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

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ClinicalTrials.gov Identifier: NCT04016571
Recruitment Status : Recruiting
First Posted : July 11, 2019
Last Update Posted : July 11, 2019
Sponsor:
Collaborator:
Queen's University, Belfast
Information provided by (Responsible Party):
Belfast Health and Social Care Trust

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.


Condition or disease
Cystic Fibrosis

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.


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Study Type : Observational
Estimated Enrollment : 30 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Parameters to Assess Response to Intra-Venous Antibiotic Treatment for Pulmonary Exacerbations in Cystic Fibrosis
Actual Study Start Date : December 1, 2016
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2019

Resource links provided by the National Library of Medicine


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.




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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

  2. 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

  3. Physical activity levels (step count, distance walked, Lightly active minutes, 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

  4. 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)

  5. Change in sputum and serum 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) serum inflammatory markers

  6. 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

  7. 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

  8. 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)

  9. 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


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.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04016571


Contacts
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Contact: Charlotte Dr Addy, BMBS BMedSci 07803009184 c.addy@qub.ac.uk
Contact: Damian Dr Downey, MBBS MD 02890263683 damian.downey@belfasttrust.hscni.net

Locations
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United Kingdom
Belfast Regional Adult CF Centre Recruiting
Belfast, United Kingdom, BT9 7AE
Contact: Damian DR Downey, MBBS    02895048136 ext 8136    Damian.Downey@belfasttrust.hscni.org   
Contact: Charlotte DR Addy, MBBS    07803009184    c.addy@qub.ac.uk   
Sponsors and Collaborators
Belfast Health and Social Care Trust
Queen's University, Belfast
Investigators
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Principal Investigator: Damian Dr Downey, MBBS MD Belfast Health and Social Care Trust

Publications:

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Responsible Party: Belfast Health and Social Care Trust
ClinicalTrials.gov Identifier: NCT04016571     History of Changes
Other Study ID Numbers: IRAS 210802
First Posted: July 11, 2019    Key Record Dates
Last Update Posted: July 11, 2019
Last Verified: July 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: No individual participant data will be made available to maintain confidentiality

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Belfast Health and Social Care Trust:
Cystic Fibrosis
Pulmonary Exacerbation
Antibiotics
Outcome measures
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Anti-Bacterial Agents
Antibiotics, Antitubercular
Anti-Infective Agents
Antitubercular Agents