Trial Outcomes & Findings for A Study to Collect Information of People With Narcolepsy in Spain (NCT NCT04667338)
NCT ID: NCT04667338
Last Updated: 2024-10-04
Results Overview
Percentage of participants with narcolepsy categorized based on pharmacological and non-pharmacological treatment as 'yes' and 'no' since study diagnosis are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point.
COMPLETED
214 participants
From narcolepsy diagnosis up to study visit (up to 12 months)
2024-10-04
Participant Flow
Participants took part in the study at 12 investigative sites in Spain from 18 February 2021 to 04 November 2022.
This is a non-interventional, retrospective (utilizing existing data from the medical records of patients), cross-sectional study with only one visit, of 214 patient records 210 participants were selected and considered evaluable for this study. 2 participants were not classified as either narcolepsy type 1 (NT1) or narcolepsy type 2 (NT2) and other 2 participants did not fulfil inclusion criteria and were not considered evaluable for this study.
Participant milestones
| Measure |
Narcolepsy Type 1
Participants with confirmed diagnosis of NT1 defined by the International Classification of Sleep Disorders, Third Edition (ICDS-3) were observed for up to 16 months.
|
Narcolepsy Type 2
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Overall Study
STARTED
|
157
|
53
|
|
Overall Study
Naive: Untreated
|
8
|
4
|
|
Overall Study
Treated
|
149
|
49
|
|
Overall Study
COMPLETED
|
157
|
53
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study to Collect Information of People With Narcolepsy in Spain
Baseline characteristics by cohort
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
Total
n=210 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
41.2 years
STANDARD_DEVIATION 13.9 • n=5 Participants
|
42.6 years
STANDARD_DEVIATION 13.0 • n=7 Participants
|
41.6 years
STANDARD_DEVIATION 13.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
79 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
101 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
78 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
109 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
147 Participants
n=5 Participants
|
50 Participants
n=7 Participants
|
197 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
7 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian / Oriental
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From narcolepsy diagnosis up to study visit (up to 12 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Percentage of participants with narcolepsy categorized based on pharmacological and non-pharmacological treatment as 'yes' and 'no' since study diagnosis are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants Categorized Based on Treatment by Narcolepsy Type Since Diagnosis up to Study Visit
Pharmacological: No
|
5.10 percentage of participants
|
7.55 percentage of participants
|
|
Percentage of Participants Categorized Based on Treatment by Narcolepsy Type Since Diagnosis up to Study Visit
Pharmacological: Yes
|
94.90 percentage of participants
|
92.45 percentage of participants
|
|
Percentage of Participants Categorized Based on Treatment by Narcolepsy Type Since Diagnosis up to Study Visit
Non-pharmacological: No
|
6.37 percentage of participants
|
1.89 percentage of participants
|
|
Percentage of Participants Categorized Based on Treatment by Narcolepsy Type Since Diagnosis up to Study Visit
Non-pharmacological: Yes
|
93.63 percentage of participants
|
98.11 percentage of participants
|
PRIMARY outcome
Timeframe: From narcolepsy diagnosis up to study visit (up to 12 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Percentage of participants with narcolepsy categorized based on non-pharmacological treatment patterns as 'yes' and 'no' by treatment status \[Naïve (untreated) and treated\] are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point. Data is collected and reported per Naïve (untreated) and treated arms for this outcome measure.
