Clinical trial NCT06505031: A Study of TAK-861 in People With 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: 2024-07-17
Enrollment: 105
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 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.

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

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: Glebe
country: Australia
facility: Takeda Site 1
state: New South Wales
city: Linz
country: Austria
facility: Takeda Site 22
city: Alken
country: Belgium
facility: Takeda Site 2
city: Erpent
country: Belgium
facility: Takeda Site 3
city: Ghent
country: Belgium
facility: Takeda Site 25
city: Leuven
country: Belgium
facility: Takeda Site 24
city: Liege
country: Belgium
facility: Takeda Site 23
city: Beijing
country: China
facility: Takeda Site 4
state: Beijing
city: Zhengzhou
country: China
facility: Takeda Site 27
state: Henan
city: Shanghai
country: China
facility: Takeda Site 28
state: Shanghai
city: Guangdong
country: China
facility: Takeda Site 26
city: Helsinki
country: Finland
facility: Takeda Site 20
city: Tampere
country: Finland
facility: Takeda Site 21
city: Leon
country: France
facility: Takeda Site 16
state: Bordeaux
city: Bron
country: France
facility: Takeda Site 6
city: Marseille
country: France
facility: Takeda Site 14
city: Montpellier
country: France
facility: Takeda Site 5
city: Nantes
country: France
facility: Takeda Site 15
city: Bologna
country: Italy
facility: Takeda Site 7
city: Pozzilli
country: Italy
facility: Takeda Site 17
city: Roma
country: Italy
facility: Takeda Site 8
city: Seoul
country: Korea, Republic of
facility: Takeda Site 11
state: Daegu
city: Suwon
country: Korea, Republic of
facility: Takeda Site 10
state: Gyeonggi-do
city: Seoul
country: Korea, Republic of
facility: Takeda Site 12
city: Krakow
country: Poland
facility: Takeda Site 9
city: Barcelona
country: Spain
facility: 28003
city: Madrid
country: Spain
facility: Takeda Site 13
city: Uppsala
country: Sweden
facility: Takeda Site 19
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: