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

NCT ID: NCT04016571

Last Updated: 2020-08-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

31 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-12-01

Study Completion Date

2019-03-01

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Cystic Fibrosis

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

No interventions assigned to this group

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Queen's University, Belfast

OTHER

Sponsor Role collaborator

Belfast Health and Social Care Trust

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Damian Dr Downey, MBBS MD

Role: PRINCIPAL_INVESTIGATOR

Belfast Health and Social Care Trust

Locations

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Belfast Regional Adult CF Centre

Belfast, , United Kingdom

Site Status

Countries

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United Kingdom

References

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Heltshe SL, Goss CH, Thompson V, Sagel SD, Sanders DB, Marshall BC, Flume PA. Short-term and long-term response to pulmonary exacerbation treatment in cystic fibrosis. Thorax. 2016 Mar;71(3):223-9. doi: 10.1136/thoraxjnl-2014-206750. Epub 2015 Apr 24.

Reference Type BACKGROUND
PMID: 25911223 (View on PubMed)

Britto MT, Kotagal UR, Hornung RW, Atherton HD, Tsevat J, Wilmott RW. Impact of recent pulmonary exacerbations on quality of life in patients with cystic fibrosis. Chest. 2002 Jan;121(1):64-72. doi: 10.1378/chest.121.1.64.

Reference Type BACKGROUND
PMID: 11796433 (View on PubMed)

de Boer K, Vandemheen KL, Tullis E, Doucette S, Fergusson D, Freitag A, Paterson N, Jackson M, Lougheed MD, Kumar V, Aaron SD. Exacerbation frequency and clinical outcomes in adult patients with cystic fibrosis. Thorax. 2011 Aug;66(8):680-5. doi: 10.1136/thx.2011.161117. Epub 2011 Jun 15.

Reference Type BACKGROUND
PMID: 21680566 (View on PubMed)

VanDevanter DR, Pasta DJ, Konstan MW. Treatment and demographic factors affecting time to next pulmonary exacerbation in cystic fibrosis. J Cyst Fibros. 2015 Nov;14(6):763-9. doi: 10.1016/j.jcf.2015.02.007. Epub 2015 Mar 6.

Reference Type BACKGROUND
PMID: 25754096 (View on PubMed)

Sanders DB, Bittner RC, Rosenfeld M, Hoffman LR, Redding GJ, Goss CH. Failure to recover to baseline pulmonary function after cystic fibrosis pulmonary exacerbation. Am J Respir Crit Care Med. 2010 Sep 1;182(5):627-32. doi: 10.1164/rccm.200909-1421OC. Epub 2010 May 12.

Reference Type BACKGROUND
PMID: 20463179 (View on PubMed)

Flume PA, Mogayzel PJ Jr, Robinson KA, Goss CH, Rosenblatt RL, Kuhn RJ, Marshall BC; Clinical Practice Guidelines for Pulmonary Therapies Committee. Cystic fibrosis pulmonary guidelines: treatment of pulmonary exacerbations. Am J Respir Crit Care Med. 2009 Nov 1;180(9):802-8. doi: 10.1164/rccm.200812-1845PP. Epub 2009 Sep 3.

Reference Type BACKGROUND
PMID: 19729669 (View on PubMed)

VanDevanter DR, O'Riordan MA, Blumer JL, Konstan MW. Assessing time to pulmonary function benefit following antibiotic treatment of acute cystic fibrosis exacerbations. Respir Res. 2010 Oct 6;11(1):137. doi: 10.1186/1465-9921-11-137.

Reference Type BACKGROUND
PMID: 20925941 (View on PubMed)

Collaco JM, Green DM, Cutting GR, Naughton KM, Mogayzel PJ Jr. Location and duration of treatment of cystic fibrosis respiratory exacerbations do not affect outcomes. Am J Respir Crit Care Med. 2010 Nov 1;182(9):1137-43. doi: 10.1164/rccm.201001-0057OC. Epub 2010 Jun 25.

Reference Type BACKGROUND
PMID: 20581166 (View on PubMed)

Smith DJ, Badrick AC, Zakrzewski M, Krause L, Bell SC, Anderson GJ, Reid DW. Pyrosequencing reveals transient cystic fibrosis lung microbiome changes with intravenous antibiotics. Eur Respir J. 2014 Oct;44(4):922-30. doi: 10.1183/09031936.00203013. Epub 2014 Jul 17.

Reference Type BACKGROUND
PMID: 25034564 (View on PubMed)

Heltshe SL, Goss CH. Optimising treatment of CF pulmonary exacerbation: a tough nut to crack. Thorax. 2016 Feb;71(2):101-2. doi: 10.1136/thoraxjnl-2015-208057. No abstract available.

Reference Type BACKGROUND
PMID: 26769012 (View on PubMed)

Shoki AH, Mayer-Hamblett N, Wilcox PG, Sin DD, Quon BS. Systematic review of blood biomarkers in cystic fibrosis pulmonary exacerbations. Chest. 2013 Nov;144(5):1659-1670. doi: 10.1378/chest.13-0693.

Reference Type BACKGROUND
PMID: 23868694 (View on PubMed)

McCourt F, O'Neill B, Logan I, Abbott J, Plant B, McCrum-Gardner E, McKeown S, Stuart Elborn J, Bradley JM. Indicators of pulmonary exacerbation in cystic fibrosis: A Delphi survey of patients and health professionals. J Cyst Fibros. 2015 Jan;14(1):90-6. doi: 10.1016/j.jcf.2014.06.007. Epub 2014 Aug 12.

Reference Type BACKGROUND
PMID: 25127921 (View on PubMed)

Goss CH, Edwards TC, Ramsey BW, Aitken ML, Patrick DL. Patient-reported respiratory symptoms in cystic fibrosis. J Cyst Fibros. 2009 Jul;8(4):245-52. doi: 10.1016/j.jcf.2009.04.003. Epub 2009 May 29.

Reference Type BACKGROUND
PMID: 19481983 (View on PubMed)

Other Identifiers

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IRAS 210802

Identifier Type: -

Identifier Source: org_study_id

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