Outcome measures
| Measure |
Narcolepsy Type 1
n=12 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=198 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants Categorized Based on Non-Pharmacological Treatments Patterns by Treatment Status
No
|
0 percentage of participants
|
5.56 percentage of participants
|
|
Percentage of Participants Categorized Based on Non-Pharmacological Treatments Patterns by Treatment Status
Yes
|
100 percentage of participants
|
94.44 percentage of participants
|
PRIMARY outcome
Timeframe: From narcolepsy diagnosis up to study visit (up to 12 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Percentage of Treated or naïve (Untreated) participants with narcolepsy categorized based on pharmacological treatment status since diagnosis are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Treated or Naïve (Untreated) Participants for the Type of Pharmacological Treatment Status Since Diagnosis
Naive (Untreated)
|
5.10 percentage of participants
|
7.55 percentage of participants
|
|
Percentage of Treated or Naïve (Untreated) Participants for the Type of Pharmacological Treatment Status Since Diagnosis
Treated
|
94.90 percentage of participants
|
92.45 percentage of participants
|
PRIMARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Percentages are rounded off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=149 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=49 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Sodium Oxybate
|
53.33 percentage of participants
|
8.33 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Antidepressants
|
26.67 percentage of participants
|
33.33 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Stimulants
|
30.60 percentage of participants
|
18.92 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Wakefulness-Promoting Agents
|
63.43 percentage of participants
|
91.89 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Sodium Oxybate
|
63.43 percentage of participants
|
5.41 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Antidepressants
|
34.33 percentage of participants
|
5.41 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Benzodiazepines
|
8.96 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Other psychotropic agents
|
4.48 percentage of participants
|
5.41 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Modafinil + Pitolisant + Sodium Oxybate
|
6.72 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Public: Modafinil + Sodium Oxybate
|
16.42 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Stimulants
|
26.67 percentage of participants
|
25.00 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Wakefulness-Promoting Agents
|
60.00 percentage of participants
|
91.67 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Benzodiazepines
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Other psychotropic agents
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Modafinil + Pitolisant + Sodium Oxybate
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)
Private: Modafinil + Sodium Oxybate
|
20.00 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Day 1Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Sociodemographic variables categorized by age, gender (male/female), ethnicity, educational level ongoing or completed level of education (without studies, primary studies, secondary studies, university studies, other superior studies, not available); occupational status and occupation (student, employed, self-employed, employed but on sick leave due to the study disease, permanent incapacity to work due to the study disease, permanent incapacity to work due to other reasons, unemployed, retired, domestic work, other); civil status (married/with partner, divorced/separated, unmarried, widow/er, unknown); living conditions (alone, wife/husband and/or sons, family caregivers, other caregivers, unknown). Clinical variables categorized by smoking status, alcohol intake, exercise status and family history of narcolepsy in first-degree and second-degree relatives\].
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Permanent incapacity to work due to other reasons
|
3 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Unemployed
|
13 Participants
|
4 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Other
|
2 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Missing
|
1 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Smoking status: Non-Smoker
|
68 Participants
|
28 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: Never
|
75 Participants
|
22 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: 2-3 times per week
|
14 Participants
|
9 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Age
|
157 Participants
|
53 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Gender: Male
|
78 Participants
|
31 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Gender: Female
|
79 Participants
|
22 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Ethnicity: Caucasian
|
147 Participants
|
50 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Ethnicity: Hispanic
|
7 Participants
|
3 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Ethnicity: Asian / Oriental
|
1 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Ethnicity: Other
|
2 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: Without studies
|
5 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: Primary Studies
|
18 Participants
|
2 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: Secondary Studies
|
63 Participants
|
21 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: University Studies
|
56 Participants
|
21 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: Other Superior Studies
|
11 Participants
|
5 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: Not available
|
3 Participants
|
3 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Educational level ongoing or completed level of education: Missing
|
1 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Student
|
18 Participants
|
2 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Employed
|
82 Participants
|
30 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Self-employed
|
12 Participants
|
8 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Employed but on sick leave due to the study disease
|
4 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Permanent incapacity to work due to the study disease
|
10 Participants
|
2 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Retired
|
9 Participants
|
4 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Occupational status and occupation: Domestic work
|
3 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Civil status: Married/with partner
|
72 Participants
|
29 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Civil status: Divorced/separated
|
12 Participants
|
5 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Civil status: Unmarried
|
65 Participants
|
15 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Civil status: Widow/er
|
1 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Civil status: Unknown
|
5 Participants
|
3 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Civil status: Missing
|
2 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Living conditions: Alone
|
32 Participants
|
12 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Living conditions: Wife/Husband and/or sons
|
82 Participants
|
34 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Living conditions: Family Caregivers
|
