NarcolepsyPhase 1NCT05015673
This is a Study to Determine the Effect of Multiple Doses of an Investigational Drug, Taken by Mouth, in People With Narcolepsy-cataplexy. Accepting Both Males and Females Ages of 18 Years to 55 Years. This Study Will be Conducted in the US and Will Require Approximately 13 Weeks Participation.
A Phase 1 study testing SEP363856 in people with Narcolepsy, run by Otsuka Pharmaceutical Development & Commercialization, Inc. at 6 sites in United States.
Trial summary
This is a study to determine the effect of multiple doses of an investigational drug, taken by mouth, in people with Narcolepsy-cataplexy. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Narcolepsy
- What is being tested?
- SEP363856
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 6 sitesUnited States
- Study dates
- 5 Jun 2014to 26 May 2015
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- To qualify for participation, subjects must meet all of the following inclusion criteria:
- Subject must give written informed consent and privacy authorization prior to participation in the study. Female subjects of childbearing potential and male subjects must agree to contraceptive requirements outlined in the informed consent form (ICF).
- Subject must be willing and able to comply with the study procedures and visit schedules and must be able to follow verbal and written instructions.
- Male or female subject between 18 to 55 years of age, inclusive, with narcolepsy-cataplexy (confirmation of diagnosis may occur during screening period).
- Subject's body mass index (BMI) must be at least 16 kg/m2 but no more than 32 kg/m2
- Subjects on medications for cataplexy (ie, selective serotonin reuptake inhibitors [SSRIs], serotonin and norepinephrine reuptake inhibitors [SNRIs], norepinephrine reuptake inhibitors [NRIs], tricyclic antidepressants [TCAs], or Xyrem) and/or EDS associated with narcolepsy (ie, modafinil, armodafinil, or classical stimulants such as methylphenidate and amphetamine), and/or over-the-counter (OTC) medications known to affect sleep-wake functions must washout the prohibited medications during the 2 weeks (or 5 half-lives) prior to BL-1.
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- Caffeine intake should be limited to < 300 mg (including coffee, tea, and/or other caffeine-containing beverages or foods [including, but not limited to, energy drinks, sodas, and candy]) per day for 2 weeks prior to BL-1 and throughout the study (through the EOS visit).
- Subject must have a negative urine drug screen (UDS) for drugs of abuse at the screening visit
- Male subjects with female partner(s) of childbearing potential must agree to avoid fathering a child and use acceptable methods of birth control from screening until 60 days after the last study drug administration.
- Female subjects must have a negative serum pregnancy test at screening.
- Female subjects of childbearing potential must agree to avoid pregnancy and use acceptable methods of birth control from at least 60 days prior to screening and for at least 60 days after the last study drug administration.
- Subject must be judged to be in a condition of general good health (defined as the absence of any clinically relevant conditions or abnormalities as determined by the Investigator), based on screening medical and psychiatric history, physical examination,neurological examination, vital signs, clinical laboratory values (hematology, chemistry, urinalysis, coagulation studies, and thyroid panel), and a 12-lead ECG.
- Subject must be willing to stay within the clinic for the required period.
- Subject must possess an educational level and degree of understanding of English that enables them to communicate suitably with the Investigator and study coordinator.
- Subject must agree to comply with all restrictions for the required length of time
- Subject must routinely spend 7 to 9 hours/night in bed and not vary bedtime by more than 2 hours in a week as determined by the sleep diary.
Who cannot join
- To qualify for study participation, subjects must not meet any of the following criteria:
- Subject has symptoms of any sleeping disorder other than narcolepsy-cataplexy as determined by the screening sleep history form administered by site personnel.
- Subject is a rotating or third-shift worker, is expected to travel (eg, transcontinental flights over 2 time zones), during the course of the study, or has a lifestyle within the prior 3 months that is disruptive to establishing a normal sleep pattern.
- Subject does not tolerate venipuncture or has poor venous access that would cause difficulty in collecting blood samples.
- Subject has participated in an investigational drug study and received investigational drug within 30 days (or longer if the elimination half-life is known to be ≥ 150 hours) prior to the screening visit, or who is currently participating in another clinical study.
- Subject has any clinically significant unstable acute or chronic medical condition that in the opinion of the Investigator, would limit the subject's ability to complete and/or participate in the study:
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- Hematologic (including deep vein thrombosis) or bleeding disorder, renal, metabolic, endocrine, pulmonary, gastrointestinal, urologic, cardiovascular, hepatic, neurologic, or allergic disease (except for untreated, asymptomatic, seasonal allergies at the time of dosing).
