An observational study in Narcolepsy Type 1, sponsored by Takeda. Not yet recruiting. Open to participants aged 18 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-10-29.
Sponsored by Takeda · Observational
Narcolepsy is a sleep disorder that disrupts a person's regular sleep-wake cycle. It causes extreme sleepiness during the day (called excessive daytime sleepiness [EDS]), where a person may fall asleep without warning. Narcolepsy Type 1 (NT1) is a form of the condition in which people also have sudden, unexpected muscle weakness while staying conscious (called cataplexy). The usual care for NT1 focuses on managing symptoms and improving the sleep and wake cycle. It pays special attention to daytime sleepiness. Information on treatment use and related effects in adults with NT1 is limited. This study aims to collect such information and help fill this gap.
The main aim of this study is to better understand how adults with NT1 are treated in real life, why treatment changes happen and how different treatment choices (both medicines and non-medicine strategies) affect symptoms, cataplexy and quality of life over time. The study also aims to learn more about adults with NT1, how they are diagnosed, how their treatment works, and how often they need medical care. It will also record how satisfied they are with treatment and how NT1 affects their symptoms, daily life, and quality of life over time.
This study will consist of a retrospective cohort and a prospective cohort. The retrospective cohort will be comprised of participants with NT1 who previously initiated any treatment for narcolepsy in a real-world clinical setting. Data will be collected via medical chart review and a one-time cross-sectional participant survey. The prospective cohort will be comprised of participants with NT1 who initiate treatment with pitolisant or sodium oxybate, as monotherapy or combined with any other narcolepsy treatment, on the day of or within 28 days after entering the study.
157 studies on the registry are indexed under Narcolepsy; 37 are open to participants now.
This study's planned enrollment of 400 is above the median of 135 across 38 observational studies indexed under Narcolepsy.
Browse Narcolepsy studies →Takeda is the lead sponsor of 1,026 studies on the registry; 89 are open to participants now.
Of its 178 completed or terminated interventional studies of FDA-regulated products, 154 (87%) have results posted.
Counted across the registry records on this site, refreshed daily.
Participants diagnosed with NT1 and who have previously undergone pharmacologic therapy or who will initiate treatment with pitolisant or sodium oxybate, as monotherapy or combined with any other narcolepsy pharmacotherapy are eligible to participate in this study.
Criteria for the Retrospective Cohort with a Cross-Sectional Survey Component Inclusion Criteria
Exclusion Criteria
Criteria for the Prospective Cohort Inclusion criteria
Participants must meet one of the following:
Exclusion criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Data will be collected for participants with NT1 who previously initiated (within 5 years before enrollment) any treatment for narcolepsy in a real-world clinical setting via medical chart review and a one-time cross-sectional participant survey at time of entry in the study/enrollment.
Other: No intervention
Data will be collected for up to 24 weeks for participants with NT1 who plan to initiate treatment with pitolisant or sodium oxybate, as monotherapy or combined with any other narcolepsy pharmacotherapy, on the day of or within 28 days after enrollment. Eligible participants from the retrospective cohort may be enrolled in the prospective cohort. Data will be collected via electronic questionnaires called patient reported outcomes (ePROs) and electronic diaries in addition to retrospective medical chart review.
Other: No intervention
This is a non-interventional study.
Retrospective Cohort: Percentage of Participants in Each Treatment Sequence
Time frame: From diagnosis of NT1 up to enrollment (baseline) [approximately past 5 years from enrollment]
Retrospective Cohort: Percentage of Participants Who Permanently Discontinued Treatment, Switched Treatment, or Experienced a Change in Treatment Dose
Time frame: From diagnosis of NT1 up to enrollment (baseline) [approximately past 5 years from enrollment]
Prospective Cohort: Number of Participants With Permanent Discontinuation, Switch, Interruptions, or Changes in Treatment Dose and Related Reasons
Time frame: Prospective Cohort: Up to 24 weeks
Retrospective Cohort and Prospective Cohort: Average Duration on Treatment
Time frame: Retrospective Cohort: From diagnosis of NT1 up to enrollment (baseline) [approximately past 5 years from enrollment]; Prospective Cohort: Up to 24 weeks
Retrospective Cohort and Prospective Cohort: Time to Permanent Discontinuation, Switch, or Change in Treatment Dose
Time frame: Retrospective Cohort: From diagnosis of NT1 up to enrollment (baseline) [approximately past 5 years from enrollment]; Prospective Cohort: Up to 24 weeks
Prospective Cohort: Percentage of Participants on Each Pharmacotherapy Who Permanently Discontinued Treatment, Switched Treatment, or Experienced a Change in Treatment Dose
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by the Epworth Sleepiness Scale (ESS)
The ESS is a validated, subjective, self-administered questionnaire used to measure daytime sleepiness. The questionnaire consists of 8 questions, each describing a common situation (e.g., sitting and reading, watching TV, sitting in a car as a passenger). Participants rate their likelihood of dozing off in each situation on a scale from 0 (would never doze off) to 3 (high chance of dozing off). The total score ranges from 0 to 24. Higher scores indicate greater daytime sleepiness.
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by the Clinical Global Impression of Severity (CGI-S)
CGI-S is a 7-point scale used by clinicians to rate the severity of a participant's illness based on a global assessment of observed and reported symptoms, behaviour, and function over the past 7 days. It assesses the overall severity of mental illness relative to the clinician's experience with similar participants and uses a scale from "1= normal, not at all ill" to "7= among the most extremely ill participants".
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by the Clinical Global Impression of Change (CGI-C)
CGI-C is a 7-point scale that requires the clinician to assess how much the participant's illness has improved since baseline. Ratings range from "very much improved" (1) to "very much worse" (7).
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by the Patient Global Impression of Severity (PGI-S)
The PGI-S is scale for the participant to rate his/her disease severity at the time of assessment on a 5-point scale ranging from a score of 0 ("none") to 4 ("very severe").
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by the Patient Global Impression of Change (PGI-C)
The PGI-C measures change due to treatment relative to PGI-S on a 7-point scale ranging from a score of 1 ("very much improved") to a score of 7 ("very much worse").
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by the Functional Impacts of Narcolepsy Instrument (FINI)
The FINI is a participant-reported outcome (PRO) measure of 28-item, 6-domain (Tiredness, Cognitive Functioning, Cataplexy \[for NT1\], Social Activities, Everyday Activities, and Everyday Responsibilities) (FINI v1.0). FINI items evaluate narcolepsy/excessive daytime sleepiness (EDS) symptoms and impacts on daily functioning. All items have a 7-day recall period on a 5-point Likert scale of frequency from "never" to "always" or severity from "not at all" to "very much." For each domain, an average item score is calculated and standardized to a 0 to 100 scale. Lower scores indicate better health/functioning. FINI will be recorded using an ePRO.
Time frame: Up to 24 weeks
Prospective Cohort: Narcolepsy Symptoms Measured by Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score
The NSS-CT is a clinical tool designed to assess the severity and impact of narcolepsy. The scale consists of 15 items, each addressing different aspects of narcolepsy symptoms: EDS (items 1-7), cataplexy (items 8-10), hallucinations (items 11 and 12), sleep paralysis (items 13 and 14), and disturbed nighttime sleep (item 15). Participants rate the frequency and severity of their symptoms over the past week. Items will be scored on a scale where mild=0-14, moderate=15-28, severe=29-42, very severe=43-57. Higher scores indicate more severe symptoms. NSS-CT will be recorded using an ePRO.
Time frame: Up to 24 weeks
Prospective Cohort: Cataplexy Measured by Weekly Cataplexy Rate (WCR)
The WCR is a measure of the average number of cataplexy episodes a person experiences in a week. Participants will be asked to complete self-reported cataplexy questions in an e-diary daily for 14 days at each timepoint (1 week before and 1 week after each timepoint). Cataplexy symptoms will also be assessed over timepoints and may include assessment of severity and location of symptoms and impact of symptoms on daily life. WCR and cataplexy symptoms will be recorded using e-diary entries.
Time frame: Up to 24 weeks
Prospective Cohort: Health-Related Quality of Life (HRQoL) Measured by the 12-Item Short Form Health Survey (SF-12) (Physical Component Summary [PCS] and Mental Component Summary [MCS] Scores)
The SF-12 is a validated questionnaire that measures general HRQoL. It contains one multi-item scale that assesses 8 domains: limitations in physical activities because of health problems; limitations in social activities because of physical or emotional problems; limitations in usual role activities because of physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities because of emotional problems; vitality (energy and fatigue); and general health perceptions. Version SF-12v2 Health Survey will be used and scores will be recorded using an ePRO.
Time frame: Up to 24 weeks
Retrospective Cohort: Age at Enrollment (Baseline)
Age will be reported as mean/median.
Time frame: At enrollment (baseline)
Retrospective Cohort: Demographics at Enrollment (Baseline)
Demographics characteristics such as sex, country, race will be reported as number/percentage.
Time frame: At enrollment (baseline)
Retrospective Cohort: Number of Participants With Medical History and Clinical Characteristics Including NT1 Diagnosis Details
NT1 medical history and clinical characteristics (since NT1 diagnosis or earlier diagnoses of other sleep related conditions) and methods of NT1 diagnosis (from medical chart review) includes details such as: age at onset of narcolepsy symptoms, age at diagnosis of narcolepsy, NT1, and any other sleep-related conditions, family history of NT1, first documented cataplexy, hypersomnolence, sleep paralysis, hypnagogic/hypnopompic hallucination, disrupted nocturnal sleep, and cognitive symptoms, methods used for diagnosis of NT1, time from onset of NT1 symptoms to NT1 diagnosis, height/body weight/body mass index.
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Number of Participants With Comorbidities
Comorbidities since NT1 diagnosis (from medical chart review) will include: * Obesity * Diabetes mellitus * Metabolic disorders * Cardiovascular disease * Hypertension * Sleep conditions (e.g., hypersomnia, insomnia, parasomnia, rapid eye movement \[REM\] sleep behaviour disorder, sleep apnoea, obstructive sleep apnoea, central sleep apnoea, restless legs syndrome, periodic limb movement disorder, and circadian rhythm disorders) * Other neurological conditions (e.g., migraine, multiple sclerosis, Parkinson's disease) * Psychiatric comorbidities (e.g., depression, anxiety, bipolar disorder, attention deficit-hyperactivity disorder, schizophrenia) * Infectious diseases * Autoimmune disorder (e.g., autoimmune thyroid disease, psoriasis, rheumatoid arthritis, transverse myelitis) * Malignancy * Autonomic dysfunction * Substance abuse disorder (e.g., alcohol, tobacco, marijuana) * Back pain * Charlson comorbidity index (derived) score
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Pharmacological Treatment Outcomes of Narcolepsy Therapies Measured by ESS Total Score
The ESS is a validated, subjective, self-administered questionnaire used to measure daytime sleepiness. The questionnaire consists of 8 questions, each describing a common situation (e.g., sitting and reading, watching TV, sitting in a car as a passenger). Participants rate their likelihood of dozing off in each situation on a scale from 0 (would never doze off) to 3 (high chance of dozing off). The total score ranges from 0 to 24. Higher scores indicate greater daytime sleepiness. ESS total score will be recorded using ePRO.
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Pharmacological Treatment Outcomes of Narcolepsy Therapies Measured by CGI-S Scores
CGI-S is a 7-point scale used by clinicians to rate the severity of a participant's illness based on a global assessment of observed and reported symptoms, behaviour, and function over the past 7 days. It assesses the overall severity of mental illness relative to the clinician's experience with similar participants and uses a scale from "1= normal, not at all ill" to "7= among the most extremely ill participants".
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Pharmacological Treatment Outcomes of Narcolepsy Therapies Measured by WCR
The WCR is a measure of the average number of cataplexy episodes a person experiences in a week. WCR will be collected from medical records for the retrospective cohort.
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Pharmacological Treatment Outcomes of Narcolepsy Therapies Measured by Narcolepsy Severity Scale (NSS) Total Score
The NSS is a clinical tool designed to assess the severity and impact of narcolepsy. The scale consists of 15 items, each addressing different aspects of narcolepsy symptoms: EDS (items 1-7), cataplexy (items 8-10), hallucinations (items 11 and 12), sleep paralysis (items 13 and 14), and disturbed nighttime sleep (item 15). Participants rate the frequency and severity of their symptoms over the past month. Items will be scored on a scale where mild=0-14, moderate=15-28, severe=29-42, very severe=43-57. Higher scores indicate more severe symptoms. Symptoms assessed include EDS, cataplexy, hallucinations, and sleep paralysis.
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Pharmacological Treatment Outcomes of Narcolepsy Therapies Measured by Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score
The NSS-CT is a clinical tool designed to assess the severity and impact of narcolepsy. The scale consists of 15 items, each addressing different aspects of narcolepsy symptoms: EDS (items 1-7), cataplexy (items 8-10), hallucinations (items 11 and 12), sleep paralysis (items 13 and 14), and disturbed nighttime sleep (item 15). Participants rate the frequency and severity of their symptoms over the past week. Items will be scored on a scale where mild=0-14, moderate=15-28, severe=29-42, very severe=43-57. Higher scores indicate more severe symptoms.
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort and Prospective Cohort: Healthcare Resource Utilization (HCRU): Number of Visits
* Number of visits to NT1-treating physicians (neurologists, sleep specialists) * Number of emergency department visits as per reasons * Number of outpatient visits as per type (office-based provider, specialist, primary care physician)
Time frame: Retrospective Cohort: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment; Prospective Cohort: Up to 24 weeks
Retrospective Cohort and Prospective Cohort: HCRU: Number of Hospitalizations as Per Reasons
Time frame: Retrospective Cohort: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment; Prospective Cohort: Up to 24 weeks
Retrospective Cohort and Prospective Cohort: HCRU: Length (in Days) of Hospitalizations as Per Reasons
Time frame: Retrospective Cohort: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment; Prospective Cohort: Up to 24 weeks
Retrospective Cohort and Prospective Cohort: HCRU: Number of Procedures Because of NT1 Symptoms or Complications (Maintenance of Wakefulness Test, Multiple Sleep Latency Test, Polysomnography)
Time frame: Retrospective Cohort: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment; Prospective Cohort: Up to 24 weeks
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by ESS Total Score
The ESS is a validated, subjective, self-administered questionnaire used to measure daytime sleepiness. The questionnaire consists of 8 questions, each describing a common situation (e.g., sitting and reading, watching TV, sitting in a car as a passenger). Participants rate their likelihood of dozing off in each situation on a scale from 0 (would never doze off) to 3 (high chance of dozing off). The total score ranges from 0 to 24. Higher scores indicate greater daytime sleepiness. ESS total score will be recorded using ePRO.
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by "Adaptive Behaviors to Cataplexy Symptoms" as Assessed Through a Study-specific Questionnaire
The "Adaptive Behaviors to Cataplexy Symptoms" is a questionnaire developed specifically for this study. The questionnaire consists of 1 question to select which daily activities a participant avoids/takes precaution or changes because of cataplexy or fear of cataplexy. Answers will be analyzed descriptively as percentage of participants selecting a specific option.
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by PGI-S
The PGI-S is scale for the participant to rate his/her disease severity at the time of assessment on a 5-point scale ranging from a score of 0 ("none") to 4 ("very severe").
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by "Pharmacotherapy Adherence to Treatment" as Assessed Through a Study-specific Questionnaire
The "Pharmacotherapy Adherence to Treatment" is a questionnaire developed specifically for this study. The questionnaire consists of 2 questions about the participant's adherence to treatment regimen and the main reasons for non-adherence. This questionnaire will be recorded using a one-week recall period for the participants who are on NT1 treatment at enrolment. Answers will be analyzed descriptively as percentage of participants selecting a specific option.
