Clinical trial NCT04667338: A Study to Collect Information of People With Narcolepsy in Spain
Official title: Observational, Multicentre, Cross-sectional Study to Describe Diagnosis and Treatment Patterns in Narcolepsy Patients in Real Life Practice in Spain
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Narcolepsy
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2020-12-14
Enrollment: 214
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with confirmed diagnosis of narcolepsy defined by ICDS-3.

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.

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).

Eligibility 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.

Primary outcomes:
measure: Percentage of Participants Categorized Based on Treatment by Narcolepsy Type Since Diagnosis up to Study Visit
timeFrame: From narcolepsy diagnosis up to study visit (up to 12 months)
measure: Percentage of Participants Categorized Based on Non-Pharmacological Treatments Patterns by Treatment Status
timeFrame: From narcolepsy diagnosis up to study visit (up to 12 months)
measure: Percentage of Treated or Naïve (Untreated) Participants for the Type of Pharmacological Treatment Status Since Diagnosis
timeFrame: From narcolepsy diagnosis up to study visit (up to 12 months)
measure: Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Secondary outcomes:
measure: Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
timeFrame: Day 1
measure: Height of Participants at Study Visit
timeFrame: Day 1
measure: Weight of Participants at Study Visit
timeFrame: Day 1
measure: Body Mass Index (BMI) of Participants at Study Visit
timeFrame: Day 1
measure: Blood Pressure (BP) of Participants at Study Visit
timeFrame: Day 1
measure: Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
timeFrame: From a year before narcolepsy diagnosis up to 12 months
measure: Time From First Symptom to Diagnosis of Narcolepsy
timeFrame: From first symptom of narcolepsy up to diagnosis maximum up to 59.92 years (retrospective data was retrieved and observed during 20 months of this study)
measure: Time From Diagnosis of Narcolepsy to First Treatment
timeFrame: From first symptom of narcolepsy up to diagnosis maximum up to 46.04 years (retrospective data was retrieved and observed during 20 months of this study)
measure: Percentage of Participants Categorized Based on Pharmacological Treatment and Interventions Received in Relation With the Presence of Most Typical Symptoms
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Percentage of Participants With Pharmacological Treatments at Each Line
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Sustained Attention to Response Task (SART)
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Maintenance of Wakefulness Test (MWT)
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Epworth Sleepiness Scale (ESS)
timeFrame: From a year before narcolepsy diagnosis up to 12 months
measure: Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
timeFrame: From a year before study visit (up to 12 months)
measure: Duration of Hospitalization
timeFrame: From a year before study visit (up to 12 months)
measure: Work Productivity and Activity Impairment (WPAI) Score
timeFrame: From a year before study visit (up to 20 months)
measure: Number of Participants Categorized Based on Occupational Accidents
timeFrame: From a year before study visit (up to 20 months)
measure: Percentage of Participants Categorized Based on Cost of Direct Healthcare Resources Utilized by Participants Per Year
timeFrame: From a year before study visit (approximately 20 months)
measure: Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Stigma Scale for Chronic Illness 8-item Version (SSCI-8) Total Score
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Treatment Satisfaction of Participants Assessed by Treatment Satisfaction Questionnaire for Medication (TSQM-9)
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Charlson Comorbidity Index (CCI)
timeFrame: From a year before narcolepsy diagnosis up to study visit (up to 20 months)
measure: Number of Participants With Adverse Events (AEs)
timeFrame: Up to 20 months
Other outcomes:
Locations:
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
state: Cataluna
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: