Narcolepsy Type 1RETRACENCT07903220
A Study About Treatment Use in Adults With Narcolepsy Type 1 (NT1) in Europe and Canada
An observational study of Narcolepsy Type 1, run by Takeda.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Narcolepsy Type 1
- How many people
- 400 participants
- Where
- No sites listed yet
- Study dates
- 20 Dec 2026to 30 Sep 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
None listed.
Who cannot join
- Has a current medical condition associated with EDS other than NT1.
- Has participated in a narcolepsy investigational drug clinical trial involving an orexin receptor agonist during the retrospective observation period.
- Criteria for the Prospective Cohort Inclusion criteria
- Agrees and is able to provide signed informed consent.
- Has a documented diagnosis of NT1, established according to internationally recognized diagnostic criteria (e.g., ICSD or equivalent European/national guidelines).
- Is between 18 and 70 years of age (inclusive) at enrollment.
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- Is planning to initiate treatment with pitolisant or sodium oxybate (as monotherapy or in combination with other narcolepsy pharmacotherapies) to treat symptoms associated with NT1 on the day of or within 28 days after enrollment.
- Has an ESS total score greater than or equal to (≥) 11 within 14 days prior to or at enrollment.
- Participants must meet one of the following:
- Currently treated Participants: The participant has been receiving an anti-cataplexy medication for ≥ 2 months prior to enrollment and had a historical WCR ≥ 4 (partial or complete episodes) prior to initiating the anticataplexy treatment; or
- Treatment-naïve participants: The participant is naïve to narcolepsy treatment at enrollment and has a WCR ≥ 4 (partial or complete episodes) within 14 days prior to or on the day of enrollment
- Has reliable internet access and is willing to complete ePROs/e-diary for the duration of the study.
- Has a current medical condition other than NT1 associated with EDS.
- Has any severe or unstable psychiatric disorder, or active major depressive episode within 6 months prior to or at enrollment, as assessed by the investigator.
- Is participating in a narcolepsy investigational drug clinical trial involving an orexin receptor agonist at study enrollment.
- Has any significant medical condition that, in the judgment of the treating physician, may compromise follow-up or study participation.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Real-World Treatment Patterns in Narcolepsy Type 1: A Retrospective, Cross-Sectional and Prospective, Observational Study in Europe and Canada
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.
Read the detailed description
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.
- Conditions
- Narcolepsy Type 1
- Being tested
- No intervention
- Keywords
- Drug therapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| Retrospective Cohort With a Cross-Sectional Survey Component | 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. Receives: Other: No intervention | |
| Prospective Cohort | 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. Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Retrospective Cohort: Percentage of Participants in Each Treatment Sequence
Measured: 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
Measured: 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
Measured: Prospective Cohort: Up to 24 weeks
Retrospective Cohort and Prospective Cohort: Average Duration on Treatment
Measured: 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
Measured: 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
Measured: 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.
Measured: 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".
Measured: 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).
Measured: 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").
Measured: 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").
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 24 weeks
Secondary outcomes (38)
Retrospective Cohort: Age at Enrollment (Baseline)
Age will be reported as mean/median.
Measured: At enrollment (baseline)
Retrospective Cohort: Demographics at Enrollment (Baseline)
Demographics characteristics such as sex, country, race will be reported as number/percentage.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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".
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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)
Measured: 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
Measured: 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
Measured: 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)
Measured: 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.
Measured: 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.
Measured: 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").
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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").
Measured: 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".
Measured: 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").
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2026
- Study start20 Dec 2026 (estimated)
- Primary completion30 Sep 2027 (estimated)
- Study completion30 Sep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Narcolepsy Type 1Studies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07903220
- Sponsor's protocol ID
- TAK-861-3009
- Acronym
- RETRACE
- Status last verified
- Oct 2026
- First submitted
- 6 Oct 2026
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- 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.
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Treatment Patterns in Narcolepsy Type 1: A Retrospective, Cross-Sectional and Prospective, Observational Study in Europe and Canada
- ClinicalTrials.gov ID
- NCT07903220
- Acronym / sponsor ID
- RETRACE / TAK-861-3009
- Conditions
- Narcolepsy Type 1
- Keywords
- Drug therapy
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 20 Dec 2026 (estimated)
- Primary completion
- 30 Sep 2027 (estimated)
- Study completion
- 30 Sep 2027 (estimated)
- First posted
- 29 Oct 2026 (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.
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.
Detailed description
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Retrospective Cohort With a Cross-Sectional Survey Component | – | Other: No interventionData 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. |
| Prospective Cohort | – | Other: No interventionData 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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- 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.
