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Utility of PCD Diagnostics to Improve Clinical Care

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ClinicalTrials.gov Identifier: NCT05889013
Recruitment Status : Recruiting
First Posted : June 5, 2023
Last Update Posted : October 2, 2023
Sponsor:
Collaborator:
UConn Health
Information provided by (Responsible Party):
Connecticut Children's Medical Center

Study Description
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Brief Summary:
This cross-sectional and longitudinal observational study is to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD). There is new testing available, called nasal nitric oxide testing, that non-invasively measures nitric oxide levels in the sinus cavity. Individuals with PCD characteristically have low levels, but this testing does not have extensive data from everyday clinical practice. The objective of this proposal is to improve the diagnostic approach to children and adults with clinical concerns for primary ciliary dyskinesia (PCD).

Condition or disease Intervention/treatment
Primary Ciliary Dyskinesia Device: Nasal Nitric Oxide testing

Detailed Description:
This is an observational study to gather data on the utility of tests that are used to make a diagnosis of primary ciliary dyskinesia (PCD). Participant selection will be on a referral basis for the consideration of a clinical suspicion for PCD. Participants will be identified by pulmonary providers that consider testing for PCD based on the clinical symptoms of the participant. These providers will then refer for enrollment in the study based on the clinical considerations for a diagnosis of PCD. Participants will be consented during the time of testing at the Pulmonary department. Any patient referred by a clinician for concerns for PCD, including those who have had PCD genetics sent, or a ciliary biopsy performed will be enrolled in the study. In addition, any participant who is referred for nasal nitric oxide testing and meets the inclusion and exclusion criteria for this testing, will also need to provide informed consent and be enrolled in the Nasal NO REDCap registry. This equipment is not currently FDA approved or CLIA certified for PCD diagnostic testing and therefore referring clinicians will be informed that the results of this test should not influence clinical decision making regarding the diagnosis of PCD. 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.
Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Target Follow-Up Duration: 3 Years
Official Title: A Clinical Study to Gather Data on the Utility of Tests That Are Used to Make a Diagnosis of Primary Ciliary Dyskinesia (PCD)
Actual Study Start Date : September 27, 2023
Estimated Primary Completion Date : June 2024
Estimated Study Completion Date : June 2024

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Nasal Nitric Oxide
Participants who are referred by his/her clinician for nasal NO testing and meet the inclusion and exclusion criteria will undergo testing. Clinical information regarding prior diagnostic testing will be collected at time of enrollment. Participants that have a confirmed diagnosis of PCD by genetics or ciliary biopsy at time of study entry
 Device: Nasal Nitric Oxide testing
Collection of already performed clinical data and nNO testing
Other Name: nNO testing


Outcome Measures
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Primary Outcome Measures :
  1. Creation of Nasal NO REDCap registry [ Time Frame: 2-3 years ]
    To establish a research registry to characterize the features of individuals that undergo PCD diagnostic testing at Connecticut Children's Medical Center and UCONN.

  2. Evaluation of utility of PCD diagnostic testing [ Time Frame: 2-3 years ]
    To evaluate the utility of PCD diagnostic testing, including potential use of nasal nitric oxide in comparison to genetic testing and ciliary biopsy, in pulmonary practice at Connecticut Children's Medical Center and UCONN.


Secondary Outcome Measures :
  1. Refinement and Improvement of PCD Diagnostic Testing [ Time Frame: 1-2 years ]
    To refine and improve current diagnostic processes for PCD diagnosis at CCMC using information gathered from the research registry established through Primary aims 1 and 2.


Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The target population are ages 2 and up. Participant selection will be on a referral basis for the consideration of a clinical suspicion for PCD. Participants will be identified by pulmonary providers that consider testing for PCD based on the clinical symptoms of the participant. These providers will thenrefer for enrollment in the study based on the clinical considerations for a diagnosis of PCD.
Criteria

Inclusion Criteria:

  • Age ≥ 2 years of age
  • Must have two of the following clinical history points:
  • Neonatal respiratory distress
  • Chronic nasal congestion/runny nose
  • Chronic cough
  • Situs/laterality defects
  • Bronchiectasis
  • Ability to provide informed consent or consent of parent/guardian and ass

Exclusion Criteria:

  • Recent history of sinus surgery or bloody nose in the past week
  • Age < 2 years of age
  • Inability to tolerate probe in nose
  • Sinusitis or other respiratory exacerbation currently being treated with antibiotics
  • Admitted to hospital for respiratory exacerbation (inpatient status)
  • Inability to understand the requirements of the study or be unwilling to provide written informed consent (as evidenced by signature on an informed consent document approved by the IRB)
  • Any other reason for which the study investigators feel the patient is not a good candidate to complete the testing
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05889013


Contacts
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Contact: Melanie S Collins, MD 860-545-9440 mcollin@connecticutchildrens.org
Contact: Sigrid M Almeida, BS 8608375343 salmeida@connecticutchildrens.org

Locations
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United States, Connecticut
Pulmonary Division Recruiting
Hartford, Connecticut, United States, 06111
Contact: Melanie S Collins, MD    860-545-9440    mcollin@connecticutchildrens.org   
Contact: Sigrid M Almeida, BS    8608375343    salmeida@connecticutchildrens.org   
Sponsors and Collaborators
Connecticut Children's Medical Center
UConn Health
Investigators
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Principal Investigator: Melanie S Collins, MD Connecticut Children's Medical Center
More Information
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Responsible Party: Connecticut Children's Medical Center
ClinicalTrials.gov Identifier: NCT05889013    
Other Study ID Numbers: 23-052
First Posted: June 5, 2023    Key Record Dates
Last Update Posted: October 2, 2023
Last Verified: August 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Connecticut Children's Medical Center:
PCD
nasal nitric oxide testing
Additional relevant MeSH terms:
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Ciliary Motility Disorders
Dyskinesias
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Respiratory Tract Diseases
Otorhinolaryngologic Diseases
Ciliopathies
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn
Nitric Oxide
Bronchodilator Agents
 Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Endothelium-Dependent Relaxing Factors
Vasodilator Agents
Gasotransmitters
Protective Agents