Use of Nasal Nitric Oxide Testing in Improving Primary Ciliary Dyskinesia Clinical Care

January 28, 2025 updated by: Arkansas Children's Hospital Research Institute
this study is aiming at learning more about primary ciliary dyskinesia (PCD) and tests that are used to diagnose this condition. One purpose of this study is to measure the level of nitric oxide in the nasal passages and examine how often the results correlate with other tests currently done to make the diagnosis.

Study Overview

Status

Recruiting

Conditions

Detailed Description

This will be an observational study. Participants who are referred by their clinician for nasal NO testing and meet the inclusion and exclusion criteria will be allowed to undergo testing. Informed consent will be completed prior to testing. The participant and/or guardian will be informed that this testing is not a clinically approved test at this time and thus it cannot confirm diagnosis of PCD. This test is to correlate with other diagnostic tests so that in the future it can be an approved diagnostic test. Data will be collected from the electronic medical records regarding participants.

The data collected will be stored on a REDCap (Research Electronic Data Capture) registry. The registry will contain participants' direct identifiers (like medical record). De-identified data will be used from the registry for monitoring, further analysis and presentations. The results of the testing may be included in the medical chart of each participant as part of the diagnosis discussion with family.

Consent forms will be obtained from participants >18 years of age or who are emancipated minors, or guardian for patients <18 years of age. Guardian refers to parent, guardian, or legally authorized personnel throughout this document.

The data collected will be used to refine and improve the diagnostic testing for PCD at Arkansas Children's Hospital. This will also involve collaboration with other sub-specialties like otolaryngology and allergy/immunology. With continued research regarding nasal nitric oxide testing, this may involve the use of this test as a screening tool for PCD in the right clinical setting. This will involve analyzing the association of nasal nitric oxide levels with approved diagnostic tests (ciliary biopsy and genetic testing).

Testing will be completed using (Eco-Physics Analyzer CLD 88 sp with DENOX 88). this testing will be done in the Arkansas Children's hospital pulmonary function laboratory, once all training is completed.

This equipment is not currently FDA approved for PCD diagnostic testing and therefore, referring clinicians will be informed of the results of this test and its limitations as a research tool will be discussed. However, there is growing evidence that this testing has utility as a non-invasive screening tool for PCD and continued data from the general pediatric pulmonary population is needed.

In addition to being non-invasive testing, it has very little risk as it simply monitors nasal nitric oxide levels in the sinus cavities during regular breathing without sedation. There is ongoing research to better determine normative values and thresholds for further PCD confirmatory testing, especially in the younger population. In the general population this testing has a greater negative predictive value than a positive predictive value. Consequently, the testing will be done on individuals who have at least two of the following clinical criteria for PCD: history of neonatal respiratory distress, chronic rhinitis, chronic cough, or situs defects.

Study Type

Observational

Enrollment (Estimated)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
        • Contact:
          • Zena Ghazala

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 21 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants will include individuals who are diagnosed with PCD or undergoing work-up for primary ciliary dyskinesia based on their clinical symptoms as determined by the primary pulmonologist or other specialist

Description

Inclusion Criteria:

  • Individuals who are diagnosed with Primary Ciliary Dyskinesia OR
  • Individuals with abnormal PCD diagnostics (abnormal ciliary biopsy or PCD genetics) OR
  • Individuals with unexplained bronchiectasis OR

  • Individuals undergoing PCD diagnostic testing (ciliary biopsy, PCD genetic testing) or concern based on clinical symptoms (at least two of the following):

    • Neonatal respiratory distress
    • Organ laterality defects
    • Year-round cough starting in first year of life or bronchiectasis on chest CT
    • Year-round nasal congestion starting in first year of life or pansinusitis
    • Multiple ear infections in the first two years of life with sequelae (e.g. ear tubes, chronic effusion, abnormal audiological exam)
    • History of recurrent pneumonias (at least 2 in one year or more than 3 at any time)
  • Ability to provide consent for participation in study by the participants or guardian
  • Ability to perform the test
  • Age >= 2 years of age

Exclusion Criteria:

  • Individuals who are unable to understand the requirements of the study.
  • Individuals (or guardians) who are unwilling to provide consent.
  • Individuals who are unable to complete the testing
  • Recent history of sinus surgery (within four weeks) or bloody nose (within one week) of testing (they can be included at a later date)
  • Patients who are currently being treated (within one week) with antibiotics for sinusitis or respiratory symptoms (they can be included at a later date)
  • Age <2 years of age

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Measuring nasal nitric oxide in comparison to ciliary biopsy and genetic testing for the diagnosis of PCD
Time Frame: throughout the study, an average of 3 years
Individuals with PCD have characteristically low values of nNO; values below an established cut-off of 77nL/min are strongly associated with PCD in compatible clinical settings. These values are not well established for individuals who are in 2-5 years of age. nNO measurements (in nL/min) in 2-5 years of age will be compared to the diagnosis of PCD and assessed to establish diagnostic cut-offs.
throughout the study, an average of 3 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Comparing tidal breathing to resistor method
Time Frame: throughout the study, an average of 3 years
comparing nNO values in nL/min obtained via tidal breathing technique to those obtained via resistor method.
throughout the study, an average of 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Arkansas Children's Hospital Research Institute

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 14, 2021

Primary Completion (Estimated)

June 1, 2030

Study Completion (Estimated)

June 1, 2030

Study Registration Dates

First Submitted

April 9, 2021

First Submitted That Met QC Criteria

March 10, 2022

First Posted (Actual)

March 18, 2022

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

January 28, 2025

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 260432

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Primary Ciliary Dyskinesia

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Venezuela

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe