Primary Ciliary DyskinesiaPROVALF-PCDNCT03704896
PRospective Observational Multicentre Study on VAriability of Lung Function in Stable PCD Patients
An observational study of Primary Ciliary Dyskinesia, run by University of Southampton at 1 site in United Kingdom.
Trial summary
Using routinely collected clinical data, this study aims to quantify intra-individual (i.e. in the same individual) variations between measurements of lung function in stable patients with primary ciliary dyskinesia (PCD), a rare genetic disease that causes lung damage. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Ciliary Dyskinesia
- What is being tested?
- Lung function measurement
- How many people
- 451 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 23 Aug 2017to 31 Dec 2019
- 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 Any age
- Child, Adult, Older adult
Study population: Patients diagnosed with PCD under regular follow-up at one of the centres participating in the study.
Who may be able to join
- Children (>5 years of age) and adults being follow-up for PCD
- Availability of at least minimal dataset (spirometry data), at least every 6 months
- Outpatients and/or in-patients
Who cannot join
- Children < 5 years of age
- Regular interval between spirometry testing > 6 months
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
University of Southampton
Southampton, Hampshire, SO16 6YD
About this study
Using routinely collected clinical data, this study aims to quantify intra-individual (i.e. in the same individual) variations between measurements of lung function in stable patients with primary ciliary dyskinesia (PCD), a rare genetic disease that causes lung damage.
Read the detailed description
Background
Lung function measurements are commonly used in PCD to monitor disease progression. Spirometry measurements are taken every 3 months and results are compared to established references, adjusted for age, height and ethnicity. Results are also compared to previous measurements from the same patient at earlier appointments. However, little is understood of the impact of intra-individual variability and the extent of spontaneous variations in these comparisons.
One of the priorities for respiratory research in the UK is to understand factors involved in determining different outcomes for lung function.[1] The precision of measurements done on the same individual conducted by different people, in different settings and using different equipment is not entirely known. Importantly, previous studies in healthy children assessing intra-individual variability have shown variations of up to 1.2 z-scores in spirometry parameters over the course of 1 year.[2] Within test-variability and daily repeatability can range from 2 to10% FEV% predicted in young healthy children.[3],[4]
In PCD, deterioration of lung function does not follow a pre-defined pattern.[5] However, none of the published studies on lung function in PCD to date have taken into consideration the imprecision of individual and repeated measurements on the same individual over time. Personal experience and unpublished small retrospective assessments suggest that there is considerable variability.
Key research question
Quantify intra-individual (i.e. in the same individual) variations between measurements of lung function in stable patients with primary ciliary dyskinesia (PCD), a rare genetic disease that causes lung damage.
Study design
Prospective multicentre cohort study using routinely collected clinical data to evaluate natural variability of lung function measurements in stable PCD patients.
The primary end-point is to assess intra-individual variations between repeated measures of lung function parameters. Secondary end-points include: a) Inter-individual variations between repeated measures of lung function parameters and correlations with baseline measures; b) intra- and inter-individual variation between repeated measures of lung function parameters during exacerbation.
Participants will be approached by their clinicians and asked to sign a consent form to allow for their anonymised routinely collected clinical data to be entered into the study. Routine clinical data will be collected at PCD follow-up clinics in participating centres. These data are already collected for clinical purposes and will be anonymised locally. Non-identifiable data will be entered into the study database by a member of the clinical team of the participating centre. The study coordinating centre (University of Southampton) will only have access to the anonymised dataset.
The data collection period will last 18 months (6 months for patients recruitment and 12 months for patient follow-up).
- Conditions
- Primary Ciliary Dyskinesia
- Being tested
- Lung function measurement
- Medical subject headings
- Ciliary Motility Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lung function measurementOther
Spirometry-derived lung function measurements
What is being measured
The primary outcome is the main question the study is designed to answer.
Intra-individual variability of FEV1 z-scores
Natural intra-individual variability of FEV1 z-score in patients that are not experiencing an episode of chest exacerbation at the time of lung function measurement.
Measured: up to one-year follow-up period
Secondary outcomes (1)
Inter-individual variability of FEV1 z-scores
Inter-individual variations between repeated measures of lung function parameters.
Measured: up to one-year follow-up period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Oct 2018
- Study start23 Aug 2017
- Primary completion31 Jul 2019 (estimated)
- Study completion31 Dec 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Ciliary DyskinesiaStudies 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
- 451 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Southampton
- Sponsor type
- University, hospital or other
- Collaborators
- University of Bern, Assistance Publique - Hôpitaux de Paris, University of Melbourne, Universitaire Ziekenhuizen KU Leuven, University Hospital, Motol, Copenhagen University Hospital, Denmark, University Hospital Muenster, Hacettepe University, University of Cyprus, University of Sydney, Ruhr University of Bochum, Marmara University, Royal Brompton & Harefield NHS Foundation Trust, Meyer Children's Hospital IRCCS, Federico II University, Bambino Gesù Hospital and Research Institute, University of Valencia, Universidade Nova de Lisboa, Hospital Vall d'Hebron
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03704896
- Sponsor's protocol ID
- PROVALF
- Acronym
- PROVALF-PCD
- Status last verified
- Oct 2018
- First submitted
- 10 Oct 2018
- Last updated
- 15 Oct 2018
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Anonymised centre-specific data will be shared between PROVALF-PCD study and prospective PCD registry and retrospective iPCD cohort, where specifcially requested by centre contributing with data. All parties involved in data transfer will sign an agreement with PROVALF-PCD prior to any data transfer.
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PRospective Observational Multicentre Study on VAriability of Lung Function in Stable PCD Patients
- ClinicalTrials.gov ID
- NCT03704896
- Acronym / sponsor ID
- PROVALF-PCD / PROVALF
- Conditions
- Primary Ciliary Dyskinesia
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 451 participants (actual)
- Lead sponsor
- University of SouthamptonUniversity, hospital or other
- Collaborators
- University of Bern, Assistance Publique - Hôpitaux de Paris, University of Melbourne, Universitaire Ziekenhuizen KU Leuven, University Hospital, Motol, Copenhagen University Hospital, Denmark, University Hospital Muenster, Hacettepe University, University of Cyprus, University of Sydney, Ruhr University of Bochum, Marmara University, Royal Brompton & Harefield NHS Foundation Trust, Meyer Children's Hospital IRCCS, Federico II University, Bambino Gesù Hospital and Research Institute, University of Valencia, Universidade Nova de Lisboa, Hospital Vall d'Hebron
- Study start
- 23 Aug 2017 (actual)
- Primary completion
- 31 Jul 2019 (estimated)
- Study completion
- 31 Dec 2019 (estimated)
- First posted
- 15 Oct 2018 (actual)
- 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.
Using routinely collected clinical data, this study aims to quantify intra-individual (i.e. in the same individual) variations between measurements of lung function in stable patients with primary ciliary dyskinesia (PCD), a rare genetic disease that causes lung damage.
Detailed description
Background
Lung function measurements are commonly used in PCD to monitor disease progression. Spirometry measurements are taken every 3 months and results are compared to established references, adjusted for age, height and ethnicity. Results are also compared to previous measurements from the same patient at earlier appointments. However, little is understood of the impact of intra-individual variability and the extent of spontaneous variations in these comparisons.
One of the priorities for respiratory research in the UK is to understand factors involved in determining different outcomes for lung function.[1] The precision of measurements done on the same individual conducted by different people, in different settings and using different equipment is not entirely known. Importantly, previous studies in healthy children assessing intra-individual variability have shown variations of up to 1.2 z-scores in spirometry parameters over the course of 1 year.[2] Within test-variability and daily repeatability can range from 2 to10% FEV% predicted in young healthy children.[3],[4]
In PCD, deterioration of lung function does not follow a pre-defined pattern.[5] However, none of the published studies on lung function in PCD to date have taken into consideration the imprecision of individual and repeated measurements on the same individual over time. Personal experience and unpublished small retrospective assessments suggest that there is considerable variability.
Key research question
Quantify intra-individual (i.e. in the same individual) variations between measurements of lung function in stable patients with primary ciliary dyskinesia (PCD), a rare genetic disease that causes lung damage.
Study design
Prospective multicentre cohort study using routinely collected clinical data to evaluate natural variability of lung function measurements in stable PCD patients.
The primary end-point is to assess intra-individual variations between repeated measures of lung function parameters. Secondary end-points include: a) Inter-individual variations between repeated measures of lung function parameters and correlations with baseline measures; b) intra- and inter-individual variation between repeated measures of lung function parameters during exacerbation.
Participants will be approached by their clinicians and asked to sign a consent form to allow for their anonymised routinely collected clinical data to be entered into the study. Routine clinical data will be collected at PCD follow-up clinics in participating centres. These data are already collected for clinical purposes and will be anonymised locally. Non-identifiable data will be entered into the study database by a member of the clinical team of the participating centre. The study coordinating centre (University of Southampton) will only have access to the anonymised dataset.
The data collection period will last 18 months (6 months for patients recruitment and 12 months for patient follow-up).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Age groups
- Child, Adult, Older adult
- Study population
- Patients diagnosed with PCD under regular follow-up at one of the centres participating in the study.
Key inclusion criteria (3 listed)
- Children (>5 years of age) and adults being follow-up for PCD
- Availability of at least minimal dataset (spirometry data), at least every 6 months
- Outpatients and/or in-patients
Key exclusion criteria (2 listed)
- Children < 5 years of age
- Regular interval between spirometry testing > 6 months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Lung function measurementSpirometry-derived lung function measurements | Other | – |
Primary outcome
Intra-individual variability of FEV1 z-scores
Time frame: up to one-year follow-up period
Natural intra-individual variability of FEV1 z-score in patients that are not experiencing an episode of chest exacerbation at the time of lung function measurement.
1 secondary outcome
Inter-individual variability of FEV1 z-scores
Time frame: up to one-year follow-up period
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2018.
- Individual participant dataSharing planned: Yes. Anonymised centre-specific data will be shared between PROVALF-PCD study and prospective PCD registry and retrospective iPCD cohort, where specifcially requested by centre contributing with data. All parties involved in data transfer will sign an agreement with PROVALF-PCD prior to any data transfer.
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Primary Ciliary Dyskinesia, run by University of Southampton at 1 site in United Kingdom.
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 3 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 15 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. 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?
Results are not available in the source record.
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?
Intra-individual variability of FEV1 z-scores (time frame: up to one-year follow-up period). 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 Oct 2018.