26 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Living conditions: Other Caregivers
|
2 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Living conditions: Unknown
|
12 Participants
|
5 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Living conditions: Missing
|
3 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Smoking status: Current Smoker
|
51 Participants
|
12 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Smoking status: Ex-Smoker
|
33 Participants
|
12 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Smoking status: Unknown
|
3 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Smoking status: Not available
|
2 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: Once or less per month
|
34 Participants
|
12 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: 2-4 times per month
|
25 Participants
|
3 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: 4 or more times per week
|
5 Participants
|
4 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: Unknown
|
2 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: Not available
|
1 Participants
|
2 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Alcohol intake: Missing
|
1 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Exercise status: Never
|
64 Participants
|
16 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Exercise status: Regular
|
41 Participants
|
25 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Exercise status: Frequently
|
29 Participants
|
10 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Exercise status: Always
|
10 Participants
|
0 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Exercise status: Unknown
|
8 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Exercise status: Not available
|
5 Participants
|
1 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Family history: First-degree relatives
|
4 Participants
|
4 Participants
|
|
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit
Family history: Second-degree relatives
|
5 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: Day 1Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Outcome measures
| Measure |
Narcolepsy Type 1
n=138 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=50 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Height of Participants at Study Visit
|
168.6 centimeters (cm)
Standard Deviation 9.8
|
171.0 centimeters (cm)
Standard Deviation 8.7
|
SECONDARY outcome
Timeframe: Day 1Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Outcome measures
| Measure |
Narcolepsy Type 1
n=137 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=50 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Weight of Participants at Study Visit
|
77.45 kilograms (kg)
Standard Deviation 17.97
|
77.17 kilograms (kg)
Standard Deviation 15.08
|
SECONDARY outcome
Timeframe: Day 1Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
BMI = weight(kg)/height(m)\^2
Outcome measures
| Measure |
Narcolepsy Type 1
n=137 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=50 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Body Mass Index (BMI) of Participants at Study Visit
|
27.18 kilograms per meters square (kg/m^2)
Standard Deviation 5.58
|
26.34 kilograms per meters square (kg/m^2)
Standard Deviation 4.71
|
SECONDARY outcome
Timeframe: Day 1Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Outcome measures
| Measure |
Narcolepsy Type 1
n=72 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=28 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Blood Pressure (BP) of Participants at Study Visit
Systolic BP
|
121.0 millimeters of mercury (mm Hg)
Standard Deviation 16.4
|
120.0 millimeters of mercury (mm Hg)
Standard Deviation 14.1
|
|
Blood Pressure (BP) of Participants at Study Visit
Diastolic BP
|
75.7 millimeters of mercury (mm Hg)
Standard Deviation 12.8
|
74.3 millimeters of mercury (mm Hg)
Standard Deviation 10.9
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis. Number analyzed is the number of participants with data available for the specified categories.
Specialist who diagnosed narcolepsy were categorized as general practitioners, neurologist, neuropediatrician, neurophysiologist, pneumologist, somnologist, somnologist-unit and specialist (Not specified). Percentage of participants are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=156 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Somnologist
|
3.21 percentage of participants
|
7.55 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
General practitioner
|
1.28 percentage of participants
|
5.66 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Neurologist
|
37.18 percentage of participants
|
24.53 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Neuropediatrician
|
1.92 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Neurophysiologist
|
49.36 percentage of participants
|
43.40 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Pneumologist
|
2.56 percentage of participants
|
18.87 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Somnologist-unit
|
3.85 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy
Specialist (Not specified)
|
0.64 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to 12 monthsPopulation: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Different procedures or tests for diagnosis included clinical history, Clinical Assessment: Epworth sleepiness scale (ESS) \[ESS consists of 8 items, on a 4-point scale (0-3) ranging from "0=No chance of dozing" to "3=High chance of dozing" while engaged in eight different activities. The score can range from 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are within the normal range\], Neurological Assessment, Multiple sleep latency test (MSLT), Apnea-hypopnea index (AHI), Other procedures, Human leucocyte antigen (HLA) typing, Hypocretin-1 cerebrospinal fluid (CSF) or Orexin. Percentages are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=143 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=45 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Apnea-hypopnea index (AHI)
|
52.87 percentage of participants
|
45.28 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Clinical History
|
91.08 percentage of participants
|
84.91 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Clinical Assessment: Epworth sleepiness scale (ESS)
|
54.14 percentage of participants
|
58.49 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Neurological Assessment
|
59.87 percentage of participants
|
43.40 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Multiple sleep latency test (MSLT)
|
96.18 percentage of participants
|
90.57 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Other procedures
|
7.64 percentage of participants
|
9.43 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Human leucocyte antigen (HLA) typing
|
51.59 percentage of participants
|
32.08 percentage of participants
|
|
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy
Hypocretin-1 cerebrospinal fluid (CSF) or Orexin
|
6.37 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
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)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Time from first symptom in participant to diagnosis is the time when the first symptom of narcolepsy was identified until its diagnosis during retrospective visit. As the first symptom and the diagnosis at retrospective visit could be any time in the past up to end of enrolment in this study. Thus, as pre-planned data for this outcome measure may exceed the duration of the study.
