PRospective Observational Multicentre Study on VAriability of Lung Function in Stable PCD Patients (PROVALF-PCD)

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.

Study Overview

• Status: Unknown
• Conditions: Primary Ciliary Dyskinesia
• Intervention / Treatment: Other: Lung function measurement

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 Personal experience and unpublished small retrospective assessments suggest that there is considerable variability.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 Type: Observational
• Enrollment (Actual): 451

Contacts and Locations

Study Locations

• United Kingdom
  • Hampshire
    • Southampton, Hampshire, United Kingdom, SO16 6YD
      • University of Southampton

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Probability Sample

Study Population

Patients diagnosed with PCD under regular follow-up at one of the centres participating in the study.

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Inter-individual variability of FEV1 z-scores	Inter-individual variations between repeated measures of lung function parameters.	up to one-year follow-up period

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): August 23, 2017
• Primary Completion (Anticipated): July 31, 2019
• Study Completion (Anticipated): December 31, 2019

Study Registration Dates

• First Submitted: October 10, 2018
• First Submitted That Met QC Criteria: October 12, 2018
• First Posted (Actual): October 15, 2018

Study Record Updates

• Last Update Posted (Actual): October 15, 2018
• Last Update Submitted That Met QC Criteria: October 12, 2018
• Last Verified: October 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Respiratory Tract Diseases; Neurologic Manifestations; Congenital Abnormalities; Genetic Diseases, Inborn; Otorhinolaryngologic Diseases; Movement Disorders; Abnormalities, Multiple; Ciliopathies; Dyskinesias; Ciliary Motility Disorders
• Other Study ID Numbers: PROVALF

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing Time Frame: Throughout the study, when requested; and at the end of the study, if requested.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ANALYTIC_CODE

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No