A Trial of TAK-861 for the Treatment of Narcolepsy With Cataplexy
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age16-70
SponsorTakeda
About this trial
The main aim of this study is to assess how effective TAK-861 is for treating narcolepsy type 1 and if this effect is maintained over time. Participants will take TAK-861 for a few months and if they meet certain criteria, they will be randomly assigned (by chance, like flipping a coin) to continue taking TAK-861 or take placebo (fake medicine) for up to 4 weeks to see if their narcolepsy symptoms return.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
The participant has a body mass index (BMI) within the range 18 to 40 kilograms per square meter (kg/m²).
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.
The participant is positive for the human leukocyte antigen (HLA) genotype Major Histocompatibility Complex, Class II, DQ Beta 1 (HLA-DQB1*06:02) or results from radioimmunoassay indicate the participant's CSF OX/hypocretin-1 concentration is ≤110 pg/mL (or less than one-third of the mean values obtained in normal participants within the same standardized assay).
Disqualifiers
The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS.
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.
The participant has current or recent (within 6 months) gastrointestinal disease that is expected to influence the absorption of drugs.
The participant has a history of cancer in the past 5 years.
Trial design
Design model
Parallel
Treatments tested in this trial
TAK-861
DrugTAK-861 tablets
Placebo
DrugTAK-861-matching placebo tablets
Treatment groups
88 Participants
are divided into 2 treatment groupsGroup A: Double-blind RW Period: TAK-861Experimental treatment 1 intervention
Group B: Double-blind RW Period: PlaceboPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Time to Loss of Response in the Epworth Sleepiness Scale (ESS) Score During the Up to 4-Week RW Period
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 normal range.
Time frame
Up to 4-week RW periodSecondary outcomes
1
Change From the End of the OL Treatment Period to Week 2 of the RW Period on 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 (EEG)-derived sleep latency in the MWT. The MWT consists of four 40-minute sessions. Sleep latency in each wake trial will be recorded. Participants will be required to stay awake in between the 4 sessions.
Time frame
From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)2
Weekly Cataplexy Rate (WCR) at Week 2 of the RW Period
Time frame
Week 2 of the RW period3
Change from the End of the OL Treatment Period to Week 2 of the RW Period in Mean Number of Lapses on the Psychomotor Vigilance Test (PVT)
The PVT is a simple reaction performance task with a duration of 10 minutes that aims to measure sustained attention.
Time frame
From the end of the OL treatment period (Week 16) to Week 2 of the RW period (Week 18)4
Number of Participants Reporting Much or Very Much Worse in Patient Global Impression of Change (PGI-C) Score at Week 2 of the RW Period
The PGI-C is a participant self-rated scale to assess improvement in daytime sleepiness and overall narcolepsy symptoms. It measures change due to treatment relative to baseline on a 7-point scale ranging from a score of 1 ("very much improved") to score of 7 ("very much worse").
Time frame
Week 2 of the RW period Source ClinicalTrials.gov