Cystic Fibrosis Microbiome-determined Antibiotic Therapy Trial in Exacerbations: Results Stratified (CFMATTERS)
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Not Applicable
Locations
Ireland
Study Type
Interventional
Intervention
Ceftazidime
Tobramycin
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Cystic Fibrosis, Microbiome, Stratified Antibacterial therapy, Resistance, Pathogen Host Interaction
Eligibility Criteria
Inclusion Criteria:
- Written and informed consent, and assent where required.
- Age 16 years or older at enrolment
- Diagnosis of CF by standard sweat test and/or genetic analysis
- Persistent pulmonary Pseudomonas aeruginosa colonization confirmed on at least 2 occasions in the preceding 12 months
- Screening FEV1 predicted of >25%
- Able to perform spirometry reproducibly prior to enrolment
- Able to expectorate and provide a sputum sample at least once daily
- ≥1 non-elective course of intravenous antibiotics in the preceding year
- Able to understand and comply with protocol requirements, restrictions and instructions and likely to complete the study as planned, as judged by the investigator
Exclusion Criteria:
- Life expectancy less than 6 months
- They are a solid organ transplant recipient
- Have a requirement for immunosuppression ≥10mg corticosteroids per day
- Previous positive culture of non-tuberculosis mycobacteria species M.avium, M.abscessus or M.intracellulare within the last 12 months or undergoing active therapy
- Positive culture of any Burkholderia cepacia species within the last 12 months or undergoing active therapy
- Allergic bronchopulmonary aspergillosis on treatment
- Known allergies to more than 3 different classes of antibiotics, and intolerance or allergy to tobramycin.
- Liver portal hypertension, determined by identification of oesophageal varices
- Advanced kidney disease requiring a dose reduction of ceftazidime or contraindicating aminoglycosides
- History of any illness that in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
- If patient undergoes a pulmonary exacerbation before the Microbiome analysis is reviewed by the Consensus Treatment Panel and i.v. antibiotics are administered. In this case, a repeat sputum will be sent for analysis 4 weeks after end of antibiotic treatment.
- Pregnant or breast-feeding at time of eligible pulmonary exacerbation
Sites / Locations
- University College Cork
Arms of the Study
Arm 1
Arm 2
Arm Type
Active Comparator
Experimental
Arm Label
Standard Empiric Treatment
Microbiome Guided Treatment
Arm Description
Ceftazidime or Aztreonam (in case of Ceftazidime allergy) and Tobramycin
Ceftazidime or Aztreonam (in case of Ceftazidime allergy) and Tobramycin and 3rd Antibiotic based on the Microbiome analysis
Outcomes
Primary Outcome Measures
The percentage change in recovery (post-exacerbation) FEV1 relative to the previous pre-exacerbation FEV1.
Secondary Outcome Measures
The time to next pulmonary exacerbation
The improvement in symptom burden by day 7 as determined by Cystic Fibrosis Respiratory Symptom Diary (CFRSD)
As determined by Cystic Fibrosis Respiratory Symptom Diary (CFRSD)
The improvement in health related quality of life at day 28 post treatment and at 3 months as determined by the Cystic Fibrosis Questionnaire Revised (CFQR)
As determined by the Cystic Fibrosis Questionnaire Revised (CFQR)
Total number of i.v. antibiotic days (home or in hospital) from time of randomisation in the trial
Change in FEV1
Total number of exacerbations post trial treatment
Full Information
NCT ID
NCT02526004
First Posted
August 10, 2015
Last Updated
June 1, 2022
Sponsor
University College Cork
Collaborators
Queen's University, Belfast, University of Paris 5 - Rene Descartes, University of Dundee, University of Washington, University Hospital Heidelberg, Teagasc, Clininfo S.A., GABO:mi, Papworth Hospital NHS Foundation Trust, KU Leuven, Assistance Publique - Hôpitaux de Paris, European Union
1. Study Identification
Unique Protocol Identification Number
NCT02526004
Brief Title
Cystic Fibrosis Microbiome-determined Antibiotic Therapy Trial in Exacerbations: Results Stratified
Acronym
CFMATTERS
Official Title
Cystic Fibrosis Microbiome-determined Antibiotic Therapy Trial in Exacerbations: Results Stratified
Study Type
Interventional
2. Study Status
Record Verification Date
June 2022
Overall Recruitment Status
Completed
Study Start Date
October 1, 2013 (Actual)
Primary Completion Date
June 30, 2017 (Actual)
Study Completion Date
June 30, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University College Cork
Collaborators
Queen's University, Belfast, University of Paris 5 - Rene Descartes, University of Dundee, University of Washington, University Hospital Heidelberg, Teagasc, Clininfo S.A., GABO:mi, Papworth Hospital NHS Foundation Trust, KU Leuven, Assistance Publique - Hôpitaux de Paris, European Union
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Antimicrobial resistance is a significant challenge facing global healthcare. The unnecessary use of antibiotics is a key driver in the development of antibiotic resistance. Cystic Fibrosis (CF) represents a unique disease model to study bacterial resistance and to explore therapeutic strategies for same, as chronic lung infection overlaps with acute lung exacerbation's caused by a multitude of organisms. With time, chronic polymicrobial infection develops, with the most dominant infecting organism being Pseudomonas aeruginosa. In acute CF infections, empiric intravenous antibiotics are usually given for two weeks. Recurrent infections and treatments result in increasing antimicrobial resistance, and alterations in pathogen host interactions in the lung and gut flora. Next-generation DNA sequencing technology now offers DNA-based personalised diagnostics and treatment strategies. Enhancing our knowledge of the microbiome allows the use of stratified targeted antibacterial therapy that can be compared with standard empirical antibacterial therapy currently used. Cystic Fibrosis Microbiome-determined Antimicrobial Therapy Trial in Exacerbations: Results Stratified (CFMATTERS) will provide a randomized multi-centre controlled trial of microbiome-derived antimicrobial treatments versus current empirical therapy.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
Cystic Fibrosis, Microbiome, Stratified Antibacterial therapy, Resistance, Pathogen Host Interaction
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Allocation
Randomized
Enrollment
223 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Standard Empiric Treatment
Arm Type
Active Comparator
Arm Description
Ceftazidime or Aztreonam (in case of Ceftazidime allergy) and Tobramycin
Arm Title
Microbiome Guided Treatment
Arm Type
Experimental
Arm Description
Ceftazidime or Aztreonam (in case of Ceftazidime allergy) and Tobramycin and 3rd Antibiotic based on the Microbiome analysis
Intervention Type
Drug
Intervention Name(s)
Ceftazidime
Intervention Type
Drug
Intervention Name(s)
Tobramycin
Primary Outcome Measure Information:
Title
The percentage change in recovery (post-exacerbation) FEV1 relative to the previous pre-exacerbation FEV1.
Time Frame
Time from enrollment into the study up to study close month 21
Secondary Outcome Measure Information:
Title
The time to next pulmonary exacerbation
Time Frame
Time from pulmonary exacerbation day 0, to next pulmonary exacerbation up to study close month 21
Title
The improvement in symptom burden by day 7 as determined by Cystic Fibrosis Respiratory Symptom Diary (CFRSD)
Description
As determined by Cystic Fibrosis Respiratory Symptom Diary (CFRSD)
Time Frame
Time from pulmonary exacerbation day 0 to day 7 of pulmonary exacerbation
Title
The improvement in health related quality of life at day 28 post treatment and at 3 months as determined by the Cystic Fibrosis Questionnaire Revised (CFQR)
Description
As determined by the Cystic Fibrosis Questionnaire Revised (CFQR)
Time Frame
Time from pulmonary exacerbation day 0 to day 28 and month 3 post study treatment
Title
Total number of i.v. antibiotic days (home or in hospital) from time of randomisation in the trial
Time Frame
Time from enrollment in the study up to study close month 21
Title
Change in FEV1
Time Frame
Time from enrollment in the study up to study close month 21
Title
Total number of exacerbations post trial treatment
Time Frame
Time from pulmonary exacerbation day 0 to study close month 21
10. Eligibility
Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Written and informed consent, and assent where required.
Age 16 years or older at enrolment
Diagnosis of CF by standard sweat test and/or genetic analysis
Persistent pulmonary Pseudomonas aeruginosa colonization confirmed on at least 2 occasions in the preceding 12 months
Screening FEV1 predicted of >25%
Able to perform spirometry reproducibly prior to enrolment
Able to expectorate and provide a sputum sample at least once daily
≥1 non-elective course of intravenous antibiotics in the preceding year
Able to understand and comply with protocol requirements, restrictions and instructions and likely to complete the study as planned, as judged by the investigator
Exclusion Criteria:
Life expectancy less than 6 months
They are a solid organ transplant recipient
Have a requirement for immunosuppression ≥10mg corticosteroids per day
Previous positive culture of non-tuberculosis mycobacteria species M.avium, M.abscessus or M.intracellulare within the last 12 months or undergoing active therapy
Positive culture of any Burkholderia cepacia species within the last 12 months or undergoing active therapy
Allergic bronchopulmonary aspergillosis on treatment
Known allergies to more than 3 different classes of antibiotics, and intolerance or allergy to tobramycin.
Liver portal hypertension, determined by identification of oesophageal varices
Advanced kidney disease requiring a dose reduction of ceftazidime or contraindicating aminoglycosides
History of any illness that in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
If patient undergoes a pulmonary exacerbation before the Microbiome analysis is reviewed by the Consensus Treatment Panel and i.v. antibiotics are administered. In this case, a repeat sputum will be sent for analysis 4 weeks after end of antibiotic treatment.
Pregnant or breast-feeding at time of eligible pulmonary exacerbation
Facility Information:
Facility Name
University College Cork
City
Cork
Country
Ireland
12. IPD Sharing Statement
Learn more about this trial
Cystic Fibrosis Microbiome-determined Antibiotic Therapy Trial in Exacerbations: Results Stratified
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