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by SF-12 PCS and MCS Scores
The SF-12 is a validated questionnaire that measures general HRQoL. It contains one multi-item scale that assesses 8 domains: limitations in physical activities because of health problems; limitations in social activities because of physical or emotional problems; limitations in usual role activities because of physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities because of emotional problems; vitality (energy and fatigue); and general health perceptions. Version SF-12v2 Health Survey will be used and scores will be recorded using an ePRO.
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by FINI Domain Scores
The FINI is a PRO measure of 28-item, 6-domain (Tiredness, Cognitive Functioning, Cataplexy \[for NT1\], Social Activities, Everyday Activities, and Everyday Responsibilities) (FINI v1.0). FINI items evaluate narcolepsy/EDS symptoms and impacts on daily functioning. All items have a 7-day recall period on a 5-point Likert scale of frequency from "never" to "always" or severity from "not at all" to "very much." For each domain, an average item score is calculated and standardized to a 0 to 100 scale. Lower scores indicate better health/functioning. FINI will be recorded using an ePRO.
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by NSS-CT Total Score
The NSS-CT is a clinical tool designed to assess the severity and impact of narcolepsy. The scale consists of 15 items, each addressing different aspects of narcolepsy symptoms: EDS (items 1-7), cataplexy (items 8-10), hallucinations (items 11 and 12), sleep paralysis (items 13 and 14), and disturbed nighttime sleep (item 15). Participants rate the frequency and severity of their symptoms over the past week. Items will be scored on a scale where mild=0-14, moderate=15-28, severe=29-42, very severe=43-57. Higher scores indicate more severe symptoms. NSS-CT total score will be recorded using an ePRO.
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: Treatment Satisfaction Measured by Treatment Satisfaction Questionnaire for Medication Version 9 (TSQM-9)
The TSQM-9 is used to measure participant satisfaction with medication, assessed in 3 domains (effectiveness, convenience, and global satisfaction). The 3 dimensions of the TSQM-9 will be recorded using an ePRO.
Time frame: Cross-sectional survey at enrollment (baseline)
Prospective Cohort: Treatment Use of Pitolisant and/or Sodium Oxybate
Number of Participants will be reported by treatment use (that is \[i.e.\] pitolisant and/or sodium oxybate).
Time frame: Up to 24 weeks
Prospective Cohort: Treatment Regimen for Pitolisant and/or Sodium Oxybate
Number of participants will be reported based on treatment regimen (i.e. daily dose and frequency).
Time frame: Up to 24 weeks
Prospective Cohort: Duration of Treatment With Pitolisant and/or Sodium Oxybate
Duration of treatment with pitolisant or sodium oxybate will be measured from the treatment start date to date of discontinuation.
Time frame: Up to 24 weeks
Prospective Cohort: Treatment Regimen Adjustment for Pitolisant and/or Sodium Oxybate
Number of participants who experience changes in pitolisant and sodium oxybate treatment (e.g., dose adjustments, discontinuing or switching medications) and related reasons will be reported.
Time frame: Up to 24 weeks
Prospective Cohort: Use of Concomitant Medications With Pitolisant and/or Sodium Oxybate
Number of participants with concomitant pharmacological treatment used for NT1 will be reported.
Time frame: Up to 24 weeks
Prospective Cohort: Change From Baseline in ESS Total Score
The ESS is a validated, subjective, self-administered questionnaire used to measure daytime sleepiness. The questionnaire consists of 8 questions, each describing a common situation (e.g., sitting and reading, watching TV, sitting in a car as a passenger). Participants rate their likelihood of dozing off in each situation on a scale from 0 (would never doze off) to 3 (high chance of dozing off). The total score ranges from 0 to 24. Higher scores indicate greater daytime sleepiness. ESS total score will be recorded using ePRO.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Perceived Change From Baseline in PGI-S Score
The PGI-S is scale for the participant to rate his/her disease severity at the time of assessment on a 5-point scale ranging from a score of 0 ("none") to 4 ("very severe").