- History of any allergic reaction to any medication.
- Presence or history of a medically diagnosed, clinically significant psychiatric disorder (including mental retardation).
- History of cancer or significant ongoing neoplasm.
- Disorder or history of a condition, or previous gastrointestinal surgery (eg, cholecystectomy, vagotomy, bowel resection, or any surgical procedure), that may interfere with drug absorption, distribution, metabolism, excretion, or gastrointestinal motility; a clinically significant abnormality of the hepatic or renal system; or a history of malabsorption.
- Known or suspected substance abuse/dependence within 12 months preceding Visit 1or a positive UDS for illicit substances at screening.
- Known or suspected excessive alcohol consumption exceeding 14 units/week (1 unit = 5 ounces of wine, 12 ounces of beer, or 1.5 ounces of hard liquor) within 6 months prior to the screening visit or a positive breath alcohol test at screening.
- Subject has a clinically significant abnormal 12-lead ECG that may jeopardize the subject's ability to complete the study or a screening 12-lead ECG demonstrating any one of the following: heart rate > 100 beats per minute (bpm), QRS > 120 msec, QTcF > 450 msec, or PR > 220 msec.
- Female subject who is pregnant or lactating.
- Subject answers "yes" to "Suicidal Ideation" Items 4 or 5 on the C-SSRS.
- Subject has a history of allergic reaction or has a known or suspected sensitivity to any substance that is contained in the formulation.
- Subject has any clinically significant abnormal laboratory values (hematology, serum chemistry, urinalysis, coagulation studies, and thyroid panel).
- Subject has a history of hospitalization within 45 days prior to the screening visit.
- Subject has a positive test for Hepatitis B surface antigen or Hepatitis C antibody at screening.
- Subject has a positive test for human immunodeficiency virus (HIV-1 or HIV-2) at screening, or subject is known to have tested positive for HIV.
- Subject has abnormal hepatic function tests (aspartate aminotransferase [AST] > 2 x upper limit of normal [ULN], alanine aminotransferase [ALT] > 2 x ULN, bilirubin > 2 x ULN) at screening.
- Subject has experienced significant blood loss (≥ 473 mL) or donated blood within 60 days prior to the first dose of study drug; has donated plasma within 72 hours prior to the first dose of study drug or intends to donate plasma or blood or undergo elective surgery during study participation or within 60 days after the last study visit.
- Subject consumes the equivalent of more than 10 cigarettes a day or smokes/uses nicotine-containing products from the time he/she goes to bed at night to the time he/she wakes up the next morning.
- Subject has a history of tobacco use associated with maintaining wakefulness or uses tobacco or nicotine-containing products (including pipe, cigar, patch, chewing tobacco, spray, inhaler, gum, or e-cigarette) to stay awake.
- Subject has used prescription or nonprescription drugs, vitamins, or dietary or herbal supplements including enzyme-inhibiting supplements within 30 days prior to dosing or anticipates the need for any medication during their participation in this study (exception: female subjects who are taking oral, patch, or intrauterine device [IUD] hormonal contraceptives, or progestin implant or injection). Enzyme-inducing herbal supplements (eg, Metabolife™) must be discontinued 30 days prior to the first dose of study drug. Medications for stable medical conditions that, in the opinion of the Investigator will not adversely affect a subject's participation in the study, may be allowed with prior approval of the Medical Monitor.
- Subject previously received study drug.
- Subject is a staff member or the relative of a staff member
- Subject is, in the opinion of the Investigator, unsuitable in any other way to participate in this study
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
NeuroTrials Research Inc.
Atlanta, Georgia, 30342
SleepMed of South Carolina
Columbia, South Carolina, 29201
Duke University Medical Center
Durham, North Carolina, 27710
Todd Swick, MD PA
Houston, Texas, 77063
SDS Clinical Trials, Inc.
Orange, California, 92868
Clinical Research Group of St. Petersburg
St. Petersburg, Florida, 33707
About this study
Official title: A Double-blind, Placebo-controlled, Randomized, 3-period, 3-treatment Crossover Study to Evaluate the Effect of Multiple Oral Dose Administration of SEP-363856 in Male and Female Adult Subjects With Narcolepsy-cataplexy
This is a study to determine the effect of multiple doses of an investigational drug, taken by mouth, in people with Narcolepsy-cataplexy.
