Nytte av PCD-diagnostikk for å forbedre klinisk behandling (PCD)
En klinisk studie for å samle data om nytten av tester som brukes til å stille en diagnose av primær ciliær dyskinesi (PCD)
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).
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Antatt)
Kontakter og plasseringer
Studiekontakt
- Navn: Melanie S Collins, MD
- Telefonnummer: 860-545-9440
- E-post: mcollin@connecticutchildrens.org
Studer Kontakt Backup
- Navn: Sigrid M Almeida, BS
- Telefonnummer: 8608375343
- E-post: salmeida@connecticutchildrens.org
Studiesteder
-
-
Connecticut
-
Hartford, Connecticut, Forente stater, 06111
- Rekruttering
- Pulmonary Division
-
Ta kontakt med:
- Sigrid M Almeida, BS
- Telefonnummer: 8608375343
- E-post: salmeida@connecticutchildrens.org
-
Ta kontakt med:
- Melanie S Collins, MD
- Telefonnummer: 8605459440
- E-post: mcollin@connecticutchildrens.org
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Prøvetakingsmetode
Studiepopulasjon
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.
Beskrivelse
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
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Observasjonsmodeller: COHORT
- Tidsperspektiver: CROSS_SECTIONAL
Kohorter og intervensjoner
Gruppe / Kohort |
Intervensjon / Behandling |
|---|---|
|
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 |
Collection of already performed clinical data and nNO testing
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Creation of Nasal NO REDCap registry
Tidsramme: 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. |
2-3 years
|
|
Evaluation of utility of PCD diagnostic testing
Tidsramme: 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. |
2-3 years
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Refinement and Improvement of PCD Diagnostic Testing
Tidsramme: 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. |
1-2 years
|
Samarbeidspartnere og etterforskere
Samarbeidspartnere
Etterforskere
- Hovedetterforsker: Melanie S Collins, MD, Connecticut Children's Medical Center
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- 23-052-CCMC
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
produkt produsert i og eksportert fra USA
Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .