PCDNCT02622061
NIOX VERO Nasal Application in Primary Ciliary Dyskinesia
An observational study of PCD, run by Aerocrine AB at 5 sites in 4 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 years and olderHealthy volunteers can take part
- Conditions the study is for
- PCD
- How many people
- 163 participants
- Where
- 5 sitesin 4 countries
- Study dates
- May 2016to 28 Apr 2017
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
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].
Who may be able to join
- 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:
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- 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).
Who cannot join
- 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
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- 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).
- PCD Patients Only: Has mutations with RSPH1 since nasal NO may not be low in these patients.
- PCD Patients Only: Has not had a standard clinical evaluation to address other potential causes of chronic oto-sino- pulmonary disease.
- Healthy Patients Only: Atopy or the presence of any of the following: a recent significant injury (i.e., within 1-2 weeks), systemic inflammation, airway or immune problem, asthma or allergies.
- In the US only: Patients may not be related to a member of the Study Personnel.
Trial locations and contacts
5 sites in 4 countries.
5 sites in 4 countries
United States2 sites
Chapel Hill
Chapel Hill, North Carolina
St Louis
St Louis, Missouri
About this study
Official title: A Clinical Investigation Determining the Discriminative Ability of the NIOX VERO NASAL to Differentiate Subjects With Primary Ciliary Dyskinesia From Healthy Controls
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
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| PCD Subjects | Participants 5 and older with PCD. | |
| Healthy Subjects | Participants 5 and older without any pulmonary disease. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: After a single 1-2 hour visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2015 (estimated)
- Study startMay 2016
- Primary completionOct 2016
- Study completion28 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PCDStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 163 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aerocrine AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kathy Rickard, MD, Study director, Aerocrine AB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02622061
- Sponsor's protocol ID
- AER-051
- Status last verified
- May 2017
- First submitted
- 1 Dec 2015
- Last updated
- 15 May 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Investigation Determining the Discriminative Ability of the NIOX VERO NASAL to Differentiate Subjects With Primary Ciliary Dyskinesia From Healthy Controls
- ClinicalTrials.gov ID
- NCT02622061
- Acronym / sponsor ID
- AER-051
- Conditions
- PCD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 163 participants (actual)
- Lead sponsor
- Aerocrine ABIndustry (pharma, biotech, devices)
- Study start
- May 2016
- Primary completion
- Oct 2016 (actual)
- Study completion
- 28 Apr 2017 (actual)
- First posted
- 4 Dec 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PCD Subjects | – | –Participants 5 and older with PCD. |
| Healthy Subjects | – | –Participants 5 and older without any pulmonary disease. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- 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].
Key inclusion criteria (9 listed)
- 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):
- 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).
Key exclusion criteria (19 listed)
- 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:
- Has an obstruction or anatomy that prevents a nasal measurement from being performed (as confirmed by simple visual inspection by the Investigator).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
Time frame: After a single 1-2 hour visit
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 May 2017.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Kathy Rickard, MD study director
Aerocrine AB
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of PCD, run by Aerocrine AB at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 May 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States, Denmark, France, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: After a single 1-2 hour visit). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 May 2017; AzTrials last refreshed its copy on 2 Nov 2026.