Narcolepsy Type 1 (NT1)Phase 2NCT04096560
A Study of TAK-994 in Adults With Type 1 and Type 2 Narcolepsy
A Phase 2 study testing TAK-994 in people with Narcolepsy Type 1 (NT1) and Narcolepsy Type 2 (NT2), run by Takeda at 78 sites in 12 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2)
- What is being tested?
- TAK-994
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 97 participants
- Where
- 78 sitesin 12 countries
- Study dates
- 27 May 2020to 5 Nov 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
78 sites in 12 countries.
78 sites in 12 countries
United States36 sites
Wright Clinical Research
Alabaster, Alabama, 35007
NeuroTrials Research, Inc.
Atlanta, Georgia, 30328
CITrials - Bellflower
Bellflower, California, 90706
Boston Children's Hospital
Boston, Massachusetts, 02115
Alpine Clinical Research Center
Boulder, Colorado, 80301
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425
Helene A. Emsellem, MD PC trading as "The Center for Sleep & Wake Disorders"
Chevy Chase, Maryland, 20815
CTI Clinical Research Center
Cincinnati, Ohio, 45245
Intrepid Research
Cincinnati, Ohio, 45245
St. Francis Medical Institute
Clearwater, Florida, 33765
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Delta Waves Sleep Disorders and Research Center
Colorado Springs, Colorado, 80918
Bogan Sleep Consultants, LLC
Columbia, South Carolina, 29201
iResearch Atlanta, LLC
Decatur, Georgia, 30030
Research Carolina Elite LLC
Denver, North Carolina, 28037
Ohio Sleep Medicine and Neuroscience Institute
Dublin, Ohio, 43017
Lutheran Sleep Disorder Center
Fort Wayne, Indiana, 46804
Clinical Research of Gastonia
Gastonia, North Carolina, 28054
Hawaii Pacific Neuroscience
Honolulu, Hawaii, 96817
University of Kansas Medical Center Research Institute, Inc.
Kansas City, Kansas, 66160
Santa Monica Clinical Trials
Los Angeles, California, 90025
Sleep Practitioners, LLC
Macon, Georgia, 31210
Sleep Medicine Specialists of South Florida
Miami, Florida, 33126
Clinical Trials of Florida
Miami, Florida, 33186
Neurocare, Inc., dba Neurocare Center for Research
Newton, Massachusetts, 02459
Mayo Clinic Arizona
Phoenix, Arizona, 85054
Raleigh Neurology Associates
Raleigh, North Carolina, 27607
Stanford School of Medicine
Redwood City, California, 94063
Sleep Therapy & Research Center
San Antonio, Texas, 78229
Pacific Research Network, Inc
San Diego, California, 92128
SDS Clinical Trials, Inc.
Santa Ana, California, 92705
Global Research Associates
Stockbridge, Georgia, 30281
Comprehensive Sleep Medicine Associates
Sugar Land, Texas, 77478
JSV Clinical Research Study, Inc
Tampa, Florida, 33624
Florida Pulmonary Research Institute, LLC
Winter Park, Florida, 32789
Respiratory Specialists
Wyomissing, Pennsylvania, 19610
Japan15 sites
Koishikawa Tokyo Hospital
Bunkyō City, Tokyo-To, 112-0012
SOUSEIKAI PS Clinic
Fukuoka, Fukuoka, 812-0025
Jinyukai Kotorii Isahaya Hospital
Isahaya-shi, Nagasaki, 854-0081
You Ariyoshi Sleep Clinic
Kitakyushu-shi, Fukuoka, 802-0084
Howakai Kuwamizu Hospital
Kumamoto, Kumamoto, 862-0954
Kurume University Hospital
Kurume-shi, Fukuoka, 830-0011
Shunkaikai Inoue Hospital
Nagasaki, Nagasaki, 850-0045
Gokeikai Osaka Kaisei Hospital
Osaka, Osaka, 532-0003
Kyowakai Hannan Hospital
Sakai-shi, Osaka, 599-8263
Yoyogi Sleep Disorder Center
Shibuya-ku, Tokyo-To, 151-0053
Sleep Support Clinic
Shinagawa-ku, Tokyo-To, 140-0011
Sleep & Stress Clinic
Shinagawa-ku, Tokyo-To, 141-6003