Outcome measures
| Measure |
Narcolepsy Type 1
n=139 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=40 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Time From First Symptom to Diagnosis of Narcolepsy
|
6.30 years
Interval 0.0 to 59.92
|
2.94 years
Interval 0.0 to 47.87
|
SECONDARY outcome
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)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Time from diagnosis to first treatment is the time required to diagnose the first symptom of narcolepsy until the first treatment is provided after diagnosis retrospectively. As the first symptom and the diagnosis at retrospective visit could be any time in the past up to end of enrolment in this study. Thus, as pre-planned data for this outcome measure may exceed the duration of the study.
Outcome measures
| Measure |
Narcolepsy Type 1
n=152 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=49 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Time From Diagnosis of Narcolepsy to First Treatment
|
0.05 years
Interval 0.0 to 46.04
|
0.02 years
Interval 0.0 to 4.43
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: Analysis population included all eligible participants whose data was retrieved and analyzed. Data was not collected for this outcome measure as data was not available in the medical records.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Pharmacological Treatments were categorized as 1st treatment after diagnosis (monotherapy and combinations), treatments used from diagnosis to study visit and treatments at study visit (monotherapy and combinations). Percentages are rounded off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants With Pharmacological Treatments at Each Line
At Study Visit: Combinations
|
24.94 percentage of participants
|
11.32 percentage of participants
|
|
Percentage of Participants With Pharmacological Treatments at Each Line
1st Treatment: Monotherapy
|
82.80 percentage of participants
|
90.57 percentage of participants
|
|
Percentage of Participants With Pharmacological Treatments at Each Line
1st Treatment: Combinations
|
12.10 percentage of participants
|
1.89 percentage of participants
|
|
Percentage of Participants With Pharmacological Treatments at Each Line
From Diagnosis up to Study Visit
|
94.90 percentage of participants
|
92.45 percentage of participants
|
|
Percentage of Participants With Pharmacological Treatments at Each Line
At Study Visit: Monotherapy
|
54.14 percentage of participants
|
71.70 percentage of participants
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: Analysis population included all eligible participants whose data was retrieved and analyzed. Data was not collected for this outcome measure as data was not available in the medical records.
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."
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: Analysis population included all eligible participants whose data was retrieved and analyzed. Data was not collected for this outcome measure as data was not available in the medical records.
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 = 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.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to 12 monthsPopulation: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
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.
Outcome measures
| Measure |
Narcolepsy Type 1
n=75 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=31 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Epworth Sleepiness Scale (ESS)
|
18.08 score on a scale
Standard Deviation 3.51
|
16.35 score on a scale
Standard Deviation 4.29
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Non-pharmacological treatment approach was categorized as take short naps, maintain a regular sleep schedule, avoid caffeine or alcohol before bedtime, avoid smoking, especially at night, exercise daily, avoid large, heavy meals right before bedtime, and other. Percentage of participants are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Other
|
10.19 percentage of participants
|
13.21 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Take short naps
|
79.62 percentage of participants
|
60.38 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Maintain a regular sleep schedule
|
68.15 percentage of participants
|
69.81 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Avoid caffeine or alcohol before bedtime
|
56.69 percentage of participants
|
47.17 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Avoid smoking, especially at night
|
36.94 percentage of participants
|
37.74 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Exercise daily
|
36.31 percentage of participants
|
30.19 percentage of participants
|
|
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach
Avoid large, heavy meals right before bedtime
|
57.96 percentage of participants
|
62.26 percentage of participants
|
SECONDARY outcome
Timeframe: From a year before study visit (up to 12 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Direct healthcare resource utilized per year were categorized as treatments for narcolepsy, routine monitoring visits, tests, emergency visits, hospitalizations and complications associated to narcolepsy. Percentage of participants are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
Treatment
|
89.81 percentage of participants
|
86.79 percentage of participants
|
|
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
Routine monitoring visits
|
78.34 percentage of participants
|
64.15 percentage of participants
|
|
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
Clinical Tests
|
78.98 percentage of participants
|
64.15 percentage of participants
|
|
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
Emergency visits
|
1.27 percentage of participants
|
1.89 percentage of participants
|
|
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
Hospitalizations
|
0 percentage of participants
|
1.89 percentage of participants
|
|
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year
Complications
|
9.55 percentage of participants
|
5.66 percentage of participants
|
SECONDARY outcome
Timeframe: From a year before study visit (up to 12 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants who were hospitalized.
Duration of hospitalization is the total number of days at which participants were admitted in hospital.
Outcome measures
| Measure |
Narcolepsy Type 1
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=1 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Duration of Hospitalization
|
—
|
1.00 days
Interval 1.0 to 1.0
|
SECONDARY outcome
Timeframe: From a year before study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis. Number analyzed is the number of participants available for analysis in the specified categories.
The WPAI is a questionnaire that assesses the impact of an intervention on work productivity, evaluating 4 areas including absenteeism \[Q2/(Q2+Q4)\], presenteeism \[Q5/10\], work productivity loss {Q2/(Q2+Q4)+\[(1-(Q2/(Q2+Q4))x(Q5/10)\]}, and activity impairment \[Q6/10\], calculated based on three items: (Q2) the number of hours missed from work due to health problems in the past seven days; (Q4) the number of actual work hours in the past seven days; and (Q5) to what degree did the disease impair the productivity while working past seven days. The data calculated for each area is then converted to percent. Each of the areas is scored separately as a percentage, ranging from 0 to 100, with higher scores indicating greater impairment and less productivity.
Outcome measures
| Measure |
Narcolepsy Type 1
n=91 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=36 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Work Productivity and Activity Impairment (WPAI) Score
Activity Impairment / Disability
|
36.2 score on a scale
Standard Deviation 31.4
|
31.1 score on a scale
Standard Deviation 31.8
|
|
Work Productivity and Activity Impairment (WPAI) Score
Absenteeism
|
11.57 score on a scale
Standard Deviation 25.24
|
3.23 score on a scale
Standard Deviation 8.87
|
|
Work Productivity and Activity Impairment (WPAI) Score
Presenteeism
|
32.4 score on a scale
Standard Deviation 29.5
|
28.1 score on a scale
Standard Deviation 29.8
|
|
Work Productivity and Activity Impairment (WPAI) Score
Work Productivity Loss
|
34.63 score on a scale
Standard Deviation 30.97
|
29.54 score on a scale
Standard Deviation 30.73
|
SECONDARY outcome
Timeframe: From a year before study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
Outcome measures
| Measure |
Narcolepsy Type 1
n=139 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Number of Participants Categorized Based on Occupational Accidents
No
|
131 Participants
|
51 Participants
|
|
Number of Participants Categorized Based on Occupational Accidents
Yes
|
8 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: From a year before study visit (approximately 20 months)Population: Analysis population included all eligible participants whose data was retrieved and analyzed. Data was not collected for this outcome measure as data was not available in the medical records.
