Intravenous Iron in Adults With Cystic Fibrosis
- Registration Number
- NCT03632525
- Lead Sponsor
- University of Oxford
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 20
- Age ≥18 years with established diagnosis of cystic fibrosis
- Iron deficiency (transferrin saturation ≤16 % or ferritin <15 μg/l, within last 4 months)
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Intravenous iron Ferric carboxymaltose All participants will receive a single dose of intravenous ferric carboxymaltose
- Primary Outcome Measures
Name Time Method Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron 8 weeks New infective events are defined as any of:
1. New microbiological isolate on routine sputum culture (organism not cultured in 12 months prior to study)
2. Clinical infection requiring IV antibiotics (as determined by clinical team)
3. Admission to hospital for infection-related reason (as determined by clinical team)
4. Significant deterioration in lung function (\>10% fall in FEV1), not otherwise explained (as determined by clinical team)
- Secondary Outcome Measures
Name Time Method Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron 16 weeks (plus 8 weeks of retrospective data collection from notes) 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 16 weeks 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 body mass index 16 weeks Calculated by standard formula: BMI=weight/(height squared)
Change in abundance of sputum Pseudomonas 16 weeks Assessed by quantitative PCR
Change in exercise capacity (shuttle walk test) 16 weeks Standardised and validated exercise test involving exercise at progressive intensity
Change in lung function (FEV1) 16 weeks Assessed by spirometry
Change in arterial oxygen saturation 16 weeks Assessed by non-invasive pulse oximetry
Change in sputum microbiological diversity 16 weeks Assessed by microbiota analysis (16s rRNA gene sequencing)
Change in quality of life (SF-36 questionnaire) 16 weeks 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) 16 weeks Assessed via changes in tricuspid regurgitant jet velocity
Percentage of patient in whom each outcome is successfully measured 16 weeks Calculated based on number of participating patients in whom each outcome is measured.
Change in quality of life (CFQ-R questionnaire) 16 weeks 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.
Percentage of eligible patients entering and completing the study 16 weeks Calculated based on number of eligible patients that enter and/or complete the study.
Trial Locations
- Locations (1)
John Radcliffe Hospital
🇬🇧Oxford, United Kingdom