Sumida Hospital
Sumida-ku, Tokyo-To, 130-0004
Nihon University Itabashi Hospital
tabashi City, Tokyo-To, 173-8610
Kaiseikai Kita Shin Yokohama Internal Medicine Clinic
Yokohama, Kanagawa, 223-0059
China4 sites
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, 100053
The First Hospital of Jilin University
Changchun, Jilin, 130021
Huashan Hospital, Fudan University
Shanghai, Shanghai Municipality, 200040
The First Affiliated Hospital of Jinan University
Tianhe, Guangdong, 510630
Italy4 sites
Universita di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
Bologna, 40123
Ospedale San Raffaele (San Raffaele Turro)
Milan, 20127
Azienda Ospedaliera Universitaria Policlinico Tor Vergata
Roma, 133
IRCCS Oasi Maria SS
Troina, Enna, 94018
Spain4 sites
Hospital Clinic de Barcelona
Barcelona, 8036
Hospital General de Castellon
Castellon, Castellon, 12004
Hospital Vithas Nuestra Senora de America
Madrid, 28043
Hospital Universitario Araba Sede Santiago
Vitoria-Gasteiz, Alava, 1004
Canada3 sites
West Ottawa Sleep Centre
Ottawa, Ontario, K2A3Z8
Toronto Sleep Institute
Toronto, Ontario, M4P 1P2
Jodha Tishon Inc.
Toronto, Ontario, M5S 3A3
Czechia3 sites
Fakultni nemocnice Hradec Kralove
Hradec Králové, 50333
Fakultni nemocnice Ostrava
Ostrava, 708 52
Vseobecna fakultni nemocnice v Praze
Prague, 128 21
Finland2 sites
Terveystalo Helsinki Uniklinikka
Helsinki, 380
Turku University Hospital
Turku, 20521
France2 sites
Hopital Roger Salengro - CHU Lille
Lille, Nord, 59037
Hopital Gui de Chauliac
Montpellier, Herault, 34295
Netherlands2 sites
Stichting Epilepsie Instelling Nederland, Heemstede
Heemstede, 2103 SW
Kempenhaeghe, Heeze
Heeze, 5591 VE
South Korea2 sites
Keimyung University Dongsan Hospital
Daegu, 42601
The Catholic University of Korea, St. Vincent's Hospital
Suwon, Gyeonggi-do, 16247
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Rising Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of TAK-994 in Patients With Narcolepsy With or Without Cataplexy (Narcolepsy Type 1 or Narcolepsy Type 2)
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.
Read more
The study will have 4 parts. Participants can only join 1 of the parts.
- 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.
- Participants with type 1 narcolepsy will take 1 of 3 doses of TAK-994 or placebo tablets for 56 days.
- Participants with type 1 narcolepsy in China only will take TAK-994 or placebo tablets for 56 days.
- Participants with type 2 narcolepsy will take either TAK-994 or placebo tablets for 28 days.
Read the detailed description
The drug being tested in this study is called TAK-994. TAK-994 is being tested in participants with NT1 and NT2.
The study will enroll up to approximately 202 participants. The study has 4 Parts: Parts A, B, C (China only) and D. Part A - Part A has 2 cohorts [Cohorts (A1a and A1b) A2] In both of these Cohorts, participants will be randomly assigned (by chance, like flipping a coin) in a 2:1 ratio to receive TAK-994 or placebo up to 28 days:
- Part B: In Part B, participants will be randomized in 1:1:1:1 ratio in four parallel arms to receive TAK-994 Dose 1, 2 or 3 or placebo for 56 days. Depending upon their eligibility participants completing Part B of the study treatment will be enrolled to participate in an Extension study.
- Part C: In Part C, participants only from China will be enrolled and randomized in a 2:1 ratio to receive TAK-994 and placebo for 56 days.
