NIOX VERO Nasal Application in Primary Ciliary Dyskinesia

NCT02622061 | Completed

• 1 other identifier: AER-051
• Study Type: observational
• Target: 163
• Locations: 4 countries
• Sites: 5

Brief Summary

This is a multi-centre, single visit clinical investigation involving patients with known PCD vs. age matched healthy volunteers. This study involves 1 visit which will last one (1) to two (2) hours. Participants (and parent as applicable) will be asked for their consent to participate in the study. A brief medical history will be recorded, including information such as age, gender, height, weight, race, current medications and living environment. If the participant is a PCD patient, they will also be asked about their disease history. Prior to performing the nasal measurements, participants will receive instructions from study personnel and have the opportunity to practice. All participants will have a brief nasal exam and will also have to blow their nose before starting the measurements. Participants will be asked to perform nasal nitric oxide measurements using the tidal breathing method followed by the velum closed with expiration against resistance method. The primary objective is to determine the feasibility and capability of the NIOX VERO to discriminate participants with PCD from those that are healthy. Information collected in this study will help researchers understand more about the diagnosis of and identification of patients with PCD.

Conditions

PCD

Outcome Measures

Primary Outcomes (1)

• The primary endpoint will be the analysis of the means of the successful nNO measurements in Subjects with PCD as compared with Healthy Subjects in the Evaluable Population.

  After a single 1-2 hour visit

Study Arms (2)

PCD Subjects

Participants 5 and older with PCD.

Healthy Subjects

Participants 5 and older without any pulmonary disease.

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This is a multi-center, single visit clinical investigation involving patients with known PCD \[Cohort 1\] vs. age matched healthy volunteers \[Cohort 2\].

You may qualify if:

• Male and female patients 5 years and older.
• Anatomically, is able to complete the nasal NO measurements in both nostrils.
• Cohort 1 - PCD Patients: Patients must have a confirmed diagnosis of PCD from one of the PCD diagnostic centres based on clinical phenotype PLUS diagnosis made by at least 1 of the following (the specifics about how diagnosis was made must be documented in their medical file):
• A nasal biopsy or scraping showing a hallmark PCD defect such as, an outer (+/- inner) dynein arm defect, microtubule defect, or
• A genetic test positive for bi-alleilic mutations in a known PCD-causing gene associated with the diagnosis of PCD (e.g., ARMC4, C21orf59, CCDC39, CCDC40, CCDC65, CCDC164, CCDC103, CCDC114, CCDC151, CCNO, DNAAF1 (LRRC50), DNAAF2 (KTU), DNAAF3, DNAH5, DNAH11, DNAI1, DNAI2, DNAL1, DYX1C1, HEATR2, HYDIN, LRRC6, MCIDAS, NME8 (TXNDC3), ODA/IDA, OFD1, RPGR, RSPH3, RSPH4A, RSPH9, SPAG1, ZMYND10), or
• EU Centres Only: A low nasal NO (determined by a chemiluminescent analyser) plus either:
• at least 2 separate occasions with 'hallmark' changes on high-speed video microscopy, or
• demonstration of mislocalisation of ciliary proteins by immunofluorescence microscopy.
• Cohort 2 - Healthy Patients: Healthy, non-atopic, non-smoking patients (defined as patient with no airway or immune problems, no recent significant injury, no systemic infection, no systemic inflammation, no allergies or asthma).

You may not qualify if:

• Currently smokes or it has been less than 6 months from quitting.
• Has had a nose bleed within the past 2 weeks.
• Has acute respiratory symptoms or signs of an upper or lower respiratory tract infection.
• Use of nasal medication as described below:
• Xolair ≤180 days prior to nNO measurement
• Oral or Systemic Corticosteroids ≤30 days prior to nNO measurement
• Inhaled, nebulized, or intranasal corticosteroids ≤30 days prior to nNO measurement
• Nasal or oral decongestants or antihistamines ≤14 days prior to nNO measurement
• Leukotriene receptor antagonists ≤30 days prior to nNO measurement
• Has an obstruction or anatomy that prevents a nasal measurement from being performed (as confirmed by simple visual inspection by the Investigator).
• Has Cystic Fibrosis.
• Has a documented primary or acquired immunodeficiency.
• Is undergoing treatment with NO-releasing drugs (such as nitrates or molsidomine).
• Has had food or beverage intake (other than water) or has participated in strenuous exercise within 1 hour of nasal NO measurement
• Is unwilling or unable to provide consent to participate (self, parent or legal guardian).

Sponsors & Collaborators

• Aerocrine AB: lead

Study Sites (5)

St Louis

St Louis, Missouri, United States

Location

Chapel Hill

Chapel Hill, North Carolina, United States

Location

Denmark

Copenhagen, Denmark

Location

Paris

Paris, France

Location

Germany

Münster, Germany

Location

Study Officials

• Kathy Rickard, MD

  Aerocrine AB

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 1, 2015
• First Posted: December 4, 2015
• Study Start: May 1, 2016
• Primary Completion: October 1, 2016
• Study Completion: April 28, 2017
• Last Updated: May 15, 2017

Record last verified: 2017-05

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1); FR (1); US (2); DK (1)