Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale (PN-PROS)

Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale (PN-PROS)

The purpose of this study is to test a pediatric narcolepsy patient reported outcomes tool to assess pediatric narcolepsy symptoms and their effect on daily functioning and quality of life. The goal is to develop a clinical survey that can improve the care of pediatric narcolepsy.

Study Overview

• Status: Completed
• Conditions: Obstructive Sleep Apnea; Narcolepsy

Detailed Description

WHO IS ELIGIBLE?

• Children and adolescents with narcolepsy between the ages of 9-17 years
• Diagnosis must be verified by a signed letter from physician in order to participate
• Participants must be able to understand the purpose of the study

PARTICIPATION DETAILS

• Upon enrollment participants will receive an email containing a link to complete HIPAA compliant online surveys about narcolepsy symptoms, quality of life, and daily function. Surveys should take no more than 30 minutes.
• One week later, some participants will receive another email with a link to complete a 10-minute follow up survey

• Study Type: Observational
• Enrollment (Actual): 212

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 9 years to 17 years (Child)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients (and a parent/guardian) with an established narcolepsy type 1, narcolepsy type 2, or OSA diagnoses ages 9-17 years.

Description

Inclusion Criteria:

• Children and adolescents ages 9-17
• Have a diagnosis of narcolepsy type 1, narcolepsy type 2, or mild to moderate obstructive sleep apnea (OSA)

Exclusion Criteria:

• A history of visual or hearing impairment
• A co-morbid neurodevelopmental disorder such as autism or schizophrenia
• No access to computer/tablet/smart phone to complete questionnaires

Study Plan

How is the study designed?

Design Details

• Observational Models: Case-Control
• Time Perspectives: Prospective

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Narcolepsy; Narcolepsy type 1 (NT1 or narcolepsy with cataplexy) and Narcolepsy type 2 (NT2 or narcolepsy without cataplexy)
Obstructive Sleep Apnea; mild to moderate obstructive sleep apnea (OSA; obstructive AHI of 1-15/hour)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survey Validation	To evaluate the content validity and reliability of the PN-PROs item pools through a field test of the item pools in a diverse, national sample of pediatric narcolepsy patients with a comparator group of pediatric mild obstructive sleep apnea patients (9-17 years).	2 Years

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Collaborators: The Hospital for Sick Children; Stanford University; Geisinger Clinic; National Jewish Health
• Investigators: Principal Investigator: Kiran Maski, MD, MPH, Boston Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 2, 2019
• Primary Completion (Actual): May 30, 2024
• Study Completion (Actual): May 30, 2024

Study Registration Dates

• First Submitted: May 10, 2022
• First Submitted That Met QC Criteria: May 10, 2022
• First Posted (Actual): May 13, 2022

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 11, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Mental Disorders; Respiratory Tract Diseases; Respiration Disorders; Sleep Wake Disorders; Apnea; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Apnea Syndromes; Disorders of Excessive Somnolence; Sleep Apnea, Obstructive; Narcolepsy
• Other Study ID Numbers: IRB-A00029346

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: No individual participant data will be shared with other researchers outside of Boston Children's Hospital.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No