- Part D: Participants will be included in two cohorts [Cohorts (D1a and D1b) and D2] and will be randomized in 2:1 ratio to receive TAK-994 or placebo for 28 days. The dose will be selected based on the safety and tolerability in Part A.
This multi-center trial will be conducted in the United States, Japan, China, Italy, France, and European Union. The overall duration of the study is 63 days. Participants will be followed up for 7 days after the last dose of study drug.
- Conditions
- Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2)
- Being tested
- TAK-994, Placebo
- Keywords
- Drug therapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TAK-994Drug
Also called Firazorexton
TAK-994 tablets.
PlaceboDrug
TAK-994 placebo-matching tablets.
| Study group | Role | What they receive |
|---|---|---|
| Part A: Placebo | Placebo comparator | TAK-994 placebo-matching tablets, orally, twice daily (BID) for 28 days, in participants with NT1. Receives: Drug: Placebo |
| Part A: TAK-994 120 mg | Experimental | TAK-994 120 mg, orally, BID for 28 days, in participants with NT1. Receives: Drug: TAK-994 |
| Part A: TAK-994 180 mg | Experimental | TAK-994 180 mg, orally, BID for 28 days, in participants with NT1. Receives: Drug: TAK-994 |
| Part B: Placebo | Placebo comparator | TAK-994 placebo-matching tablets, orally, BID for 56 days, in participants with NT1. Receives: Drug: Placebo |
| Part B: TAK-994 30 mg | Experimental | TAK-994 30 mg tablets, orally, BID for 56 days, in participants with NT1. Receives: Drug: TAK-994 |
| Part B: TAK-994 90 mg | Experimental | TAK-994 90 mg tablets, orally, BID for 56 days, in participants with NT1. Receives: Drug: TAK-994 |
| Part B: TAK-994 180 mg | Experimental | TAK-994 180 mg tablets, orally, BID for 56 days, in participants with NT1. Receives: Drug: TAK-994 |
| Part C: TAK-994 180 mg | Experimental | TAK-994 180 mg tablets, orally, BID for 56 days, in Chinese participants with NT1. Receives: Drug: TAK-994 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Number of Participants Who Experience at Least One Treatment Emergent Adverse Event (TEAE) During the Study
An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as an AE with an onset that occurs after receiving study drug.
Measured: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
Part A: Number of Participants Who Meet the Markedly Abnormal Value Criteria for Safety Laboratory Tests at Least Once Postdose During the Study
Standard safety laboratory values (hematology, serum chemistry, urinalysis) were collected and compared to pre-specified criteria for markedly abnormal values. Markedly abnormal values criteria: Erythrocytes(10^12/L:<0.8xlower limit of normal(LLN), >1.2xupper limit of normal(ULN); Hemoglobin grams per litre(g/L):<0.8xLLN, >1.2xULN; Hematocrit voltage/volts(V/V):<0.8xLLN, >1.2xULN; Platelets(10^9/L):<75, >600; Leukocytes(10^9/L):<0.5xLLN, >1.5xULN; Alanine Aminotransferase units/litre(U/L):>3xULN, Aspartate Aminotransferase(U/L):>3xULN; Bilirubin micromoles/litre (umol/L):>1.5xULN; Alkaline Phosphatase(U/L):>3xULN; Gamma Glutamyl Transferase(U/L):>3 x ULN; Albumin(g/L):<25; Protein Total(g/L):<0.8xLLN, >1.2xULN;Glucose millimoles/litre(mmol/L):<2.8, >19.4; Calcium(mmol/L):<1.92, >2.77; Creatinine(umol/L):>1.5xULN; Urea(mmol/L):>10.7; Sodium(mmol/L):<130, >150; Potassium(mmol/L):<3.0, >5.3. Only categories with at least one participant with event are reported.
Measured: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
Part A: Number of Participants Who Meet the Markedly Abnormal Value Criteria for Vital Sign Measurements at Least Once Postdose During the Study
Vital signs (body temperature and sitting blood pressure) were collected and compared to pre-specified criteria for markedly abnormal values throughout the study. Markedly abnormal values criteria: Heart Rate (beats/min): <40, >115; Systolic Blood Pressure millimeters of mercury (mmHg): <90, ≥160, Change from Pre-Dose >20, Change from Pre-Dose >30, Time-matched Change from Baseline > 20, Time-matched Change from Baseline > 30; Diastolic Blood Pressure (mmHg): <50, ≥100, Change from Pre-Dose >20, Change from Pre-Dose >30, Time-matched Change from Baseline > 20, Time-matched Change from Baseline > 30; Respiratory Rate (breaths/min): >21; Temperature Celsius (C): >38.5. Only categories with at least one participant with event are reported.
Measured: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
Part A: Number of Participants Who Meet the Markedly Abnormal Value Criteria for Safety Electrocardiogram (ECG) Parameters at Least Once Postdose During the Study
A 12 lead ECG was performed, the ECG values were compared to pre-specified criteria for markedly abnormal values. Markedly abnormal values criteria: ECG Mean Heart Rate (beats/min): <40, >115; PR Interval milliseconds (msec): ≤80, ≥200; corrected QT interval by Fredericia (QTcF) Interval (msec): ≤300, >500, ≥30 change from baseline and >450; QRS Duration (msec): ≤80, ≥180. Only categories with at least one participant with event are reported.
Measured: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
Parts B and C: Change From Baseline in Average Sleep Latency as Assessed by the Maintenance of Wakefulness Test (MWT)
MWT: validated, objective measure that evaluates person's ability to remain awake under soporific conditions for defined period. During each MWT session (1 session=40 minutes), participants were instructed to sit quietly and remain awake for as long as possible. Sleep latency in each session was recorded on electroencephalography (EEG). If no sleep had been observed according to these rules, then latency= 40 minutes. Mixed-effect model for repeated measures (MMRM) was used for analysis. Due to early termination of the study, no post-baseline efficacy data for this outcome measure was collected and analyzed for participants in Part C: TAK-994 180 mg.
Measured: Baseline and Week 8 (Day 56) in Parts B and C
Secondary outcomes (12)
Part A: Cmax: Maximum Observed Plasma Concentration After Single Dose of TAK-994 at Day 1
Measured: Pre-dose and at multiple time points (up to 14 hours) post-dose at Day 1 in Part A
Part A: Tmax: Time of First Occurrence of Cmax After Single Dose of TAK-994 at Day 1
Measured: Pre-dose and at multiple time points (Up to 14 hours) post-dose at Day 1 in Part A
Part A: AUC(0-last): Area Under the Concentration-time Curve From Time 0 to Time of the Last Quantifiable Concentration After Single Dose of TAK-994 at Day 1
Measured: Pre-dose and at multiple time points (up to 24 hours) post-dose at Day 1 in Part A
Part A: Cmax: Maximum Observed Plasma Concentration After Multiple Doses of TAK-994 at Day 28
Measured: Pre-dose and at multiple time points (up to 14 hours) post-dose at Day 28 in Part A
Part A: Tmax: Time of First Occurrence of Cmax After Multiple Doses of TAK-994 at Day 28
Measured: Pre-dose and at multiple time points (up to 14 hours) post-dose at Day 28 in Part A
Part A: AUC(0-t): Area Under the Concentration-time Curve From Time 0 to Time Tau Over a Dosing Interval of TAK-994 at Day 28
Measured: Pre-dose and at multiple time points (Up to 12 hours) post-dose at Day 28 in Part A
Parts B and C: Change From Baseline in the Epworth Sleepiness Scale (ESS) Total Score to Week 8
The ESS is a subjective, self-administered, validated scale (scored 0 to 3) to respond to each of the 8 questions of daily life that asks participants how likely they are to fall asleep in those situations. 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 the normal range. MMRM was used for analysis. Due to early termination of the study, no post-baseline secondary efficacy data for this outcome measure was collected and analyzed for participants in Part C: TAK-994 180 mg.
