Narcolepsy with cataplexy (Narcolepsy Type 1) MedDRA version: 20.0 Level: PT Classification code 10028713 Term: Narcolepsy System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Takeda Development Center Americas, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The participant is willing and able to understand and fully comply with study procedures and requirements (including digital tools and applications), in the opinion of the investigator. 2. The participant has provided informed consent (that is, in writing, documented via a signed and dated informed consent form [ICF] and/or electronic consent) and any required privacy authorization before the initiation of any study procedures. 3. The participant is aged 18 to 70 years, inclusive, at the time of signing the ICF. 4. The participant has body mass index within the range 18 to 40 kg/m2 (inclusive) 5. The participant has an ICSD-3 diagnosis of NT1 by polysomnography (PSG)/Multiple Sleep Latency Test (MSLT), performed within the past 10 years and meeting the minimal acceptable criteria for the proper performance of PSG/MSLT as outlined in the ICSD-3. Note: If there is a potential participant with NT1 for whom a diagnostic nPSG/MSLT was performed more than 10 years ago or is not available, the site may repeat the diagnostic PSG/MSLT before Day -2. Note: For participants with results from a CSF test indicating an OX/hypocretin-1 concentration of =110 pg/mL (or less than one-third of the mean values obtained in normal participants within the same standardized assay), the PSG/MSLT requirement may be waived after a discussion with the sponsor or designee. 6. The participant has an ESS score >12 on Day -1. 7. The participant has =4 partial and/or complete episodes of cataplexy/week (WCR), calculated as the weekly average over 14 days (Days -16 to -3 of the screening period). Before the start of WCR recording, participants must complete washout of anticataplexy medications (see protocol body, Section 6.8.1 for washout requirements for specific medications). Participants must complete self-reported cataplexy questions in the ediary for at least 11 of 14 days during Days -16 to -3, to be considered compliant. In cases where the e-diary becomes unavailable, the study site may use alternative methods to collect these data with approval from the sponsor or designee. 8. The participant is positive for the HLA genotype HLA-DQB1*06:02 (positive results for either homozygous or heterozygous alleles will be considered “positive” and acceptable) or results from CSF testing indicate the participant’s CSF OX/hypocretin-1 concentration is =65 years) yes F.1.3.1 Number of subjects for this age range 15
Exclusion criteria
Exclusion criteria: 1. The participant has a current medical disorder, other than narcolepsy with cataplexy, associated with EDS. a) This includes restless legs syndrome/periodic limb movement disorder that has a significant impact on daytime sleepiness. b) Participants with clinically significant moderate-to-severe obstructive sleep apnea may be eligible if they are compliant with continuous positive airway pressure (CPAP), defined as having at least 4 hours of CPAP use per night on at least 70% of nights for approximately 1 month before randomization (assessed by machine tracking time) and have apnea hypopnea index (AHI) =10 with CPAP or other modes of positive airway pressure. c) For all participants, past PSG data demonstrating any of the following is exclusionary: AHI =15, apnea index =10, or periodic leg movement arousal index of =15/hour, unless a more recent PSG and/or clinical evaluation by the investigator indicates a meaningful change in clinical status. All attempts should be made to confirm eligibility based on Day -2 nPSG data. 2. The participant has a current medical condition such as unstable cardiovascular, pulmonary, renal, or gastrointestinal disease, that would preclude enrollment in the view of the investigator. 3. The participant has medically significant hepatic or thyroid disease. 4. The participant has current or recent (within 6 months) gastrointestinal disease that is expected to influence the absorption of drugs (ie, a history of malabsorption, esophageal reflux, peptic ulcer disease, erosive esophagitis, frequent [more than once per week] occurrence of heartburn, or any surgical intervention). Any history of Roux-en-Y gastric bypass is considered exclusionary, and any other surgical intervention that may influence the absorption of drugs should be discussed and approved by the sponsor or designee before enrolling the participant. 5. 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; these participants may be included after approval by the sponsor or designee). 6. The participant has clinically significant coronary artery disease, a history of myocardial infarction, clinically significant angina, clinically significant cardiac rhythm abnormality, or heart failure. 7. The participant has a clinically significant history of head injury or head trauma. 8. The participant has a 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). 9. The participant has one or more of the following psychiatric disorders: a) Any current unstable psychiatric disorder. b) 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, mental retardation, 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). c) Current diagnosis or history of substance use disorder as defined in the DSM-5. Note: If the history of substance use disorder is more than 12 months before baseline, the participant may be allowed to enroll in the study after consultation with the sponsor or designee. (Participant must also
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the effect of TAK-861 on excessive daytime sleepiness (EDS) as measured by sleep latency from the Maintenance of Wakefulness Test (MWT);Secondary Objective: - To assess the effect of TAK-861 on EDS as measured by the Epworth Sleepiness Scale (ESS) total score. - To assess the effect of TAK-861 on cataplexy as assessed by the weekly cataplexy rate (WCR). - To evaluate the safety and tolerability of TAK-861. ;Primary end point(s): Change from baseline to Week 8 in mean sleep latency from the MWT;Timepoint(s) of evaluation of this end point: week 8 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change from baseline to Week 8 in ESS total score - WCR at week 8 - Occurrence of at least 1 treatment-emergent adverse event (TEAE);Timepoint(s) of evaluation of this end point: Week 8 | — |
Countries
Australia, Finland, France, Germany, Italy, Japan, Netherlands, Norway, Spain, Sweden, Switzerland, United States
Contacts
Public ContactStudy Registration Call Center
Takeda Development Center Americas, Inc
Outcome results
None listed