TElemedicine for NARcolepsy

Study Overview
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Brief Summary
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The TENAR trial is the first randomized controlled trial (RCT) designed to evaluate the feasibility, efficacy, safety, and costs of a Telemedicine multidisciplinary approach for the management of narcolepsy. Open RCT assessing the non-inferiority of the multidisciplinary management of narcolepsy via Video Consultation (VC) through Mobile Telemedicine devices compared to usual in-office care. 202 children and adults with narcolepsy will be randomly allocated in 1:1 ratio to VC or in-office usual care for a 12 months follow-up. At baseline, all patients will undergo a neurologic, metabolic, and psychosocial assessment. Primary (i.e., excessive daytime sleepiness according to the Epworth Sleepiness Scale) and secondary endpoints (i.e., other symptoms, metabolic control, quality of life, patient and family satisfaction with care, feasibility, safety, and costs) will be measured at 6 and 12 months. The investigators expect the Telemedicine approach not only to be non-inferior for sleepiness control but also to significantly improve other patient-centred outcomes compared to the usual in-office care.

Official Title
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Telemedicine With Mobile Internet Devices for Innovative Multidisciplinary Patient-centred Care of Patients With Narcolepsy

Conditions
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Narcolepsy

Intervention / Treatment
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* Procedure: Tele-multidisciplinary care
* Other: Standard care


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: • Adolescents (≥14 years old) and adults with a diagnosis of narcolepsy according to ICSD-3 criteria at their first (incident) or with established diagnosis (prevalent subjects), able to provide consent Exclusion Criteria: inability to read, write, or using a tablet; major psychiatric disorders.

Ages Eligible for Study
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Minimum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Tele-multidisciplinary participants<br>Patients undergoing televisit | Procedure: Tele-multidisciplinary care<br>* Scheduled televisit by sleep medicine specialists, endocrinologists, legal-medicine specialists<br>|
| Other: In-office standard participants<br>Patients undergoing in-office visits | Other: Standard care<br>* Scheduled in-office visit by sleep medicine specialists, endocrinologists, legal-medicine specialists<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| excessive daytime sleepiness | Measured by the Epworth Sleepiness Scale. The Epworth Sleepiness Scale is a self-administered form with eight items investigating sleep propensity in different real-life situations during the preceding months. Each item can received 0-3 points, thus the final score ranges from 0 (best score) to 24 (worse score). The proposed range for normal subjects is 0-10 | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| metabolic control - weight Changes | metabolic control - weight Changes of weight in kilograms. [Time frame: 12 months] | 12 months | 
| metabolic control - lipid profile Changes | metabolic control - lipid profile Changes of: total cholesterol; high-density lipoprotein cholesterol; low-density lipoprotein cholesterol; total triglycerides. All are measured in milligrams per deciliter (mg/dl). [Time frame: 12 months] | 12 months | 
| metabolic control - glycemic profile Changes | metabolic control - glycemic profile Changes of fasting glycemia measured in milligrams per deciliter (mg/dl). [Time frame: 12 months] | 12 months | 
| metabolic control - caloric intake Changes | metabolic control - caloric intake Changes of caloric intake measured by a food diary. [Time frame: 12 months] | 12 months | 
| metabolic control - physical activity Changes | metabolic control - physical activity Changes of physical activity measured by the International Physical Activity Questionnaire (IPAQ). Total physical activity is measured in MET-min/week and time spent sitting, and it is ranked in three levels: low; moderate; high. [Time frame: 12 months] | 12 months | 
| quality of life - Short-Form 36-Item Questionnaire | Quality of life - Short-Form 36-Item Questionnaire (SF-36) Changes of SF-36 health related quality of life (HRQOL) scales. This tool presents 36 questions. Eight health domains are assessed (physical activities; social activities; limitations in role activities because of physical health problems; bodily pain; mental health; limitations in role activities because of emotional problems; vitality; general health perceptions). Each domain measures a level of HRQOL with a scale between 0-100, with 0 being bad quality of life and 100 being good. [Time frame: 12 months] | 12 months | 
| quality of life - Pediatric Quality of Life Inventory | Quality of life - Pediatric Quality of Life Inventory (PedsQL) Changes of PedsQL health related quality of life (HRQOL) scales. This is a tool of 23 questions comprising four generic domains: physical functioning; emotional functioning; social functioning; school functioning. These domains are measured by a 0-100 scale. Higher scores indicate better HRQOL. [Time frame: 12 months] | 12 months | 
| patient and family satisfaction with care | patient and family satisfaction with care, measured by an ad-hoc devised form (named Co-Tenar), assessing qualitatively the satisfaction level. | 12 months | 
| safety | safety, in terms of number (percent) of full drop-outs, partial drop-outs (patients changing procedure), adverse reactions to drugs in both group of intervention (standard in-office visit arm, video-consultation arm). | 12 months | 
| costs | costs related to the disease management (drugs, tests, hospitalization, journeys) will be assessed, by means of a standardized interview, in both group of intervention (standard in-office visit arm, video-consultation arm). | 12 months |