Development and Validation of Pediatric Narcolepsy Patien... | NCT05373979

Studies
Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale

NCT05373979

Sponsor

Boston Children's Hospital

Status

Completed

Last Update Posted

Feb 13, 2025Actual

Enrollment

212Actual

Phase

Not provided

Conditions

Narcolepsy
Obstructive Sleep Apnea

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2025

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 2, 2019Actual

Primary Completion Date

May 30, 2024Actual

Completion Date

May 30, 2024Actual

First Submitted Date

May 10, 2022

First Submission Date that Met QC Criteria

May 10, 2022

First Posted Date

May 13, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 11, 2025

Last Update Posted Date

Feb 13, 2025Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

Kiran Maski, Principal Investigator, Boston Children's HospitalPrincipal Investigator

Lead Sponsor

Boston Children's HospitalOTHER

Collaborators

Name	Class
National Jewish Health	OTHER
Geisinger Clinic	OTHER
The Hospital for Sick Children	OTHER
Stanford University

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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.

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

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

212Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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)

Interventions

Not provided

Outcomes Module

Primary Outcomes

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

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

9 Years

Maximum Age

17 Years

Standard Ages

Child

Study Population

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

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Kiran Maski, MD, MPH	Boston Children's Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Boston Children's Hospital		Boston	Massachusetts	02115	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

No individual participant data will be shared with other researchers outside of Boston Children's Hospital.

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D009290	Narcolepsy
D020181	Sleep Apnea, Obstructive

Ancestor Terms

ID	Term
D006970	Disorders of Excessive Somnolence
D020919	Sleep Disorders, Intrinsic
D020920	Dyssomnias
D012893	Sleep Wake Disorders

Browse Leaves

Not provided

Browse Branches

Not provided