Sueno	Vitoria	Alava
Sweden	Sahlgrenska Universitetssjukhuset	Goteborg	Vastra Gotalands Lan
Switzerland	Klinik Barmelweid AG	Barmelweid	Aargau (de)
Switzerland	Universitaetsspital Bern, Department of Neurology	Bern
Switzerland	Neurocenter of Southern Switzerland	Lugano	Ticino (it)
United States	Neurotrials Research	Atlanta	Georgia
United States	Sleep Disorders Center of Alabama	Birmingham	Alabama
United States	Medical University of South Carolina - PPDS	Charleston	South Carolina
United States	St. Lukes Sleep Medicine and Research Center	Chesterfield	Missouri
United States	Intrepid Research	Cincinnati	Ohio
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	Delta Waves LLC - Hunt - PPDS, Sleep Disorder and Research Center	Colorado Springs	Colorado
United States	Bogan Sleep Consultants, LLC	Columbia	South Carolina
United States	Research Carolina Elite	Denver	North Carolina
United States	Ohio Sleep Medicine Institute	Dublin	Ohio
United States	Georgia Neuro Center	Gainesville	Georgia
United States	Comprehensive Sleep Medicine Associates - Sugar Land	Houston	Texas
United States	ARSM Research, LLC	Huntersville	North Carolina
United States	University of Kansas Medical Center Research Institute, Inc.	Kansas City	Kansas
United States	Neurocare Inc	Newton	Massachusetts
United States	Children's Specialty Group	Norfolk	Virginia
United States	Henry Ford Medical Center - Columbus	Novi	Michigan
United States	Florida Pediatric Research Institute	Orlando	Florida
United States	Stanford Center for Sleep Sciences and Medicine	Redwood City	California
United States	Sleep Therapy and Research Center	San Antonio	Texas
United States	SDS Clinical Trials, Inc.	Santa Ana	California

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline to Week 8 in Mean Sleep Latency from the Maintenance of Wakefulness Test (MWT)	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.	Baseline, Week 8
Secondary	Change from Baseline to Week 8 in Epworth Sleepiness Scale (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 8
Secondary	Weekly Cataplexy Rate at Week 8		Week 8
Secondary	Occurrence of at Least One Related 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.	Baseline up to approximately 16 weeks

External Links

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