PRospective Observational Multicentre Study on VAriability of Lung Function in Stable PCD Patients

Primary Ciliary Dyskinesia Clinical Trial

— PROVALF-PCD  

Official title:

PRospective Observational Multicentre Study on VAriability of Lung Function in Stable PCD Patients

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03704896
• Other study ID #: PROVALF
• Status: Active, not recruiting
• Start date: August 23, 2017
• Est. completion date: December 31, 2019

Study information

• Verified date: October 2018
• Source: University of Southampton
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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 Personal experience and unpublished small retrospective assessments suggest that there is considerable variability.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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 451
• Est. completion date: December 31, 2019
• Est. primary completion date: July 31, 2019
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• 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

Exclusion Criteria:

• Children < 5 years of age

• Regular interval between spirometry testing > 6 months

Related Conditions & MeSH terms

• Ciliary Motility Disorders
• Dyskinesias
• Kartagener Syndrome
• Primary Ciliary Dyskinesia

Intervention

Other:
• Lung function measurement
Spirometry-derived lung function measurements

Locations

Country	Name	City	State
United Kingdom	University of Southampton	Southampton	Hampshire

Sponsors (20)

Lead Sponsor

Collaborator

University of Southampton

Assistance Publique - Hôpitaux de Paris, Azienda Ospedaliero, Universitaria Meyer, Bambino Gesù Hospital and Research Institute, Copenhagen University Hospital, Denmark, Federico II University, Hacettepe University, Hospital Vall d'Hebron, Marmara University, Royal Brompton & Harefield NHS Foundation Trust, Ruhr University of Bochum, Universidade Nova de Lisboa, Universitaire Ziekenhuizen Leuven, University Hospital Muenster, University Hospital, Motol, University of Bern, University of Cyprus, University of Melbourne, University of Sydney, University of Valencia

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	up to one-year follow-up period
Secondary	Inter-individual variability of FEV1 z-scores	Inter-individual variations between repeated measures of lung function parameters.	up to one-year follow-up period