Clinical trial NCT06470828: A Study of TAK-861 for the Treatment of Narcolepsy Type 1
Official title: 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)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Narcolepsy Type 1
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2024-06-24
Enrollment: 168
Sex: ALL
Minimum age (years): 16
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.

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 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Change From Baseline to Week 12 in Mean Sleep Latency From the 4 Maintenance of Wakefulness Test (MWT) Wake Trials
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Change From Baseline to Week 12 in ESS Total Score
timeFrame: Baseline, Week 12
measure: Weekly Cataplexy Rate (WCR) at Week 12
timeFrame: Week 12
measure: Change From Baseline to Week 12 in Mean Number of Lapses on the 3 Psychomotor Vigilance Test (PVT)
timeFrame: Baseline, Week 12
measure: Patient Global Impression of Change (PGI-C) Score at Week 12
timeFrame: Week 12
measure: Change From Baseline to Week 12 in Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score
timeFrame: Baseline, Week 12
measure: Change From Baseline to Week 12 in Functional Impacts of Narcolepsy Instrument (FINI) Domain Scores
timeFrame: Baseline, Week 12
measure: Change From Baseline to Week 12 in Short Form-36 Survey (SF-36) Mental and Physical Component Scores
timeFrame: Baseline, Week 12
measure: Number of Participants with At Least one Treatment-Emergent Adverse Event (TEAE)
timeFrame: Up to 16 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Takeda Site 48
state: Arizona
city: Redwood City
country: United States
facility: Takeda Site 39
state: California
city: Santa Ana
country: United States
facility: Takeda Site 31
state: California
city: Upland
country: United States
facility: Takeda Site 18
state: California
city: Orlando
country: United States
facility: Takeda Site 4
state: Florida
city: Atlanta
country: United States
facility: Takeda Site 3
state: Georgia
city: Boston
country: United States
facility: Takeda Site 45
state: Massachusetts
city: Saint Louis
country: United States
facility: Takeda Site 5
state: Missouri
city: Denver
country: United States
facility: Takeda Site 2
state: North Carolina
city: Cincinnati
country: United States
facility: Takeda Site 6
state: Ohio
city: Cleveland
country: United States
facility: Takeda Site 42
state: Ohio
city: Danville
country: United States
facility: Takeda Site 49
state: Pennsylvania
city: Columbia
country: United States
facility: Takeda Site 1
state: South Carolina
city: Dallas
country: United States
facility: Takeda Site 44
state: Texas
city: Norfolk
country: United States
facility: Takeda Site 10
state: Virginia
city: Ottawa
country: Canada
facility: Takeda Site 21
state: Ontario
city: Toronto
country: Canada
facility: Takeda Site 11
state: Ontario
city: Montreal
country: Canada
facility: Takeda Site 50
state: Quebec
city: Montpellier
country: France
facility: Takeda Site 25
state: Herault
city: Toulouse
country: France
facility: Takeda Site 43
state: Midi-Pyrenees
city: Lyon
country: France
facility: Takeda Site 40
city: Paris
country: France
facility: Takeda Site 29
city: Regensberg
country: Germany
facility: Takeda Site 47
state: Bayern
city: Berlin
country: Germany
facility: Takeda Site 34
city: Schwerin
country: Germany
facility: Takeda Site 26
city: Roma
country: Italy
facility: Takeda Site 32
state: Lazio
city: Bologna
country: Italy
facility: Takeda Site 30
city: Kurume-shi
country: Japan
facility: Takeda Site 16
state: Fukuka-Ken
city: Fukuoka
country: Japan
facility: Takeda Site 14
state: Fukuoka-Shi Hakata-Ku
city: Kitakyushu-shi
country: Japan
facility: Takeda Site 13
state: Fukuoka
city: Kohoku
country: Japan
facility: Takeda Site 9
state: Kanagawa
city: Kumamoto-shi
country: Japan
facility: Takeda Site 7
state: Kumamoto-Ken
city: Isahaya-shi
country: Japan
facility: Takeda Site 17
state: Nagasaki-Ken
city: Urasoe-Shi
country: Japan
facility: Takeda Site 15
state: Okinawa
city: Yodogawa
country: Japan
facility: Takeda Site 12
state: Osaka
city: Bunkyo-ku
country: Japan
facility: Takeda Site 8
state: Tokyo
city: Kodaira-shi
country: Japan
facility: Takeda Site 19
state: Tokyo
city: Shibuya-ku
country: Japan
facility: Takeda Site 20
state: Tokyo
city: Zwolle
country: Netherlands
facility: Takeda Site 28
city: Bergen
country: Norway
facility: Takeda Site 27
state: Hordaland
city: Oslo
country: Norway
facility: Takeda Site 33
city: Vitoria
country: Spain
facility: Takeda Site 37
state: Alava
city: Castello de la Plana
country: Spain
facility: Takeda Site 38
state: Castellon
city: Barcelona
country: Spain
facility: Takeda Site 35
city: Barcelona
country: Spain
facility: Takeda Site 36
city: Madrid
country: Spain
facility: Takeda Site 24
city: Valencia
country: Spain
facility: Takeda Site 41
city: Barmelweid
country: Switzerland
facility: Takeda Site 22
state: Aargau
city: Bern
country: Switzerland
facility: Takeda Site 23
city: Lugano
country: Switzerland
facility: Takeda Site 51
city: Cambridge
country: United Kingdom
facility: Takeda Site 46
city: London
country: United Kingdom
facility: Takeda Site 52
city: London
country: United Kingdom
facility: Takeda Site 53
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: