nNO Testing for Primary Ciliary Dyskinesia

Overseen By Michael O'Connor, MD

Age: Any Age

Sex: Any

Trial Phase: Academic

Sponsor: Vanderbilt University Medical Center

Disqualifiers: Age less than 2, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

200 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to find better ways to diagnose Primary Ciliary Dyskinesia (PCD), a rare genetic disorder affecting breathing. It evaluates the effectiveness of current tests, such as nasal nitric oxide (nNO) testing, for diagnosing this condition. The trial gathers key health data and performs nNO tests on some participants. It suits individuals referred for PCD diagnosis who can follow the study procedures. As an unphased trial, it offers participants the chance to contribute to important research that could enhance diagnostic methods for PCD.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications.

What prior data suggests that these PCD diagnostic tests are safe?

Research has shown that testing nasal nitric oxide (nNO) is generally safe. This test measures the amount of nitric oxide in the nose and aids in diagnosing primary ciliary dyskinesia (PCD), a rare genetic disorder. Studies have found that individuals with PCD usually have lower nNO levels, making this test useful for identifying the condition.

Importantly, nNO testing is easy to tolerate, with most people experiencing no discomfort or side effects. It does not involve needles or invasive procedures, which many find reassuring. So far, there have been no major reports of negative effects, making nNO testing a safe choice for those considering participation in clinical trials or screening for PCD. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about nasal nitric oxide (nNO) testing for Primary Ciliary Dyskinesia (PCD) because it offers a non-invasive way to diagnose this rare genetic disorder. Unlike traditional methods like genetic testing or ciliary biopsy, which can be invasive and time-consuming, nNO testing is quick and easy, providing immediate results. This method not only helps in confirming PCD but also aids in ongoing monitoring of the condition, making it a potentially game-changing tool for both patients and healthcare providers.

What evidence suggests that nasal nitric oxide testing is effective for diagnosing Primary Ciliary Dyskinesia?

Research has shown that measuring nasal nitric oxide (nNO) levels is useful for diagnosing primary ciliary dyskinesia (PCD). Studies have found that individuals with PCD typically exhibit very low nNO levels, making this test a simple and non-invasive diagnostic tool. In this trial, participants will undergo nNO testing, and researchers will collect clinical data to evaluate its effectiveness in diagnosing PCD. The consistently low nNO levels in PCD patients offer a reliable indicator for detection, aiding doctors in identifying PCD early and accurately. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

This trial is for individuals suspected of having Primary Ciliary Dyskinesia (PCD) and are referred to a center for diagnosis. Participants must be able to perform the required study procedures and be older than 2 years. Those under 2 or unable to give informed consent cannot join.

Inclusion Criteria

Referred to center for PCD diagnosis considerations

I can follow the study's required procedures.

I am older than 2 years.

Exclusion Criteria

I am under 2 years old.

I am unable to understand and give consent for treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Data Collection

Participants have clinical data collected and may undergo nasal nitric oxide (nNO) testing

3 years

Follow-up

Participants are monitored for safety and effectiveness after data collection

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PCD Diagnostic Tests

Trial Overview The study is evaluating the effectiveness of current diagnostic tests for PCD, focusing on nasal nitric oxide (nNO) testing, which measures the amount of nitric oxide in breath as an indicator of ciliary function.

How Is the Trial Designed?

Group I: Nasal Nitric Oxide testing and collection of clinical data Experimental Treatment 1 Intervention

Participants will have nNO testing is indicated. All participants in this study have some basic clinical data collected at time of enrollment. Participants with a confirmed diagnosis of PCD or in those participants with a working diagnosis of PCD in which ongoing nNO testing is performed have prospective data collection. Some participants have a confirmed diagnosis of PCD by genetics or ciliary biopsy at time of study entry and thus do not need nNO testing, but are followed prospectively with collection of basic clinical data

Who Is Running the Clinical Trial?

Vanderbilt University Medical Center

Lead Sponsor

Trials

922

Recruited

939,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12171850/

Nasal nitric oxide measurement for the diagnosis of ... Nasal nitric oxide (nNO) measurement is important in the primary ciliary dyskinesia (PCD) diagnostic pathway because levels are consistently very low in most ...

2. publications.ersnet.org publications.ersnet.org/content/erj/44/6/1589

Nasal nitric oxide screening for primary ciliary dyskinesia Abstract. Nasal nitric oxide (nNO) concentrations are low in patients with primary ciliary dyskinesia (PCD) providing a noninvasive screening test.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05287022

Use of Nasal Nitric Oxide Testing in Improving Primary ... 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.

4. withpower.com withpower.com/trial/nasal-nitric-oxide-test-for-primary-ciliary-dyskinesia-ca6f2

Nasal Nitric Oxide Test for Primary Ciliary Dyskinesia Nasal nitric oxide testing is a non-invasive method used to diagnose primary ciliary dyskinesia and is generally considered safe for humans, including children ...

5. mdpi.com mdpi.com/1422-0067/24/22/16159

Role of Nasal Nitric Oxide in Primary Ciliary Dyskinesia ... We conducted a narrative review of the literature to examine the relationship between nNO and some respiratory diseases with a particular focus on primary ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12046229/

Impact of Aerobic Exercise on Oxygenation, Pulmonary ... Primary Ciliary Dyskinesia (PCD) is a rare genetic disorder characterized by impaired mucociliary clearance, resulting in chronic respiratory complications.

7. sciencedirect.com sciencedirect.com/science/article/pii/S2950008725000420

Primary ciliary dyskinesia: A review Introduction. Primary ciliary dyskinesia (PCD) is a rare genetic disorder that affects the functions of the cilia lining, primarily the respiratory epithelium.

8. icliniq.com icliniq.com/articles/genetic-disorders/nasal-nitric-oxide-measurement-as-a-test-for-primary-ciliary-dyskinesia

Can Nasal Nitric Oxide Measurement Tests Help in ... Numerous studies indicate that nasal nitric oxide (nNO) levels are significantly lower in individuals with primary ciliary dyskinesia (PCD) ...

9. nhlbi.nih.gov nhlbi.nih.gov/health/primary-ciliary-dyskinesia/diagnosis

Primary Ciliary Dyskinesia - Diagnosis - NHLBI - NIH Your healthcare provider or your child's provider will diagnose primary ciliary dyskinesia (PCD) based on your symptoms, family history, and test results.

10. discovery.dundee.ac.uk discovery.dundee.ac.uk/files/72718147/1_s2.0_S0272523121012466_main.pdf

University of Dundee Diagnosis of Primary Ciliary Dyskinesia ... They present an overview of the diagnostic pathway for PCD and discuss why an accurate diagnosis is important. CLINICAL FEATURES OF PRIMARY CILIARY. DYSKINESIA.