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

NCT04016571 | Completed

• 1 other identifier: IRAS 210802
• Study Type: observational
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis

Keywords

Cystic Fibrosis; Pulmonary Exacerbation; Antibiotics; Outcome measures

Outcome Measures

Primary Outcomes (1)

• Forced Expiratory Volume in 1 second (FEV1)

  Lung function Measure

  Change in lung function between baseline and day 0, Day 5 and Day 14 of treatment

Secondary Outcomes (10)

• Treatment Failure defined by need for further IV antibiotic treatment within 30 days

  Need for a further course of IV antibiotics within 30 days of treatment completion

• Change in Respiratory Symptoms

  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)

  Change between Day 0, day 5 and day 14 of treatment

• Sleep parameters measured by consumable wearable device

  Change between Day 0, day 5 and day 14 of treatment

• Change in serum inflammatory marker (C-Reactive Protein)

  Change between Day 0, day 5 and day 14 of treatment

Study Arms (1)

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.

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Belfast Health and Social Care Trust: lead
• Queen's University, Belfast: collaborator

Study Sites (1)

Belfast Regional Adult CF Centre

Belfast, BT9 7AE, United Kingdom

Location

Related Publications (13)

• 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.

  PMID: 25911223 BACKGROUND

• 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.

  PMID: 11796433 BACKGROUND

• 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.

  PMID: 21680566 BACKGROUND

• 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.

  PMID: 25754096 BACKGROUND

• 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.

  PMID: 20463179 BACKGROUND

• 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.

  PMID: 19729669 BACKGROUND

• 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.

  PMID: 20925941 BACKGROUND

• 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.

  PMID: 20581166 BACKGROUND

• 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.

  PMID: 25034564 BACKGROUND

• 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.

  PMID: 26769012 BACKGROUND

• 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.

  PMID: 23868694 BACKGROUND

• 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.

  PMID: 25127921 BACKGROUND

• 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.

  PMID: 19481983 BACKGROUND

Biospecimen

Retention: SAMPLES WITHOUT DNA

Expectorated sputum and blood samples will be collected. Samples will be processed within 24 hours or stored at - 80°. Processing will remove any human DNA or HTA relevant material. Samples from participants who have given consent for samples to be for future research will be stored at - 80°C, in a locked freezer in a lab with keypad access. Access to the samples will be limited to researchers involved in the study. Standard University custodial arrangements will apply. For samples from individuals who did not give consent for samples to be stored for the purposes of future research, they will be disposed in accordance with the HTA's code of practice.

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Damian Dr Downey, MBBS MD

  Belfast Health and Social Care Trust

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: January 18, 2019
• First Posted: July 11, 2019
• Study Start: December 1, 2016
• Primary Completion: August 10, 2018
• Study Completion: March 1, 2019
• Last Updated: August 5, 2020

Record last verified: 2020-08

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)