Cost of direct healthcare resources utilized by participants per year based on costs of following direct healthcare resources was to be included: treatment received, routine monitoring visits (specialists and GPs), tests performed, emergency rooms visits, hospitalizations and complications derived from narcolepsy, and unitary costs.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
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. Percentage of participants are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=149 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Mobility: I have slight problems in walking about
|
8.05 percentage of participants
|
15.09 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Self-Care: I have no problems washing or dressing myself
|
93.96 percentage of participants
|
90.57 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Self-Care: I have severe problems washing or dressing myself
|
0.70 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Pain / Discomfort: I have moderate pain or discomfort
|
12.08 percentage of participants
|
16.98 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Pain / Discomfort: I have extreme pain or discomfort
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Mobility: I have no problems in walking about
|
85.91 percentage of participants
|
84.91 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Mobility: I have moderate problems in walking about
|
4.70 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Mobility: I have severe problems in walking about
|
1.34 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Mobility: I am unable to walk about
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Self-Care: I have slight problems washing or dressing myself
|
4.69 percentage of participants
|
9.43 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Self-Care: I have moderate problems washing or dressing myself
|
0.70 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Self-Care: I am unable to wash or dress myself
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Usual activities: I have no problems doing my usual activities
|
51.01 percentage of participants
|
58.49 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Usual activities: I have slight problems doing my usual activities
|
17.45 percentage of participants
|
24.53 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Usual activities: I have moderate problems doing my usual activities
|
24.83 percentage of participants
|
16.98 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Usual activities: I have severe problems doing my usual activities
|
6.71 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Usual activities: I am unable to do my usual activities
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Pain / Discomfort: I have no pain or discomfort
|
63.09 percentage of participants
|
67.92 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Pain / Discomfort: I have slight pain or discomfort
|
17.45 percentage of participants
|
13.21 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Pain / Discomfort: I have severe pain or discomfort
|
7.38 percentage of participants
|
1.89 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Anxiety / Depression: I am not anxious or depressed
|
48.99 percentage of participants
|
52.83 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Anxiety / Depression: I am slightly anxious or depressed
|
26.18 percentage of participants
|
20.75 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Anxiety / Depression: I am moderately anxious or depressed
|
18.79 percentage of participants
|
20.75 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Anxiety / Depression: I am severely anxious or depressed
|
4.03 percentage of participants
|
5.66 percentage of participants
|
|
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire
Anxiety / Depression: I am extremely anxious or depressed
|
2.01 percentage of participants
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis.
SSCl-8 is an 8 items scale (2 items measuring internalized stigma, 5 items measuring enacted stigma, and 1 item, which exhibited split loading, measuring either or both internalized and enacted stigma) developed to assess internalized and enacted/experienced stigma across neurological conditions. It uses a 5-point Likert scale ranging from 1 (never) to 5 (always) for scoring each item. The total score is calculated by summing individual items, ranging from 8 to 40. Higher scores indicate worsening of condition.
Outcome measures
| Measure |
Narcolepsy Type 1
n=149 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=58 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Stigma Scale for Chronic Illness 8-item Version (SSCI-8) Total Score
|
15.5 score on a scale
Standard Deviation 6.1
|
14.1 score on a scale
Standard Deviation 5.5
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. Overall number of participants analyzed is the number of participants available for analysis. Number analyzed is the number of participants available for analysis in the specified data set.
TSQM-9 measures participant satisfaction with the medication in 3 domains: Effectiveness, convenience, and global satisfaction. The scores were computed by adding items for each domain, i.e., 1 to 3 for effectiveness, 4 to 6 for convenience, and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) x 3 items = 18 for effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. TSQM-9 domain scores range from 0 to 100, with higher scores indicating greater satisfaction for that domain.