Measured: Baseline and Week 8 (Day 56) in Parts B and C
Parts B and C: Change From Baseline in Weekly Cataplexy Rate (WCR) at Week 8
Participants will complete a daily patient-reported sleep diary to record self-reported narcolepsy symptoms. Participants will record episodes of cataplexy in the diary. The total number of events averaged for a week will be reported. WCR = (Total number of cataplexy over a number of non-missing diary days for a given duration/number of Non-missing diary days in that duration)*7. MMRM was used for the analysis. Due to early termination of the study, no secondary efficacy data was collected and analyzed for participants in Part C: TAK-994 180 mg.
Measured: Baseline and Week 8 (Day 56) in Parts B and C
Parts B and C: Number of Participants Who Experience at Least 1 TEAE During the Study
An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as an AE with an onset that occurs after receiving study drug.
Measured: First dose of study drug to end of study follow-up (up to Day 63) in Parts B and C
Parts B and C: Number of Participants Who Met the Markedly Abnormal Value Criteria for Safety Laboratory Tests at Least Once Postdose During the Study
Standard safety laboratory values (serum chemistry, hematology, and urine analysis) were collected and compared to pre-specified criteria for Markedly Abnormal Values throughout the study. Markedly abnormal values criteria: Alanine Aminotransferase (U/L):>3xULN, Aspartate Aminotransferase(U/L):>3xULN; Bilirubin micromoles/litre (umol/L):>1.5xULN; Calcium(mmol/L):<1.92, >2.77; Potassium(mmol/L):<3.0, >5.3. Only categories with at least one participant with event are reported.
Measured: Up to Day 63 in Parts B and C
Parts B and C: Number of Participants Who Met the Markedly Abnormal Value Criteria for Vital Sign Measurements at Least Once Postdose During the Study
Vital signs (body temperature and sitting blood pressure) were collected and compared to pre-specified criteria for markedly abnormal values throughout the study. Markedly abnormal values criteria: Systolic Blood Pressure millimeters of mercury (mmHg): <90, ≥160, Change from Pre-Dose >20, Change from Pre-Dose >30, Time-matched Change from Baseline > 20, Time-matched Change from Baseline > 30; Diastolic Blood Pressure (mmHg): <50, ≥100, Change from Pre-Dose >20, Change from Pre-Dose >30, Time-matched Change from Baseline > 20, Time-matched Change from Baseline > 30; Respiratory Rate (breaths/min): >21.Only categories with at least one participant with event are reported.
Measured: Up to Day 63 in Parts B and C
Parts B and C: Number of Participants Who Met the Markedly Abnormal Value Criteria for ECG Parameters at Least Once Postdose During the Study
A 12 lead ECG was performed, the ECG values were compared to pre-specified criteria for markedly abnormal values. Markedly abnormal values criteria: PR Interval (msec): ≤80, ≥200; QRS Duration (msec): ≤80, ≥180. Only categories with at least one participant with event are reported.
Measured: Up to Day 63 in Parts B and C
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2019
- Study start27 May 2020
- Primary completion5 Nov 2021
- Study completion5 Nov 2021
Why it stopped: A safety signal has emerged in Phase 2 studies of TAK-994. As an immediate precautionary measure, Takeda has suspended dosing of patients and has decided to stop Phase 2 studies early.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Narcolepsy Type 1 (NT1)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 97 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04096560
- Sponsor's protocol ID
- TAK-994-1501
- Other IDs
- JapicCTI-205178, 2020-000777-24, U1111-1240-0346
- Status last verified
- Oct 2024
- First submitted
- 18 Sep 2019
- Last updated
- 29 Oct 2024
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Publications
Dauvilliers Y, Mignot E, Del Rio Villegas R, Du Y, Hanson E, Inoue Y, Kadali H, Koundourakis E, Meyer S, Rogers R, Scammell TE, Sheikh SI, Swick T, Szakacs Z, von Rosenstiel P, Wu J, Zeitz H, Murthy NV, Plazzi G, von Hehn C. Oral Orexin Receptor 2 Agonist in Narcolepsy Type 1. N Engl J Med. 2023 Jul 27;389(4):309-321. doi: 10.1056/NEJMoa2301940.
PubMed 37494485
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Multiple Rising Oral Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of TAK-994 in Patients With Narcolepsy With or Without Cataplexy (Narcolepsy Type 1 or Narcolepsy Type 2)
- ClinicalTrials.gov ID
- NCT04096560
- Acronym / sponsor ID
- TAK-994-1501
- Conditions
- Narcolepsy Type 1 (NT1), Narcolepsy Type 2 (NT2)
- Keywords
- Drug therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: A safety signal has emerged in Phase 2 studies of TAK-994. As an immediate precautionary measure, Takeda has suspended dosing of patients and has decided to stop Phase 2 studies early.
- Enrolment
- 97 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 27 May 2020 (actual)
- Primary completion
- 5 Nov 2021 (actual)
- Study completion
- 5 Nov 2021 (actual)
- First posted
- 20 Sep 2019 (actual)
- Results
- Posted 29 Oct 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
- 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.
- Participants with type 1 narcolepsy will take 1 of 3 doses of TAK-994 or placebo tablets for 56 days.
- Participants with type 1 narcolepsy in China only will take TAK-994 or placebo tablets for 56 days.
- Participants with type 2 narcolepsy will take either TAK-994 or placebo tablets for 28 days.
Detailed description
The drug being tested in this study is called TAK-994. TAK-994 is being tested in participants with NT1 and NT2.
The study will enroll up to approximately 202 participants. The study has 4 Parts: Parts A, B, C (China only) and D. Part A - Part A has 2 cohorts [Cohorts (A1a and A1b) A2] In both of these Cohorts, participants will be randomly assigned (by chance, like flipping a coin) in a 2:1 ratio to receive TAK-994 or placebo up to 28 days:
- Part B: In Part B, participants will be randomized in 1:1:1:1 ratio in four parallel arms to receive TAK-994 Dose 1, 2 or 3 or placebo for 56 days. Depending upon their eligibility participants completing Part B of the study treatment will be enrolled to participate in an Extension study.
- Part C: In Part C, participants only from China will be enrolled and randomized in a 2:1 ratio to receive TAK-994 and placebo for 56 days.
- Part D: Participants will be included in two cohorts [Cohorts (D1a and D1b) and D2] and will be randomized in 2:1 ratio to receive TAK-994 or placebo for 28 days. The dose will be selected based on the safety and tolerability in Part A.
This multi-center trial will be conducted in the United States, Japan, China, Italy, France, and European Union. The overall duration of the study is 63 days. Participants will be followed up for 7 days after the last dose of study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 8
| Arm | Type | Receives |
|---|---|---|
| Part A: Placebo | Placebo comparator | Drug: PlaceboTAK-994 placebo-matching tablets, orally, twice daily (BID) for 28 days, in participants with NT1. |
| Part A: TAK-994 120 mg | Experimental | Drug: TAK-994TAK-994 120 mg, orally, BID for 28 days, in participants with NT1. |
| Part A: TAK-994 180 mg | Experimental | Drug: TAK-994TAK-994 180 mg, orally, BID for 28 days, in participants with NT1. |
| Part B: Placebo | Placebo comparator | Drug: PlaceboTAK-994 placebo-matching tablets, orally, BID for 56 days, in participants with NT1. |
| Part B: TAK-994 30 mg | Experimental | Drug: TAK-994TAK-994 30 mg tablets, orally, BID for 56 days, in participants with NT1. |
| Part B: TAK-994 90 mg | Experimental | Drug: TAK-994TAK-994 90 mg tablets, orally, BID for 56 days, in participants with NT1. |
| Part B: TAK-994 180 mg | Experimental | Drug: TAK-994TAK-994 180 mg tablets, orally, BID for 56 days, in participants with NT1. |
| Part C: TAK-994 180 mg | Experimental | Drug: TAK-994TAK-994 180 mg tablets, orally, BID for 56 days, in Chinese participants with NT1. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TAK-994Also: FirazorextonTAK-994 tablets. | Drug | Part A: TAK-994 120 mg, Part A: TAK-994 180 mg, Part B: TAK-994 180 mg, Part B: TAK-994 30 mg, Part B: TAK-994 90 mg, Part C: TAK-994 180 mg |
| PlaceboTAK-994 placebo-matching tablets. | Drug | Part A: Placebo, Part B: Placebo |
Primary outcome
Part A: Number of Participants Who Experience at Least One Treatment Emergent Adverse Event (TEAE) During the Study
Time frame: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (example, a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A TEAE is defined as an AE with an onset that occurs after receiving study drug.
Part A: Number of Participants Who Meet the Markedly Abnormal Value Criteria for Safety Laboratory Tests at Least Once Postdose During the Study
Time frame: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
Standard safety laboratory values (hematology, serum chemistry, urinalysis) were collected and compared to pre-specified criteria for markedly abnormal values. Markedly abnormal values criteria: Erythrocytes(10^12/L:<0.8xlower limit of normal(LLN), >1.2xupper limit of normal(ULN); Hemoglobin grams per litre(g/L):<0.8xLLN, >1.2xULN; Hematocrit voltage/volts(V/V):<0.8xLLN, >1.2xULN; Platelets(10^9/L):<75, >600; Leukocytes(10^9/L):<0.5xLLN, >1.5xULN; Alanine Aminotransferase units/litre(U/L):>3xULN, Aspartate Aminotransferase(U/L):>3xULN; Bilirubin micromoles/litre (umol/L):>1.5xULN; Alkaline Phosphatase(U/L):>3xULN; Gamma Glutamyl Transferase(U/L):>3 x ULN; Albumin(g/L):<25; Protein Total(g/L):<0.8xLLN, >1.2xULN;Glucose millimoles/litre(mmol/L):<2.8, >19.4; Calcium(mmol/L):<1.92, >2.77; Creatinine(umol/L):>1.5xULN; Urea(mmol/L):>10.7; Sodium(mmol/L):<130, >150; Potassium(mmol/L):<3.0, >5.3. Only categories with at least one participant with event are reported.
Part A: Number of Participants Who Meet the Markedly Abnormal Value Criteria for Vital Sign Measurements at Least Once Postdose During the Study
Time frame: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
Vital signs (body temperature and sitting blood pressure) were collected and compared to pre-specified criteria for markedly abnormal values throughout the study. Markedly abnormal values criteria: Heart Rate (beats/min): <40, >115; Systolic Blood Pressure millimeters of mercury (mmHg): <90, ≥160, Change from Pre-Dose >20, Change from Pre-Dose >30, Time-matched Change from Baseline > 20, Time-matched Change from Baseline > 30; Diastolic Blood Pressure (mmHg): <50, ≥100, Change from Pre-Dose >20, Change from Pre-Dose >30, Time-matched Change from Baseline > 20, Time-matched Change from Baseline > 30; Respiratory Rate (breaths/min): >21; Temperature Celsius (C): >38.5. Only categories with at least one participant with event are reported.
Part A: Number of Participants Who Meet the Markedly Abnormal Value Criteria for Safety Electrocardiogram (ECG) Parameters at Least Once Postdose During the Study
Time frame: First dose of study treatment to end of study follow-up (up to Day 35) in Part A
A 12 lead ECG was performed, the ECG values were compared to pre-specified criteria for markedly abnormal values. Markedly abnormal values criteria: ECG Mean Heart Rate (beats/min): <40, >115; PR Interval milliseconds (msec): ≤80, ≥200; corrected QT interval by Fredericia (QTcF) Interval (msec): ≤300, >500, ≥30 change from baseline and >450; QRS Duration (msec): ≤80, ≥180. Only categories with at least one participant with event are reported.
