A Study to Evaluate the Efficacy and Safety of TAK-861 for the Treatment of Narcolepsy with Cataplexy
- Conditions
- Narcolepsy with Cataplexy (Narcolepsy Type 1)
- Interventions
- Drug: Placebo
- Registration Number
- 2024-511998-30-00
- Lead Sponsor
- Takeda Development Center Americas Inc.
- Brief Summary
To assess the effect of TAK-861 on EDS as measured by the Epworth Sleepiness Scale (ESS) total score.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Not specified
- Target Recruitment
- 80
Male or female participants aged 18 to 70 years, inclusive, at the time of signing the ICF.
The participant has a body mass index within the range 18 to 40 kg/m2 (inclusive).
The participant has an ICSD-3 or ICSD-3-TR diagnosis of NT1.
The participant has ≥4 partial or complete episodes of cataplexy/week (WCR).
The participant is positive for the human leukocyte antigen (HLA) genotype HLA-DQB1*06:02 or results from radioimmunoassay indicate the participant’s CSF OX/hypocretin-1 concentration is <110 pg/mL
The participant is judged by the investigator to be sufficiently healthy to participate in the study, based on clinical evaluations including laboratory safety tests, medical history, physical examination, 12-lead electrocardiogram (ECG), and vital sign measurements performed at the screening visit and before the first dose of study drug.
The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS.
The participant has had major surgery or donated or lost 1 unit of blood (approximately 500 mL) within 4 weeks before the screening visit.
The participant has a history of myocardial infarction, has a history of clinically significant hepatic disease, thyroid disease, coronary artery disease, cardiac rhythm abnormality or heart failure; or 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 (does not apply to participants with carcinoma in situ that has been resolved without further treatment or basal cell carcinoma; these participants may be included after approval by the sponsor or designee).
The participant has a clinically significant history of head injury or head trauma.
The participant has a history of epilepsy, seizure, or convulsion.
The participant has any current unstable psychiatric disorder or current active major depressive episode (MDE) or an active MDE in the past 6 months.
The participant has a current history of significant multiple or severe allergies (eg, food, drug, latex allergy) or has had an anaphylactic reaction or significant intolerance to prescription or nonprescription drugs or food.
The participant has a known hypersensitivity to any component of the formulation of TAK-861 or related compounds.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description TAK-861 TAK-861 Participants will receive TAK-861 tablets, orally, for 12 weeks. Placebo Placebo Participants will receive TAK-861-matching placebo tablets, orally, for 12 weeks.
- Primary Outcome Measures
Name Time Method Change from baseline to Week 12 in ESS total score Change from baseline to Week 12 in ESS total score
- Secondary Outcome Measures
Name Time Method Change from baseline to Week 12 in mean sleep latency from the 4 MWT wake trials. Change from baseline to Week 12 in mean sleep latency from the 4 MWT wake trials.
WCR at Week 12 WCR at Week 12
Change from baseline to Week 12 in mean number of lapses on the Change from baseline to Week 12 in mean number of lapses on the
PGI-C score at Week 12. PGI-C score at Week 12.
Change from baseline to Week 12 in NSS-CT total score Change from baseline to Week 12 in NSS-CT total score
Change from baseline to Week 12 in FINI domain scores for: Tiredness, Cognitive Functioning, Cataplexy, Social Activities, Everyday Activities, and Everyday Responsibilities. Change from baseline to Week 12 in FINI domain scores for: Tiredness, Cognitive Functioning, Cataplexy, Social Activities, Everyday Activities, and Everyday Responsibilities.
Change from baseline to Week 12 in SF-36 mental and physical component summary scores Change from baseline to Week 12 in SF-36 mental and physical component summary scores
Occurrence of at least 1 TEAE during the study including the follow-up period, as applicable Occurrence of at least 1 TEAE during the study including the follow-up period, as applicable
Trial Locations
- Locations (36)
Všeobecná fakultní nemocnice v Praze
🇨🇿Praha 2, Czechia
Fakultní nemocnice Ostrava
🇨🇿Ostrava, Czechia
Aarhus Universitetshospital
🇩🇰Aarhus N, Denmark
Centrum Medyczne Linden
🇵🇱Krakow, Poland
Tampere University Hospital
🇫🇮Tampere, Finland
Suomen Terveystalo Oy
🇫🇮Helsinki, Finland
Hospital Clinic De Barcelona
🇪🇸Barcelona, Spain
Hospital Universitario Araba
🇪🇸Vitoria, Spain
Hospital Universitari Vall D Hebron
🇪🇸Barcelona, Spain
Hospital Nuestra Senora De America
🇪🇸Madrid, Spain
Scroll for more (26 remaining)Všeobecná fakultní nemocnice v Praze🇨🇿Praha 2, CzechiaKarel ŠonkaSite contact+420224965568karel.sonka@vfn.cz