Key inclusion criteria (0 listed)
Key exclusion criteria (16 listed)
- Has a current medical condition associated with EDS other than NT1.
- Has participated in a narcolepsy investigational drug clinical trial involving an orexin receptor agonist during the retrospective observation period.
- Criteria for the Prospective Cohort Inclusion criteria
- Agrees and is able to provide signed informed consent.
- Has a documented diagnosis of NT1, established according to internationally recognized diagnostic criteria (e.g., ICSD or equivalent European/national guidelines).
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 |
|---|---|---|
| No interventionThis is a non-interventional study. | Other | Prospective Cohort, Retrospective Cohort With a Cross-Sectional Survey Component |
Primary outcome
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)
Time frame: Up to 24 weeks
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.
Prospective Cohort: Narcolepsy Symptoms Measured by the Clinical Global Impression of Severity (CGI-S)
Time frame: Up to 24 weeks
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".
Prospective Cohort: Narcolepsy Symptoms Measured by the Clinical Global Impression of Change (CGI-C)
Time frame: Up to 24 weeks
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).
Prospective Cohort: Narcolepsy Symptoms Measured by the Patient Global Impression of Severity (PGI-S)
Time frame: Up to 24 weeks
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").
Prospective Cohort: Narcolepsy Symptoms Measured by the Patient Global Impression of Change (PGI-C)
Time frame: Up to 24 weeks
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").
Prospective Cohort: Narcolepsy Symptoms Measured by the Functional Impacts of Narcolepsy Instrument (FINI)
Time frame: Up to 24 weeks
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.
Prospective Cohort: Narcolepsy Symptoms Measured by Narcolepsy Severity Scale for Clinical Trials (NSS-CT) Total Score
Time frame: Up to 24 weeks
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.
Prospective Cohort: Cataplexy Measured by Weekly Cataplexy Rate (WCR)
Time frame: Up to 24 weeks
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.
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)
Time frame: Up to 24 weeks
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.
38 secondary outcomes
Retrospective Cohort: Age at Enrollment (Baseline)
Time frame: At enrollment (baseline)
Retrospective Cohort: Demographics at Enrollment (Baseline)
Time frame: At enrollment (baseline)
Retrospective Cohort: Number of Participants With Medical History and Clinical Characteristics Including NT1 Diagnosis Details
Time frame: From NT1 diagnosis up to enrollment (baseline), approximately past 5 years from enrollment
Retrospective Cohort: Number of Participants With Comorbidities
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
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
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
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
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
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
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
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
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by PGI-S
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
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by SF-12 PCS and MCS Scores
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by FINI Domain Scores
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: HRQoL and Clinical Outcomes Measured by NSS-CT Total Score
Time frame: Cross-sectional survey at enrollment (baseline)
Retrospective Cohort: Treatment Satisfaction Measured by Treatment Satisfaction Questionnaire for Medication Version 9 (TSQM-9)
Time frame: Cross-sectional survey at enrollment (baseline)
Prospective Cohort: Treatment Use of Pitolisant and/or Sodium Oxybate
Time frame: Up to 24 weeks
Prospective Cohort: Treatment Regimen for Pitolisant and/or Sodium Oxybate
Time frame: Up to 24 weeks
Prospective Cohort: Duration of Treatment With Pitolisant and/or Sodium Oxybate
Time frame: Up to 24 weeks
Prospective Cohort: Treatment Regimen Adjustment for Pitolisant and/or Sodium Oxybate
Time frame: Up to 24 weeks
Prospective Cohort: Use of Concomitant Medications With Pitolisant and/or Sodium Oxybate
Time frame: Up to 24 weeks
Prospective Cohort: Change From Baseline in ESS Total Score
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Perceived Change From Baseline in PGI-S Score
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Perceived Change From Baseline in CGI-S Score
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in PGI-C Score
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in CGI-C Score
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in WCR
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Adaptive Behaviors to Cataplexy Symptoms
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Domain Scores Measured by FINI
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Domain Scores Measured by NSS-CT Total Score
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Change From Baseline in Domain Scores Measured by SF-12 PCS and MCS Scores
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)
Time frame: Baseline, Up to 24 weeks
Prospective Cohort: Adherence to Treatment Measured by E-Diary Over 24 weeks
Time frame: Up to 24 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Oct 2026.
- Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.Link 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.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
An observational study of Narcolepsy Type 1, run by Takeda.
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 0 inclusion and 16 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: opening soon, as of the sponsor's last update on 29 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Takeda Contact (+1-877-825-3327, medinfoUS@takeda.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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]). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 2026; AzTrials last refreshed its copy on 3 Nov 2026.