Read the detailed description
This was a multi-center, double-blind, placebo-controlled, randomized, 3-way crossover study of SEP-363856 that was conducted in adult subjects with narcolepsy-cataplexy. Two oral doses of SEP-363856 (25 and 50 mg QD for 14 days) were administered and compared with matching placebo using a 6-treatment sequence, 3-period, 3-treatment, crossover design.
- Conditions
- Narcolepsy
- Being tested
- SEP363856, Placebo
- Keywords
- Narcolepsy, Narcolepsy-cataplexy
- Medical subject headings
- Narcolepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SEP363856Drug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| SEP-363856 25mg | Experimental | SEP-363856 25 mg given orally Receives: Drug: SEP363856 |
| SEP-363856 50mg | Experimental | SEP-363856 50mg given orally Receives: Drug: SEP363856 |
| Placebo | Placebo comparator | Placebo given orally Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in the total number of cataplexy attacks over the 2-week treatment period as assessed by the cataplexy diary.
Measured: Baseline and Week 2
Clinical Global Impression-Improvement (CGI-I)at the end of the 2-week treatment period to assess change in narcolepsy-cataplexy symptoms
The CGI-I scale is a standard 7-point scales that requires the clinician and subject, respectively, to assess how much the subject's overall narcolepsy-cataplexy symptoms improved or worsened by the end of the 2-week treatment period compared to baseline.
Measured: Week 2
Secondary outcomes (5)
Change from baseline in Electromyogram (EDS )at the end of the 2-week treatment period as assessed by the Mean sleep latency (mSL) from the Multiple Sleep Latency Test (MSLT).
Measured: Baseline and Week 2
Change from baseline in Excessive day time sleepiness (EDS) at the end of the 2-week treatment period as assessed by the Epworth Sleepiness Scale ( ESS).
The ESS is a self-administered questionnaire that provides subjective measure of a subject's general level of daytime sleepiness. The ESS consists of 8 items, which are rated from 0 ("would never doze") to 3 ("high chance of dozing").
Measured: Baseline and Week 2
Change from baseline at Nights 1, 13, 30, and 47 for the following sleep architecture parameters as assessed by Nocturnal polysomnography (NPSG)
- Latency to Rapid Eye Movement (REM) sleep, REM time (total minutes of REM sleep), REM percent (REM time as a percent of Total Sleep Time [TST]), and REM density (number of 3-second units per 30-second epoch with at least 1 REM).
- Latency to persistent sleep (LPS), TST, wake after sleep onset (WASO), and number of awakenings.
- NREM sleep stages N1, N2, and N3 time (total minutes in each stage) and percent (time in each stage as a percent of TST)
Measured: Baseline, Night 1, Night 13, Night 30, and Night 47
Frequency of subjects with suicidal ideation or suicidal behavior using the Columbia-Suicide Severity Rating Scale (C-SSRS) at baseline and each postbaseline visit
The C-SSRS is a tool designed to systematically assess and track suicidal adverse events (suicidal behavior and suicidal ideation)
Measured: Week 2
Treatment-emergent adverse events, serious adverse events, concomitant medications, and change from baseline and results at each postbaseline visit in clinical evaluations
Treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), concomitant medications, and change from baseline and results at each postbaseline visit in clinical evaluations (vital signs, clinical laboratory tests [hematology, serum chemistry, urinalysis, coagulation panel, and thyroid panel], and 12-lead ECGs).
Measured: From screening to the end-of-study, an average of 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2021
- Study start5 Jun 2014
- Primary completion26 May 2015
- Study completion26 May 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for NarcolepsyStudies of the same condition, open and under way.
- More trials run by Otsuka Pharmaceutical Development & Commercialization, Inc.The 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Double-blind, Placebo-controlled, Randomized, 3-period, 3-treatment Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05015673
- Sponsor's protocol ID
- SEP361-108
- Status last verified
- Jun 2024
- First submitted
- 10 Aug 2021
- Last updated
- 26 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.
Publications
Szabo ST, Hopkins SC, Lew R, Loebel A, Roth T, Koblan KS. A multicenter, double-blind, placebo-controlled, randomized, Phase 1b crossover trial comparing two doses of ulotaront with placebo in the treatment of narcolepsy-cataplexy. Sleep Med. 2023 Jul;107:202-211. doi: 10.1016/j.sleep.2023.04.019. Epub 2023 Apr 28.
