Parameters to Assess Response to Intra-Venous Antibiotic Treatment for Pulmonary Exacerbations in Cystic Fibrosis (PRIVATE)

August 4, 2020 updated by: Belfast Health and Social Care Trust

Cystic fibrosis (CF) is a multisystem disease characterized by viscid secretions in multiple organ systems. Lung infection and damage account for most of the disease burden. Acute changes in respiratory signs and symptoms termed Pulmonary Exacerbations require treatment with intravenous antibiotics and hospital admission. These episodes cause substantial disruption to people's lives and impact on lung function, quality of life and lifespan. Current treatment regimes require improvement but further study is needed to identify who might benefit from a different approach.

This observational study aims to assess if multi-dimensional measurements taken during treatment correspond with later treatment response. This may allow us to personalise treatment more effectively in the future and to better understand how individuals respond to treatment.

Study Overview

Status

Completed

Conditions

Detailed Description

Pulmonary Exacerbations in CF require treatment with intravenous (IV) antibiotics and hospital admission for between 10-21 days. These episodes cause substantial disruption to people's lives and impact on lung function, quality of life and lifespan.Robust evidence to support current treatment approaches is lacking with awareness that current regimes could be optimised.

As yet there is no model for predicting how patients with CF will respond to IV antibiotic treatment- other than clinical judgement and lung function response.This is due to lack of robust measures to identify clinical response at the time of treatment and safely predict later clinical outcomes. The heterogeneity of the 21st Century CF population means a multi-dimensional composite measure is needed. This study has therefore been designed to provide an overall picture of people's response including clinical, biochemical and patient related outcome measures. Using multi-dimensional assessment we hope the measures assessed in this study will give a better picture of how people feel and how they respond to treatment.

Study Type

Observational

Enrollment (Actual)

31

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
      • Belfast, United Kingdom, BT9 7AE
        • Belfast Regional Adult CF Centre

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Adults with a confirmed diagnosis of Cystic Fibrosis being admitted for treatment with intravenous antibiotics for a pulmonary exacerbation using defined criteria (Physician and patient agreed need for admission for intravenous antibiotic treatment)

Description

Inclusion Criteria:

  1. Patients attending the adult service with a confirmed diagnosis of CF by sweat test or genotype
  2. Able to provide written informed consent
  3. Able to understand and comply with protocol requirements and instructions
  4. Confirmed Pulmonary Exacerbation requiring treatment with intravenous antibiotics using defined criteria (Physician and patient agreed need for admission for intravenous antibiotic treatment)

Exclusion Criteria:

  1. Previous recipient of a lung or other solid organ transplant
  2. Inability to complete questionnaires
  3. Current enrolment in other investigational medicinal product randomised trial
  4. Admission for reasons other than pulmonary exacerbation of CF

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Adults with Cystic Fibrosis

All Adults with a confirmed diagnosis of Cystic Fibrosis being admitted for Intra-Venous Antibiotic Treatment of a Pulmonary Exacerbation

This study is observational so no intervention will be carried out.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Forced Expiratory Volume in 1 second (FEV1)
Time Frame: Change in lung function between baseline and day 0, Day 5 and Day 14 of treatment
Lung function Measure
Change in lung function between baseline and day 0, Day 5 and Day 14 of treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment Failure defined by need for further IV antibiotic treatment within 30 days
Time Frame: Need for a further course of IV antibiotics within 30 days of treatment completion
The number of participants requiring a further course of IV antibiotics within 30 days
Need for a further course of IV antibiotics within 30 days of treatment completion
Change in Respiratory Symptoms
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in Respiratory Symptoms assessed by CF-Respiratory Symptom Diary
Change between Day 0, day 5 and day 14 of treatment
Physical activity levels (step count, distance walked, Lightly active minutes, Moderate velocity physical activity (MVPA) and Exercise)
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in Physical Activity Levels measured by consumable wearable device
Change between Day 0, day 5 and day 14 of treatment
Sleep parameters measured by consumable wearable device
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in time in bed, time asleep and time awake/restless (mins)
Change between Day 0, day 5 and day 14 of treatment
Change in serum inflammatory marker (C-Reactive Protein)
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in serum inflammatory marker (CRP)
Change between Day 0, day 5 and day 14 of treatment
Change in sputum inflammatory markers
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in sputum (Interleukins, Tumour Necrosis Factor-Alpha, Calprotectin and Human Mobility Growth Box Protein -1) inflammatory markers (measured in pg/ml)
Change between Day 0, day 5 and day 14 of treatment
Change in microbiological growth measured by culture
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in microbiological growth measured by culture
Change between Day 0, day 5 and day 14 of treatment
Change in microbiological community composition measured by microbiota analysis
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in microbiological relative abundance, dominance, evenness, diversity and richness
Change between Day 0, day 5 and day 14 of treatment
Change in 24 hour sputum volume
Time Frame: Change between Day 0, day 5 and day 14 of treatment
Change in 24 hour sputum volume (mls)
Change between Day 0, day 5 and day 14 of treatment
Time to next exacerbation
Time Frame: Time to next course of intravenous antibiotics within 12 month follow up period (study end)
Time to next exacerbation
Time to next course of intravenous antibiotics within 12 month follow up period (study end)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Belfast Health and Social Care Trust

Collaborators

Queen's University, Belfast

Investigators

  • Principal Investigator: Damian Dr Downey, MBBS MD, Belfast Health and Social Care Trust

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 1, 2016

Primary Completion (Actual)

August 10, 2018

Study Completion (Actual)

March 1, 2019

Study Registration Dates

First Submitted

January 18, 2019

First Submitted That Met QC Criteria

July 10, 2019

First Posted (Actual)

July 11, 2019

Study Record Updates

Last Update Posted (Actual)

August 5, 2020

Last Update Submitted That Met QC Criteria

August 4, 2020

Last Verified

August 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRAS 210802

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

IPD Plan Description

No individual participant data will be made available to maintain confidentiality

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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