Outcome measures
| Measure |
Narcolepsy Type 1
n=141 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=46 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Treatment Satisfaction of Participants Assessed by Treatment Satisfaction Questionnaire for Medication (TSQM-9)
Effectiveness
|
67.53 score on a scale
Standard Deviation 19.12
|
63.05 score on a scale
Standard Deviation 21.19
|
|
Treatment Satisfaction of Participants Assessed by Treatment Satisfaction Questionnaire for Medication (TSQM-9)
Convenience
|
74.21 score on a scale
Standard Deviation 19.99
|
73.06 score on a scale
Standard Deviation 16.41
|
|
Treatment Satisfaction of Participants Assessed by Treatment Satisfaction Questionnaire for Medication (TSQM-9)
Global Satisfaction
|
68.54 score on a scale
Standard Deviation 20.33
|
63.36 score on a scale
Standard Deviation 24.92
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
Percentage of participants are rounded-off to the nearest decimal point.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Depression
|
14.65 percentage of participants
|
7.55 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Bipolar disorder
|
0 percentage of participants
|
1.89 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Anxiety disorders
|
12.74 percentage of participants
|
7.55 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Post-traumatic stress disorder
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Panic disorder
|
0 percentage of participants
|
1.89 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Phobia disorder
|
0.64 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Obsessive compulsive disorder
|
0.64 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Diagnosis of attention deficit hyperactivity disorder (ADHD)
|
1.91 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Obesity
|
22.29 percentage of participants
|
15.09 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Endocrine disorders
|
7.01 percentage of participants
|
9.43 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Myocardial Infarction
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Urinary Incontinence
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Congestive heart failure (CHF)
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Peripheral vascular disease
|
1.27 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Cardiovascular accident or transient ischemic attack (TIA)
|
0.64 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Dementia
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Chronic obstructive pulmonary disease (COPD)
|
0.64 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Connective tissue disease
|
0.64 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Peptic ulcer disease
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Liver Disease
|
1.27 percentage of participants
|
1.89 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Diabetes Mellitus
|
1.91 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Hemiplegia
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Moderate to severe chronic kidney disease (CKD)
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Solid tumor
|
2.55 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Leukemia
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Lymphoma
|
0 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Acquired Immunodeficiency Syndrome (AIDS)
|
0.64 percentage of participants
|
0 percentage of participants
|
|
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy
Others
|
17.20 percentage of participants
|
9.43 percentage of participants
|
SECONDARY outcome
Timeframe: From a year before narcolepsy diagnosis up to study visit (up to 20 months)Population: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments.
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.
Outcome measures
| Measure |
Narcolepsy Type 1
n=157 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
n=53 Participants
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Charlson Comorbidity Index (CCI)
|
0.4 score on a scale
Standard Deviation 1.1
|
0.2 score on a scale
Standard Deviation 0.7
|
SECONDARY outcome
Timeframe: Up to 20 monthsPopulation: SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and statistical analysis plan (SAP) the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, a new disease or worsening in severity or frequency of a concomitant disease, temporally associated with the use of a medicinal product, whether or not the event is considered causally related to the use of the product.
Outcome measures
| Measure |
Narcolepsy Type 1
n=210 Participants
Participants with confirmed diagnosis of NT1 defined by the ICDS-3 were observed for up to 16 months.
|
Narcolepsy Type 2
Participants with confirmed diagnosis of NT2 defined by the ICDS-3 were observed for up to 16 months.
|
|---|---|---|
|
Number of Participants With Adverse Events (AEs)
|
11 Participants
|
—
|
Adverse Events
Overall
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Overall
n=210 participants at risk
Participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) since diagnosis to study visit (up to 12 months).
|
|---|---|
|
Cardiac disorders
Tachycardia
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Injury, poisoning and procedural complications
Fall
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Metabolism and nutrition disorders
Cell death
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Nervous system disorders
Dizziness
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Psychiatric disorders
Confusional state
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Psychiatric disorders
Enuresis
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Psychiatric disorders
Nervousness
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.95%
2/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.48%
1/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
|
Vascular disorders
Hypertension
|
0.95%
2/210 • Up to 20 months
SAF contained all enrolled participants who received at least one dose of narcolepsy treatments. As pre-specified in the protocol and SAP the adverse events were collected for overall participants who received any narcolepsy treatment (Pharmacological or non-pharmacological) irrespective of the narcolepsy type.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place