PubMed 37209427
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Placebo-controlled, Randomized, 3-period, 3-treatment Crossover Study to Evaluate the Effect of Multiple Oral Dose Administration of SEP-363856 in Male and Female Adult Subjects With Narcolepsy-cataplexy
- ClinicalTrials.gov ID
- NCT05015673
- Acronym / sponsor ID
- SEP361-108
- Conditions
- Narcolepsy
- Keywords
- Narcolepsy, Narcolepsy-cataplexy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Jun 2014 (actual)
- Primary completion
- 26 May 2015 (actual)
- Study completion
- 26 May 2015 (actual)
- First posted
- 20 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a study to determine the effect of multiple doses of an investigational drug, taken by mouth, in people with Narcolepsy-cataplexy.
Detailed description
This was a multi-center, double-blind, placebo-controlled, randomized, 3-way crossover study of SEP-363856 that was conducted in adult subjects with narcolepsy-cataplexy. Two oral doses of SEP-363856 (25 and 50 mg QD for 14 days) were administered and compared with matching placebo using a 6-treatment sequence, 3-period, 3-treatment, crossover design.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Double-blind, Placebo-controlled, Randomized, 3-period, 3-treatment Crossover
| Arm | Type | Receives |
|---|---|---|
| SEP-363856 25mg | Experimental | Drug: SEP363856SEP-363856 25 mg given orally |
| SEP-363856 50mg | Experimental | Drug: SEP363856SEP-363856 50mg given orally |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo given orally |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (16 listed)
- To qualify for participation, subjects must meet all of the following inclusion criteria:
Key exclusion criteria (29 listed)
- To qualify for study participation, subjects must not meet any of the following criteria:
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 |
|---|---|---|
| SEP363856 | Drug | SEP-363856 25mg, SEP-363856 50mg |
| Placebo | Drug | Placebo |
Primary outcome
Change from baseline in the total number of cataplexy attacks over the 2-week treatment period as assessed by the cataplexy diary.
Time frame: Baseline and Week 2
Clinical Global Impression-Improvement (CGI-I)at the end of the 2-week treatment period to assess change in narcolepsy-cataplexy symptoms
Time frame: Week 2
The CGI-I scale is a standard 7-point scales that requires the clinician and subject, respectively, to assess how much the subject's overall narcolepsy-cataplexy symptoms improved or worsened by the end of the 2-week treatment period compared to baseline.
5 secondary outcomes
Change from baseline in Electromyogram (EDS )at the end of the 2-week treatment period as assessed by the Mean sleep latency (mSL) from the Multiple Sleep Latency Test (MSLT).
Time frame: Baseline and Week 2
Change from baseline in Excessive day time sleepiness (EDS) at the end of the 2-week treatment period as assessed by the Epworth Sleepiness Scale ( ESS).
Time frame: Baseline and Week 2
Change from baseline at Nights 1, 13, 30, and 47 for the following sleep architecture parameters as assessed by Nocturnal polysomnography (NPSG)
Time frame: Baseline, Night 1, Night 13, Night 30, and Night 47
Frequency of subjects with suicidal ideation or suicidal behavior using the Columbia-Suicide Severity Rating Scale (C-SSRS) at baseline and each postbaseline visit
Time frame: Week 2
Treatment-emergent adverse events, serious adverse events, concomitant medications, and change from baseline and results at each postbaseline visit in clinical evaluations
Time frame: From screening to the end-of-study, an average of 90 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2024.
- Derived from this study: PubMed 37209427Szabo ST, Hopkins SC, Lew R, Loebel A, Roth T, Koblan KS. A multicenter, double-blind, placebo-controlled, randomized, Phase 1b crossover trial comparing two doses of ulotaront with placebo in the treatment of narcolepsy-cataplexy. Sleep Med. 2023 Jul;107:202-211. doi: 10.1016/j.sleep.2023.04.019. Epub 2023 Apr 28.
- Individual participant dataSharing planned: Yes. Anonymized Individual participant data (IPD) that underlie the results of this study will be shared with researchers to achieve aims pre-specified in a methodologically sound research proposal. Small studies with less than 25 participants are excluded from data sharing.
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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.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing SEP363856 in people with Narcolepsy, run by Otsuka Pharmaceutical Development & Commercialization, Inc. at 6 sites in United States.
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 16 inclusion and 29 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: finished, as of the sponsor's last update on 26 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change from baseline in the total number of cataplexy attacks over the 2-week treatment period as assessed by the cataplexy diary. (time frame: Baseline and Week 2). The study lists 2 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 NCT05015673, 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 26 Jun 2024.