NarcolepsyPN-PROSNCT05373979
Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale
An observational study of Narcolepsy and Obstructive Sleep Apnea, run by Boston Children's Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 9 to 17 years
- Conditions the study is for
- Narcolepsy, Obstructive Sleep Apnea
- How many people
- 212 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Apr 2019to 30 May 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Patients (and a parent/guardian) with an established narcolepsy type 1, narcolepsy type 2, or OSA diagnoses ages 9-17 years.
Who may be able to join
- Children and adolescents ages 9-17
- Have a diagnosis of narcolepsy type 1, narcolepsy type 2, or mild to moderate obstructive sleep apnea (OSA)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
Official title: 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.
Read the 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
- Narcolepsy, Obstructive Sleep Apnea
- Medical subject headings
- Narcolepsy, Sleep Apnea, Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2022
- Study start2 Apr 2019
- Primary completion30 May 2024
- Study completion30 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NarcolepsyStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 212 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Jewish Health, Geisinger Clinic, The Hospital for Sick Children, Stanford University
- Responsible party
- Principal investigator, Kiran Maski, Boston Children's Hospital
- Investigators
- Kiran Maski, MD, MPH, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05373979
- Sponsor's protocol ID
- IRB-A00029346
- Acronym
- PN-PROS
- Status last verified
- Feb 2025
- First submitted
- 10 May 2022
- Last updated
- 13 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No individual participant data will be shared with other researchers outside of Boston Children's Hospital.
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development and Validation of Pediatric Narcolepsy Patient Reported Outcomes Scale (PN-PROS)
- ClinicalTrials.gov ID
- NCT05373979
- Acronym / sponsor ID
- PN-PROS / IRB-A00029346
- Conditions
- Narcolepsy, Obstructive Sleep Apnea
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 212 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- National Jewish Health, Geisinger Clinic, The Hospital for Sick Children, Stanford University
- Study start
- 2 Apr 2019 (actual)
- Primary completion
- 30 May 2024 (actual)
- Study completion
- 30 May 2024 (actual)
- First posted
- 13 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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) |
Who may qualify
- Ages
- 9 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Patients (and a parent/guardian) with an established narcolepsy type 1, narcolepsy type 2, or OSA diagnoses ages 9-17 years.
Key inclusion criteria (2 listed)
- Children and adolescents ages 9-17
- Have a diagnosis of narcolepsy type 1, narcolepsy type 2, or mild to moderate obstructive sleep apnea (OSA)
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Survey Validation
Time frame: 2 Years
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).
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Feb 2025.
- Individual participant dataSharing planned: No. No individual participant data will be shared with other researchers outside of Boston Children's Hospital.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kiran Maski, MD, MPH principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Narcolepsy and Obstructive Sleep Apnea, run by Boston Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Survey Validation (time frame: 2 Years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Feb 2025; AzTrials last refreshed its copy on 6 Nov 2026.