A Study to Collect Information of People With Narcolepsy in Spain (SOMNUS)

• ClinicalTrials.gov Identifier: NCT04667338
• Recruitment Status: Recruiting
• First Posted: December 14, 2020
• Last Update Posted: January 21, 2022
• Sponsor: Takeda
• Information provided by (Responsible Party): Takeda

Study Description

Brief Summary:

This study aims to review information of people with a sleep condition called narcolepsy. Narcolepsy is a condition that causes extreme sleepiness during the day including falling asleep suddenly. Study doctors will review the medical records of the participants from sleep clinics in Spain. They will do this from 1 year before the participant was diagnosed with narcolepsy up to the start of this study.

Participants will visit the sleep clinic once. During this visit, the study doctors will carry out a short medical exam. Participants will also complete a few questionnaires during this visit. If participants cannot visit the clinic for any reason, the clinic staff will arrange a phone call instead.

Condition or disease
Narcolepsy

Detailed Description:

This is a non-interventional, cross-sectional study with retrospective medical chart review conducted in public and private Spanish sleep clinics, to describe the management of adult participants diagnosed with narcolepsy defined by ICDS-3 in real world practice.

This study will enroll approximately 196 participants (137 with NT1 and 59 with NT2). Participants will be enrolled in 2 cohorts:

• Cohort A: NT1 Participants
• Cohort B: NT2 Participants

The data will be collected retrospectively through clinical records and at study visit (clinical and demographic data, and participant related outcomes [PRO]) in an electronic case report form (eCRF).

This multi-center study will be conducted in Spain. The overall duration of this study is approximately 24 months. Participants will attend a single visit in the participating centers. In the event of a possible outbreak of coronavirus disease 2019 (COVID-19) participants will have a telepathic visits, the invitation to participate, the obtention of participant's informed consent and the collection of participant's questionnaires will be carried out remotely (by phone or via email).

Study Design

• Study Type: Observational
• Estimated Enrollment: 196 participants
• Observational Model: Cohort
• Time Perspective: Cross-Sectional
• Official Title: Observational, Multicentre, Cross-sectional Study to Describe Diagnosis and Treatment Patterns in Narcolepsy Patients in Real Life Practice in Spain
• Actual Study Start Date: February 24, 2021
• Estimated Primary Completion Date: April 15, 2022
• Estimated Study Completion Date: April 15, 2022

Groups and Cohorts

Group/Cohort
Cohort A: NT1 Participants; Participants with confirmed diagnosis of narcolepsy type 1 (NT1) defined by the International Classification of Sleep Disorders, Third Edition (ICDS-3) will be observed for up to 24 months.
Cohort B: NT2 Participants; Participants with confirmed diagnosis of narcolepsy type 2 (NT2) defined by the ICDS-3 will be observed for up to 24 months.

Outcome Measures

Primary Outcome Measures :

1. Percentage of Participants Categorized Based on Treatments Patterns [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
   Percentage of participants will be reported categorized based on treatment patterns including use of treatments (yes/no), use of different treatments (pharmacological and non-pharmacological), and use of each type of treatment stratified by type of hospital (public or private centers).

Secondary Outcome Measures :