Parts B and C: Change From Baseline in Average Sleep Latency as Assessed by the Maintenance of Wakefulness Test (MWT)
Time frame: Baseline and Week 8 (Day 56) in Parts B and C
MWT: validated, objective measure that evaluates person's ability to remain awake under soporific conditions for defined period. During each MWT session (1 session=40 minutes), participants were instructed to sit quietly and remain awake for as long as possible. Sleep latency in each session was recorded on electroencephalography (EEG). If no sleep had been observed according to these rules, then latency= 40 minutes. Mixed-effect model for repeated measures (MMRM) was used for analysis. Due to early termination of the study, no post-baseline efficacy data for this outcome measure was collected and analyzed for participants in Part C: TAK-994 180 mg.
12 secondary outcomes
Part A: Cmax: Maximum Observed Plasma Concentration After Single Dose of TAK-994 at Day 1
Time frame: Pre-dose and at multiple time points (up to 14 hours) post-dose at Day 1 in Part A
Part A: Tmax: Time of First Occurrence of Cmax After Single Dose of TAK-994 at Day 1
Time frame: Pre-dose and at multiple time points (Up to 14 hours) post-dose at Day 1 in Part A
Part A: AUC(0-last): Area Under the Concentration-time Curve From Time 0 to Time of the Last Quantifiable Concentration After Single Dose of TAK-994 at Day 1
Time frame: Pre-dose and at multiple time points (up to 24 hours) post-dose at Day 1 in Part A
Part A: Cmax: Maximum Observed Plasma Concentration After Multiple Doses of TAK-994 at Day 28
Time frame: Pre-dose and at multiple time points (up to 14 hours) post-dose at Day 28 in Part A
Part A: Tmax: Time of First Occurrence of Cmax After Multiple Doses of TAK-994 at Day 28
Time frame: Pre-dose and at multiple time points (up to 14 hours) post-dose at Day 28 in Part A
Part A: AUC(0-t): Area Under the Concentration-time Curve From Time 0 to Time Tau Over a Dosing Interval of TAK-994 at Day 28
Time frame: Pre-dose and at multiple time points (Up to 12 hours) post-dose at Day 28 in Part A
Parts B and C: Change From Baseline in the Epworth Sleepiness Scale (ESS) Total Score to Week 8
Time frame: Baseline and Week 8 (Day 56) in Parts B and C
Parts B and C: Change From Baseline in Weekly Cataplexy Rate (WCR) at Week 8
Time frame: Baseline and Week 8 (Day 56) in Parts B and C
Parts B and C: Number of Participants Who Experience at Least 1 TEAE During the Study
Time frame: First dose of study drug to end of study follow-up (up to Day 63) in Parts B and C
Parts B and C: Number of Participants Who Met the Markedly Abnormal Value Criteria for Safety Laboratory Tests at Least Once Postdose During the Study
Time frame: Up to Day 63 in Parts B and C
Parts B and C: Number of Participants Who Met the Markedly Abnormal Value Criteria for Vital Sign Measurements at Least Once Postdose During the Study
Time frame: Up to Day 63 in Parts B and C
Parts B and C: Number of Participants Who Met the Markedly Abnormal Value Criteria for ECG Parameters at Least Once Postdose During the Study
Time frame: Up to Day 63 in Parts B and C
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37494485Dauvilliers Y, Mignot E, Del Rio Villegas R, Du Y, Hanson E, Inoue Y, Kadali H, Koundourakis E, Meyer S, Rogers R, Scammell TE, Sheikh SI, Swick T, Szakacs Z, von Rosenstiel P, Wu J, Zeitz H, Murthy NV, Plazzi G, von Hehn C. Oral Orexin Receptor 2 Agonist in Narcolepsy Type 1. N Engl J Med. 2023 Jul 27;389(4):309-321. doi: 10.1056/NEJMoa2301940.
- : To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Takeda
78 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing TAK-994 in people with Narcolepsy Type 1 (NT1) and Narcolepsy Type 2 (NT2), run by Takeda at 78 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 29 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 78 sites in United States, Japan, China, Italy, Spain and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Number of Participants Who Experience at Least One Treatment Emergent Adverse Event (TEAE) During the Study (time frame: First dose of study treatment to end of study follow-up (up to Day 35) in Part A). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04096560, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Oct 2024.