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Perceived Change From Baseline in CGI-S Score
CGI-S is a 7-point scale used by clinicians to rate the severity of a participant's illness based on a global assessment of observed and reported symptoms, behaviour, and function over the past 7 days. It assesses the overall severity of mental illness relative to the clinician's experience with similar participants and uses a scale from "1= normal, not at all ill" to "7= among the most extremely ill participants".
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in PGI-C Score
The PGI-C measures change due to treatment relative to PGI-S on a 7-point scale ranging from a score of 1 ("very much improved") to a score of 7 ("very much worse").
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in CGI-C Score
CGI-C is a 7-point scale that requires the clinician to assess how much the participant's illness has improved since baseline. Ratings range from "very much improved" (1) to "very much worse" (7). CGI-C scores will be recorded using physician's reported observations and ePRO.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in WCR
The WCR is a measure of the average number of cataplexy episodes a person experiences in a week. Participants will be asked to complete self-reported cataplexy questions in an e-diary daily for 14 days at each timepoint (1 week before and 1 week after each timepoint). WCR will be recorded using e-diary entries.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Adaptive Behaviors to Cataplexy Symptoms
Adaptive behaviors to cataplexy symptoms will also be assessed over timepoints and may include activity avoidance, precautionary measures and modifications to daily activities due to cataplexy or fear of cataplexy. Adaptive behaviors to cataplexy symptoms will be recorded using e-diary entries.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Domain Scores Measured by FINI
The FINI is a PRO measure of 28-item, 6-domain (Tiredness, Cognitive Functioning, Cataplexy \[for NT1\], Social Activities, Everyday Activities, and Everyday Responsibilities) (FINI v1.0). FINI items evaluate narcolepsy/EDS symptoms and impacts on daily functioning. All items have a 7-day recall period on a 5-point Likert scale of frequency from "never" to "always" or severity from "not at all" to "very much." For each domain, an average item score is calculated and standardized to a 0 to 100 scale. Lower scores indicate better health/functioning. FINI will be recorded using an ePRO.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Domain Scores Measured by NSS-CT Total Score
The NSS-CT is a clinical tool designed to assess the severity and impact of narcolepsy. The scale consists of 15 items, each addressing different aspects of narcolepsy symptoms: EDS (items 1-7), cataplexy (items 8-10), hallucinations (items 11 and 12), sleep paralysis (items 13 and 14), and disturbed nighttime sleep (item 15). Participants rate the frequency and severity of their symptoms over the past week. Items will be scored on a scale where mild=0-14, moderate=15-28, severe=29-42, very severe=43-57. Higher scores indicate more severe symptoms. NSS-CT will be recorded using an ePRO.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Domain Scores Measured by SF-12 PCS and MCS Scores
The SF-12 is a validated questionnaire that measures general HRQoL. It contains one multi-item scale that assesses 8 domains: limitations in physical activities because of health problems; limitations in social activities because of physical or emotional problems; limitations in usual role activities because of physical health problems; bodily pain; general mental health (psychological distress and well-being); limitations in usual role activities because of emotional problems; vitality (energy and fatigue); and general health perceptions. Version SF-12v2 Health Survey will be used and scores will be recorded using an ePRO.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Treatment Satisfaction Measured by the Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9)
The TSQM-9 is used to measure participant satisfaction with medication, assessed in 3 domains (effectiveness, convenience, and global satisfaction). The 3 dimensions of the TSQM-9 will be recorded using ePRO.
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Adherence to Treatment Measured by E-Diary Over 24 weeks
Daily adherence (medication adherence, reasons for non-adherence. intentional dose or timing adjustments of pitolisant or sodium oxybate based on daily activities or schedule may also be evaluated), as measured by adherence questions for 14 days around each timepoint will be recorded.
Time frame: Up to 24 weeks
No study locations are listed for this record.
Plan to share: 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.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Data sharing details ↗ · vivli.org
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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