A Study of TAK-994 in Adults With Type 1 and Type 2 Narcolepsy

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2)

In brief

The main aims of the study are: * To check for side effects from TAK-994 and check what dose of TAK-994 participants can tolerate. * To check what dose range provides adequate relief of narcolepsy symptoms. * To check how much TAK-994 stays in the blood of participants, over time. The study will have 4 parts. Participants can only join 1 of the parts. A. Participants with type 1 narcolepsy will take either TAK-994 or placebo tablets for 28 days. A placebo looks just like TAK-994 but will not have any medicine in it. B. Participants with type 1 narcolepsy will take 1 of 3 doses of TAK-994 or placebo tablets for 56 days. C. Participants with type 1 narcolepsy in China only will take TAK-994 or placebo tablets for 56 days. D. Participants with type 2 narcolepsy will take either TAK-994 or placebo tablets for 28 days.

Key facts

Study ID

NCT04096560

Run by

Takeda

People needed

97

Starts

2020-05-27

Expected to finish

2021-11-05

Last updated by the study team

2024-10-29

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Has a diagnosis of narcolepsy type 1 (NT1) (Parts A-C) or NT2 (Part D) by polysomnography (PSG)/ multiple sleep latency test (MSLT) performed within the past 10 years meeting the minimal acceptable criteria for the proper performance of the PSG/MSLT as outlined by the International Classification of Sleep Disorders, 3rd edition criteria.
• The participant's Epworth Sleepiness Scale (ESS) score must be greater than or equal to (>=) 10 at Day -1.
• Must be willing to discontinue all medications used for the treatment of NT1/NT2.
• The human leukocyte antigen (HLA) genotype: Part A: should test positive for human leukocyte antigen (HLADQB1)* 06:02 (PARTs A-C)- (positive results for either homozygous or heterozygous alleles will be considered "positive" and acceptable). However, if the HLA test is negative (i.e. negative for the heterozygous allele) and the PI feels strongly that the participant has narcolepsy with cataplexy (NT1) then a discussion should be initiated between the PI and the sponsor or designee about the advisability of doing a spinal tap to determine the participant's cerebrospinal fluid (CSF) orexin-1 (OX-1) level. If the CSF result shows the orexin 1 (OX-1) concentration is either less than or equal to<110 pg/mL, or less than one-third of mean values obtained in normal participants with the same standardized assay, then the diagnosis of NT1 is established allowing the participant to be enrolled and randomized, If the CSF OX-1 concentration is >110 pg/mL then the participant will not be allowed to continue in the study .
• For Parts A, B, and C, during the screening period, participant, must have >=4 partial or complete episodes of cataplexy/week (WCR), and >=4 partial or complete episodes of cataplexy/week during the screening period when off of anticataplexy medications, averaged over 2 weeks (14 consecutive days) minimum. WCR recording taken during following period will be considered for study eligibility: after the participant has stopped taking anticataplexy medications for at least 7 days (minimum 7-day washout) and study Day -2.

You may not qualify if…

• Has a risk of suicide according to endorsement of Item 4 or 5 of the screening/baseline visit Columbia suicide severity rating scale (C-SSRS) or has made a suicide attempt in the previous 12 months.
• Is an excessive (>600 mg/day) caffeine user 1 week before to the study screening.
• Has a history of cancer (except carcinoma in situ that has been resolved without further treatment or basal cell skin cancer); past or current epilepsy, seizure; a lifetime history of major psychiatric disorder other than depression or anxiety; a clinically significant history of head injury or head trauma; a history of cerebral ischemia, transient ischemic attack, intracranial aneurysm, or arteriovenous malformation; known coronary artery disease, a history of myocardial infarction, angina, cardiac rhythm abnormality, or heart failure; or current or recent (within 6 months) gastrointestinal disease expected to influence the absorption of drugs. 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 participants.
• Has a medical disorder, other than narcolepsy, associated with EDS. This includes clinically significant moderate to severe obstructive sleep apnea and/or with or without treatment with mandibular advanced device hypoglossal nerve stimulation and/or positive airway pressure (PAP) therapy) and/or restless legs syndrome (RLS)/periodic limb movement disorder that has a significant impact on daytime sleepiness. This is evidenced by a clinical history of sleep apnea syndrome (loud snoring with observed respiratory pauses in the absence of nPSG) and/or RLS causing historical sleep onset/maintenance insomnia with resultant insufficient sleep. Or any as evaluated during the clinical interview at screening. pPast PSG data demonstrating any of the following sleep disturbances: apnea Hypopnea Index ≥15 or apnea index ≥10, an oxygen saturation of <80 for >10 seconds, periodic leg movement arousal index of ≥15/h) or as evaluated on interview at the time of screening. Asshould be considered exclusionary unless, based on a clinical evaluation by the investigator, a meaningful change in clinical status has occurred that would impact the results. Because nPSG data is obtained on Day -2, subjects may fail screening if criteria are not meet on the Day -2 nPSG.
• Has a usual bedtime later than 2400 (12:00 AM, midnight) or an occupation requiring nighttime shift work or variable shift work within the past 6 months or travel within more than 3 time zones, within 14 days before Study Day -2.
• Has a nicotine dependence that is likely to have an effect on sleep (e.g., a participant who routinely awakens at night to smoke) and/or an unwillingness to discontinue all smoking and nicotine use during the confinement portions of the study. Participants undergoing optional CSF collection.
• Has a local infection at the puncture site.
• Has developed signs of lumbar radiculopathy, including lower extremity pain and paresthesia.
• Has any known focal neurological deficit that might suggest an increase in intracranial pressure.

Where it is running

• Mayo Clinic Arizona — Phoenix, Arizona, United States
• CITrials - Bellflower — Bellflower, California, United States
• Santa Monica Clinical Trials — Los Angeles, California, United States
• Stanford School of Medicine — Redwood City, California, United States
• Pacific Research Network, Inc — San Diego, California, United States
• SDS Clinical Trials, Inc. — Santa Ana, California, United States
• Alpine Clinical Research Center — Boulder, Colorado, United States
• Delta Waves Sleep Disorders and Research Center — Colorado Springs, Colorado, United States
• St. Francis Medical Institute — Clearwater, Florida, United States
• Sleep Medicine Specialists of South Florida — Miami, Florida, United States
• Clinical Trials of Florida — Miami, Florida, United States
• JSV Clinical Research Study, Inc — Tampa, Florida, United States
• Florida Pulmonary Research Institute, LLC — Winter Park, Florida, United States
• NeuroTrials Research, Inc. — Atlanta, Georgia, United States
• iResearch Atlanta, LLC — Decatur, Georgia, United States
• Sleep Practitioners, LLC — Macon, Georgia, United States
• Global Research Associates — Stockbridge, Georgia, United States
• Hawaii Pacific Neuroscience — Honolulu, Hawaii, United States
• Lutheran Sleep Disorder Center — Fort Wayne, Indiana, United States
• University of Kansas Medical Center Research Institute, Inc. — Kansas City, Kansas, United States
• Helene A. Emsellem, MD PC trading as "The Center for Sleep & Wake Disorders" — Chevy Chase, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Neurocare, Inc., dba Neurocare Center for Research — Newton, Massachusetts, United States
• Research Carolina Elite LLC — Denver, North Carolina, United States
• Wright Clinical Research — Alabaster, Alabama, United States

Full record on ClinicalTrials.gov

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