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.

NCT05015673 | Completed Phase 1 FDA Drug

• 1 other identifier: SEP361-108
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 6

Brief Summary

This is a study to determine the effect of multiple doses of an investigational drug, taken by mouth, in people with Narcolepsy-cataplexy.

Conditions

Narcolepsy

Keywords

Narcolepsy; Narcolepsy-cataplexy

Outcome Measures

Primary Outcomes (2)

• Change from baseline in the total number of cataplexy attacks over the 2-week treatment period as assessed by the cataplexy diary.

  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.

  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).

  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).

  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)

  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

  Week 2

• Treatment-emergent adverse events, serious adverse events, concomitant medications, and change from baseline and results at each postbaseline visit in clinical evaluations

  From screening to the end-of-study, an average of 90 days

Study Arms (3)

SEP-363856 25mg

EXPERIMENTAL

SEP-363856 25 mg given orally

Drug: SEP363856

SEP-363856 50mg

EXPERIMENTAL

SEP-363856 50mg given orally

Drug: SEP363856

Placebo

PLACEBO COMPARATOR

Placebo given orally

Drug: Placebo

Interventions

SEP363856 DRUG

SEP-363856 25mg; SEP-363856 50mg

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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:
• 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.

Sponsors & Collaborators

• Otsuka Pharmaceutical Development & Commercialization, Inc.: lead

Study Sites (6)

SDS Clinical Trials, Inc.

Orange, California, 92868, United States

Location

Clinical Research Group of St. Petersburg

St. Petersburg, Florida, 33707, United States

Location

NeuroTrials Research Inc.

Atlanta, Georgia, 30342, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

SleepMed of South Carolina

Columbia, South Carolina, 29201, United States

Location

Todd Swick, MD PA

Houston, Texas, 77063, United States

Location

Related Publications (1)

• 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.

  PMID: 37209427 DERIVED

MeSH Terms

Conditions

Narcolepsy

Interventions

SEP-363856

Condition Hierarchy (Ancestors)

Disorders of Excessive Somnolence; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: double blind
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double-blind, Placebo-controlled, Randomized, 3-period, 3-treatment Crossover

Study Record Dates

• First Submitted: August 10, 2021
• First Posted: August 20, 2021
• Study Start: June 5, 2014
• Primary Completion: May 26, 2015
• Study Completion: May 26, 2015
• Last Updated: June 26, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be available after marketing approval in global markets, or beginning 1-3 years following article publication. There is no end date to the availability of the data.
• Access Criteria: Otsuka will share data on the Vivli data sharing platform which can be found here: https://vivli.org/ourmember/Otsuka/

Locations

US (6)