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

NCT03704896 | Unknown

• 1 other identifier: PROVALF
• Study Type: observational
• Target: 451
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Primary Ciliary Dyskinesia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Inter-individual variability of FEV1 z-scores

  up to one-year follow-up period

Interventions

Lung function measurement OTHER

Spirometry-derived lung function measurements

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

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

You may qualify if:

• 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

You may not qualify if:

• Children \< 5 years of age
• Regular interval between spirometry testing \> 6 months

Sponsors & Collaborators

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

Study Sites (1)

University of Southampton

Southampton, Hampshire, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Ciliary Motility Disorders

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Study Design

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

Study Record Dates

• First Submitted: October 10, 2018
• First Posted: October 15, 2018
• Study Start: August 23, 2017
• Primary Completion: July 31, 2019
• Study Completion: December 31, 2019
• Last Updated: October 15, 2018

Record last verified: 2018-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ANALYTIC CODE
• Time Frame: Throughout the study, when requested; and at the end of the study, if requested.

Locations

GB (1)