1. Percentage of Participants Categorized Based on Different Specialists who Diagnosed Narcolepsy [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
2. Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
   Percentage of participants will be reported based on clinical history, absence/presence of cataplexy, number of cataplexy attacks, number of sleep-onset REM periods (SOREMPs), absence/presence of apneas and /or Apnea-hypopnea Index (AHI), hipocrexine levels, Epworth Sleepiness Scale (ESS), polysomnogram (PSG), multiple sleep latency test (MSLT) and types of human leucocyte antigen (HLA).
3. Time From First Symptom to Diagnosis of Narcolepsy [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
4. Time from Diagnosis of Narcolepsy to First Treatment [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
5. Percentage of Participants Categorized Based on Pharmacological Treatment and Interventions Received in Relation With the Presence of Most Typical Symptoms [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
   Most typical symptoms will include excessive day time sleepiness, cataplexy, sleep paralysis, and hallucinations.
6. Percentage of Participants With Pharmacological Treatments at Each Line [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
7. Sustained Attention to Response Task (SART) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
   Behavioral measure of working memory, attention, and impulse/inhibitory control - The Sustained Attention to Response Task (SART) is a computer-based go/no-go task that requires participants to withhold behavioral response to a single, infrequent target (often the digit 3) presented amongst a background of frequent non-targets (0-2, 4-9). After each block, two probe questions are presented in succession. The first asks, "Where was your attention focused during this block of trials?" Participants respond on a 6-point Likert scale, where 1 represents, "on task," and 6, "off task." A second question asks, "How aware were you of where your attention was during this block of trials?" Participants respond on a similar scale, where 1 represents, "aware," and 6, "unaware."
8. Maintenance of Wakefulness Test (MWT) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
   The MWT is a validated objective measure that evaluates a person's ability to remain awake under soporific conditions for a defined period of time. This tendency to fall asleep is measured via electroencephalography-derived sleep latency. Sleep onset is defined as the first epoch of greater than 15 seconds of cumulative sleep in a 30-second epoch. During each MWT session (1 session equals to [=] 40 minutes), participants will be instructed to sit quietly and remain awake for as long as possible. If no sleep has been observed according to these rules, then the latency is defined as 40 minutes. MWT sleep latency ranges from 0 to 40 minutes, with higher scores indicating greater ability to stay awake.
9. Epworth Sleepiness Scale (ESS) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
   ESS is a subjective, self-administered questionnaire to assess daytime sleepiness and consists of 8 items, on a 4-point scale (0-3) ranging from "No chance of dozing" to "High chance of dozing" while engaged in eight different activities. The score can range from 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are considered to be within the normal range.
10. Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
    Percentage of participants will be reported based on different non-pharmacological treatment approach assessed through ad-hoc questionnaire including taking short naps, maintaining a regular sleep schedule, avoiding caffeine or alcohol before bed, avoiding smoking especially at night, daily exercise, avoiding large and heavy meals before bedtime, and relaxation before bed.
11. Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized per Year [ Time Frame: From a year before study visit (approximately 24 months) ]
    Percentage of participants will be reported based on assessment of type and number of direct healthcare resource utilized per year which include routine monitoring visits (specialists involved, tests conducted, treatment variations), number of emergency visits (specialists involved, tests/procedures conducted, treatment administered), number and duration of hospitalizations and complications derived from narcolepsy.
12. Work Productivity and Activity Impairment (WPAI) [ Time Frame: From a year before study visit (approximately 24 months) ]
    WPAI scores will be used to describe the indirect healthcare resources. It is self-administered 6-item questionnaire (scale of 0-10) which assess the effect of disease on work productivity in the past 7 days obtained for absenteeism (work time missed), presenteeism (impairment at work / reduced on-the-job effectiveness), work productivity loss (overall work impairment / absenteeism plus presenteeism) and activity Impairment / disability. Higher scores indicates greater impairment.
13. Number of Participants Categorized Based on Occupational Accidents [ Time Frame: From a year before study visit (approximately 24 months) ]
    Number of participants will be reported based on occupational accidents. Occupational accidents will be assessed through ad-hoc questions for motor vehicle accidents (MVAs) and near miss accidents (NMAs) and will be described through the answer of the two following questions by participants: "Have you had a motor vehicle accident at work during the last years?" and "Have you had a near-miss driving accident during the last year?
14. Percentage of Participants Categorized Based on Cost of Direct Healthcare Resources Utilized by Participants per Year [ Time Frame: From a year before study visit (approximately 24 months) ]
    Percentage of participants will be reported based on costs of following direct healthcare resources will be included: treatment received, routine monitoring visits (specialists and GPs), tests performed, emergency rooms visits, hospitalizations and complications derived from narcolepsy, and unitary costs.
15. Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
    The EQ-5D questionnaire is a generic instrument for describing and valuing health. It is based on a descriptive system that defines health in terms of 5 dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression. Each dimension has 3 response categories corresponding to no problems, some problems, and extreme problems. The instrument is designed for self-completion, and respondents also rate their overall health on the day of the interview on a 0-100 hash-marked, vertical visual analogue scale (EQ-VAS). Higher score indicates better health state.
16. Stigma Scale for Chronic Illness 8-item version (SSCI-8) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
    SSCI-8 is a 8 items scale developed to assess internalized and experienced stigma across neurological conditions. It uses a 5-point Likert scale ranging from 1 (never) to 5 (always).
17. Treatment Satisfaction of Participants Assessed by Treatment Satisfaction Questionnaire for Medication (TSQM-9) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
    TSQM-9, derived from the TSQM-14, is 9-item treatment satisfaction questionnaire for medication as a general measure of satisfaction with treatment. It measures scores on 3 scales: effectiveness scale (questions 1 to 3), the convenience scale (questions 9 to 11) and the overall satisfaction scale (questions 12 to 14). The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain.
18. Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
19. Charlson Comorbidity Index (CCI) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]
    CCI is a scale to estimate 10-year mortality based on a score from a range of 12 comorbidities, the comorbidity score ranges from 0 to a maximum of 24 points. Higher scores indicate the higher chances of mortality.
20. Number of Participants With Adverse Events (AEs) [ Time Frame: From a year before narcolepsy diagnosis up to study visit (approximately 24 months) ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

Participants with confirmed diagnosis of narcolepsy defined by ICDS-3.

Criteria

Inclusion Criteria:

1. With confirmed diagnosis of narcolepsy defined by ICDS-3.
2. With at least 1-year follow-up with data available at the participating site after initial narcolepsy diagnosis and before study inclusion.
3. With data available at the participant site at least 1-year before first narcolepsy diagnosis.
4. Capable to fulfill the study questionnaires.

Exclusion Criteria:

1. Participating in a clinical trial (less than or equal to [<=] 12 months).
2. With any serious degenerative disease (Alzheimer, Parkinson or epilepsy) or psychiatric condition.

Contacts and Locations

Contacts

Contact: Takeda Contact; +1-877-825-3327; medinfoUS@takeda.com

Locations

Spain

Hospital Clinic de Barcelona; Recruiting

Barcelona, Cataluna, Spain, 8036

Contact: Site Contact airanzo@clinic.cat

Principal Investigator: Alejandro Iranzo

Sponsors and Collaborators

Takeda

Investigators

• Study Director: Study Director; Takeda

More Information

Additional Information:

To obtain more information on the study, click here/on this link 

• Responsible Party: Takeda
• ClinicalTrials.gov Identifier: NCT04667338
• Other Study ID Numbers: TAK-994-5001; U1111-1259-8720 ( Registry Identifier: WHO )
• First Posted: December 14, 2020
• Last Update Posted: January 21, 2022
• Last Verified: January 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: 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 Materials: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)
• 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.
• URL: https://vivli.org/ourmember/takeda/

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Takeda:

Drug Therapy

Additional relevant MeSH terms:

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