Back to resultsIntravenous Iron in Adults With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis, Iron-deficiency
Status
Completed
Phase
Phase 4
Locations
United Kingdom
Study Type
Interventional
Intervention
Ferric carboxymaltose
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
Inclusion Criteria:
- Age ≥18 years with established diagnosis of cystic fibrosis
- Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)
Exclusion Criteria:
- Urgent (<6 weeks) need for iron supplementation
- Active infection (currently requiring IV antibiotics)
- Previous intravenous iron supplementation (within last 4 months)
- Current oral iron supplementation
- Hypersensitivity to ferric carboxymaltose
- Active non-tuberculous mycobacterial pulmonary disease (as defined by ATS-IDSA criteria)
- Liver failure
- Ferritin >300 μg/l or transferrin saturation >45%
- Pregnancy or breast feeding
- Previous transplantation
- Judged by member of trial team to be unlikely to comply with safety aspects of trial
Sites / Locations
- John Radcliffe Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Intravenous iron
Arm Description
All participants will receive a single dose of intravenous ferric carboxymaltose
Outcomes
Primary Outcome Measures
Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
New infective events are defined as any of:
New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study)
Clinical infection requiring IV antibiotics (as determined by clinical team)
Admission to hospital for infection-related reason (as determined by clinical team)
Significant deterioration in lung function (>10% fall in FEV1), not otherwise explained (as determined by clinical team)
Secondary Outcome Measures
Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron
Infective events are defined as per primary outcome. Data relating to the 8 weeks prior to the 16-week prospective study period will be obtained from the medical records.
Change in number of antibiotic days
Assessed by review of clinical notes and patient self-reporting, to determine total number of days on which the patient was treated with antibiotics
Change in abundance of sputum Pseudomonas
Assessed by quantitative PCR
Change in sputum microbiological diversity
Assessed by microbiota analysis (16s rRNA gene sequencing)
Change in exercise capacity (shuttle walk test)
Standardised and validated exercise test involving exercise at progressive intensity
Change in lung function (FEV1)
Assessed by spirometry
Change in arterial oxygen saturation
Assessed by non-invasive pulse oximetry
Change in body mass index
Calculated by standard formula: BMI=weight/(height squared)
Change in quality of life (CFQ-R questionnaire)
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a 48-item questionnaire that provides scores in twelve quality of life domains (physical functioning, vitality, emotional state, social limitations, role limitations, embarrassment, body image, eating disturbance, treatment constraints) and three symptom domains (respiratory, digestive, weight). Scores ranging from 0 to 100 are calculated for each quality of life domain, using a published method, where a higher score indicates a more favourable health status.
Change in quality of life (SF-36 questionnaire)
The 36-item short form questionnaire (SF-36) provides scores in eight major domains of health (physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions). Each is scored on a scale from 0-100, where a higher value represents a more favourable health status. The domains may be combined to provide two summary scores, namely the 'physical component summary' and the 'mental component summary', each of which is also scored from 0-100. In calculating the respective summary scores, subscales related to physical or psychological health (as appropriate) are positively weighted, according to a published method.
Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)
Assessed via changes in tricuspid regurgitant jet velocity
Percentage of eligible patients entering and completing the study
Calculated based on number of eligible patients that enter and/or complete the study.
Percentage of patient in whom each outcome is successfully measured
Calculated based on number of participating patients in whom each outcome is measured.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03632525
Brief Title
Intravenous Iron in Adults With Cystic Fibrosis
Official Title
A Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic Fibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Completed
Study Start Date
February 22, 2019 (Actual)
Primary Completion Date
March 27, 2020 (Actual)
Study Completion Date
October 26, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Oxford
4. Oversight
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
Data Monitoring Committee
No
5. Study Description
Brief Summary
This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency.
Detailed Description
Iron deficiency is common in adults with cystic fibrosis, and is associated with adverse outcomes. Oral iron supplementation is poorly tolerated and may be ineffective. In some centres, intravenous iron is used to correct iron deficiency, but concerns have been raised about the safety of this treatment in the setting of chronic airways infection. The investigators are therefore planning a pilot interventional cohort study examining the effects of intravenous iron in a group of adults with cystic fibrosis. Patients will be recruited in Oxford and studied prospectively over 16 weeks, with iron given at week 4. The primary focus of this single-centre pilot/feasibility study is safety, specifically in relation to infection.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis, Iron-deficiency
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Intravenous iron
Arm Type
Experimental
Arm Description
All participants will receive a single dose of intravenous ferric carboxymaltose
Intervention Type
Drug
Intervention Name(s)
Ferric carboxymaltose
Other Intervention Name(s)
Ferrinject
Intervention Description
Single dose of 20 mg/kg ferric carboxymaltose (maximum 1000 mg for patients with haemoglobin <14 g/dL or 500 mg for patients with haemoglobin ≥14 g/dL).
Primary Outcome Measure Information:
Title
Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
Description
New infective events are defined as any of:
New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study)
Clinical infection requiring IV antibiotics (as determined by clinical team)
Admission to hospital for infection-related reason (as determined by clinical team)
Significant deterioration in lung function (>10% fall in FEV1), not otherwise explained (as determined by clinical team)
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron
Description
Infective events are defined as per primary outcome. Data relating to the 8 weeks prior to the 16-week prospective study period will be obtained from the medical records.
Time Frame
16 weeks (plus 8 weeks of retrospective data collection from notes)
Title
Change in number of antibiotic days
Description
Assessed by review of clinical notes and patient self-reporting, to determine total number of days on which the patient was treated with antibiotics
Time Frame
16 weeks
Title
Change in abundance of sputum Pseudomonas
Description
Assessed by quantitative PCR
Time Frame
16 weeks
Title
Change in sputum microbiological diversity
Description
Assessed by microbiota analysis (16s rRNA gene sequencing)
Time Frame
16 weeks
Title
Change in exercise capacity (shuttle walk test)
Description
Standardised and validated exercise test involving exercise at progressive intensity
Time Frame
16 weeks
Title
Change in lung function (FEV1)
Description
Assessed by spirometry
Time Frame
16 weeks
Title
Change in arterial oxygen saturation
Description
Assessed by non-invasive pulse oximetry
Time Frame
16 weeks
Title
Change in body mass index
Description
Calculated by standard formula: BMI=weight/(height squared)
Time Frame
16 weeks
Title
Change in quality of life (CFQ-R questionnaire)
Description
The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a 48-item questionnaire that provides scores in twelve quality of life domains (physical functioning, vitality, emotional state, social limitations, role limitations, embarrassment, body image, eating disturbance, treatment constraints) and three symptom domains (respiratory, digestive, weight). Scores ranging from 0 to 100 are calculated for each quality of life domain, using a published method, where a higher score indicates a more favourable health status.
Time Frame
16 weeks
Title
Change in quality of life (SF-36 questionnaire)
Description
The 36-item short form questionnaire (SF-36) provides scores in eight major domains of health (physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions). Each is scored on a scale from 0-100, where a higher value represents a more favourable health status. The domains may be combined to provide two summary scores, namely the 'physical component summary' and the 'mental component summary', each of which is also scored from 0-100. In calculating the respective summary scores, subscales related to physical or psychological health (as appropriate) are positively weighted, according to a published method.
Time Frame
16 weeks
Title
Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)
Description
Assessed via changes in tricuspid regurgitant jet velocity
Time Frame
16 weeks
Title
Percentage of eligible patients entering and completing the study
Description
Calculated based on number of eligible patients that enter and/or complete the study.
Time Frame
16 weeks
Title
Percentage of patient in whom each outcome is successfully measured
Description
Calculated based on number of participating patients in whom each outcome is measured.
Time Frame
16 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Age ≥18 years with established diagnosis of cystic fibrosis
Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)
Exclusion Criteria:
Urgent (<6 weeks) need for iron supplementation
Active infection (currently requiring IV antibiotics)
Previous intravenous iron supplementation (within last 4 months)
Current oral iron supplementation
Hypersensitivity to ferric carboxymaltose
Active non-tuberculous mycobacterial pulmonary disease (as defined by ATS-IDSA criteria)
Liver failure
Ferritin >300 μg/l or transferrin saturation >45%
Pregnancy or breast feeding
Previous transplantation
Judged by member of trial team to be unlikely to comply with safety aspects of trial
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nick P Talbot, BMBCh DPhil
Organizational Affiliation
University of Oxford
Official's Role
Principal Investigator
Facility Information:
Facility Name
John Radcliffe Hospital
City
Oxford
Country
United Kingdom
12. IPD Sharing Statement
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Intravenous Iron in Adults With Cystic Fibrosis
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