## Protocol Section
### Identification Module
**NCT ID:** NCT05373979

**Acronym:** PN-PROS

**Brief Title:** Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale

**Official Title:** Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale (PN-PROS)

#### Organization Study ID Info
**ID:** IRB-A00029346
#### Organization
**Class:** OTHER
**Full Name:** Boston Children's Hospital
### Status Module
#### Completion Date
**Date:** 2023-04-30

**Type:** ESTIMATED

#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2022-06-01

**Type:** ACTUAL

**Last Update Submit Date:** 2022-05-26

**Overall Status:** UNKNOWN

#### Primary Completion Date
**Date:** 2023-04-30

**Type:** ESTIMATED

#### Start Date
**Date:** 2019-04-02

**Type:** ACTUAL

**Status Verified Date:** 2022-05

#### Study First Post Date
**Date:** 2022-05-13
**Type:** ACTUAL
**Study First Submit Date:** 2022-05-10
**Study First Submit QC Date:** 2022-05-10
### Sponsor Collaborators Module

#### Collaborators

**Class:** OTHER

**Name:** National Jewish Health

**Class:** OTHER

**Name:** Geisinger Clinic

**Class:** OTHER

**Name:** The Hospital for Sick Children

**Class:** OTHER

**Name:** Stanford University

#### Lead Sponsor

**Class:** OTHER

**Name:** Boston Children's Hospital

#### Responsible Party

**Investigator Affiliation:** Boston Children's Hospital

**Investigator Full Name:** Kiran Maski

**Investigator Title:** Principal Investigator

**Type:** PRINCIPAL_INVESTIGATOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** False

**Oversight Has DMC:** False

### 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

### Conditions Module

**Conditions:**

- Narcolepsy

- Obstructive Sleep Apnea

### Design Module

#### Design Info

**Observational Model:** CASE_CONTROL

**Time Perspective:** PROSPECTIVE

#### Enrollment Info

**Count:** 340

**Type:** ESTIMATED

**Study Type:** OBSERVATIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Narcolepsy type 1 (NT1 or narcolepsy with cataplexy) and Narcolepsy type 2 (NT2 or narcolepsy without cataplexy)

**Label:** Narcolepsy

#### Arm Group 2

**Description:** mild to moderate obstructive sleep apnea (OSA; obstructive AHI of 1-15/hour)

**Label:** Obstructive Sleep Apnea

### Outcomes Module

#### Primary Outcomes

**Description:** 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).

**Measure:** Survey Validation

**Time Frame:** 2 Years

### 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

**Maximum Age:** 17 Years

**Minimum Age:** 9 Years

**Sampling Method:** NON_PROBABILITY_SAMPLE

**Sex:** ALL

**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.

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** Gillian.Heckler@childrens.harvard.edu

**Name:** Gillian Heckler

**Phone:** 617-919-6212

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Boston

**Contacts:**

***Contact 1:***

- **Email:** Gillian.Heckler@childrens.harvard.edu

- **Name:** Gillian Heckler

- **Phone:** 617-919-6212

- **Role:** CONTACT

***Contact 2:***

- **Name:** Kiran Maski, MD, MPH

- **Role:** PRINCIPAL_INVESTIGATOR

**Country:** United States

**Facility:** Boston Children's Hospital

**State:** Massachusetts

**Status:** RECRUITING

**Zip:** 02115

#### Overall Officials

**Official 1:**

**Affiliation:** Boston Children's Hospital

**Name:** Kiran Maski, MD, MPH

**Role:** PRINCIPAL_INVESTIGATOR

### IPD Sharing Statement Module

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

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000012891

- Term: Sleep Apnea Syndromes

- ID: D000001049

- Term: Apnea

- ID: D000012120

- Term: Respiration Disorders

- ID: D000012140

- Term: Respiratory Tract Diseases

- ID: D000020919

- Term: Sleep Disorders, Intrinsic

- ID: D000020920

- Term: Dyssomnias

- ID: D000012893

- Term: Sleep Wake Disorders

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000006970

- Term: Disorders of Excessive Somnolence

- ID: D000001523

- Term: Mental Disorders

### Condition Browse Module - Browse Branches

- Abbrev: BC08

- Name: Respiratory Tract (Lung and Bronchial) Diseases

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BXM

- Name: Behaviors and Mental Disorders

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M4361

- Name: Apnea

- Relevance: LOW

- As Found: Unknown

- ID: M15694

- Name: Sleep Apnea Syndromes

- Relevance: LOW

- As Found: Unknown

- ID: M22010

- Name: Sleep Apnea, Obstructive

- Relevance: HIGH

- As Found: Obstructive Sleep Apnea

- ID: M12241

- Name: Narcolepsy

- Relevance: HIGH

- As Found: Narcolepsy

- ID: M16355

- Name: Syndrome

- Relevance: LOW

- As Found: Unknown

- ID: M14957

- Name: Respiration Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M14977

- Name: Respiratory Tract Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22242

- Name: Parasomnias

- Relevance: LOW

- As Found: Unknown

- ID: M22654

- Name: Sleep Disorders, Intrinsic

- Relevance: LOW

- As Found: Unknown

- ID: M22655

- Name: Dyssomnias

- Relevance: LOW

- As Found: Unknown

- ID: M15696

- Name: Sleep Wake Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M1717

- Name: Sleepiness

- Relevance: LOW

- As Found: Unknown

- ID: M10021

- Name: Disorders of Excessive Somnolence

- Relevance: LOW

- As Found: Unknown

- ID: M4815

- Name: Mental Disorders

- Relevance: LOW

- As Found: Unknown

- ID: M14473

- Name: Psychotic Disorders

- Relevance: LOW

- As Found: Unknown

- ID: T4044

- Name: Narcolepsy

- Relevance: HIGH

- As Found: Narcolepsy

### Condition Browse Module - Meshes

- ID: D000020181

- Term: Sleep Apnea, Obstructive

- ID: D000009290

- Term: Narcolepsy

### Misc Info Module

- Version Holder: 2024-05-24
