Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale

NCT05373979 | Completed

• 1 other identifier: IRB-A00029346
• Study Type: observational
• Target: 212
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Narcolepsy; Obstructive Sleep Apnea

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

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)

Eligibility Criteria

• Age: 9 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Boston Children's Hospital: lead
• National Jewish Health: collaborator
• Geisinger Clinic: collaborator
• The Hospital for Sick Children: collaborator
• Stanford University: collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Narcolepsy; Sleep Apnea, Obstructive

Condition Hierarchy (Ancestors)

Disorders of Excessive Somnolence; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Mental Disorders; Sleep Apnea Syndromes; Apnea; Respiration Disorders; Respiratory Tract Diseases

Study Officials

• Kiran Maski, MD, MPH

  Boston Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: May 10, 2022
• First Posted: May 13, 2022
• Study Start: April 2, 2019
• Primary Completion: May 30, 2024
• Study Completion: May 30, 2024
• Last Updated: February 13, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)