Clinical trial NCT03632525: 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
Overall status: COMPLETED
Phase: PHASE4
Conditions: Cystic Fibrosis
Iron-deficiency
Lead sponsor: University of Oxford
Sponsor class: OTHER
First posted: 2018-08-15
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Incidence of new infective events during 4 weeks before intravenous iron, compared with 4 weeks after intravenous iron
timeFrame: 8 weeks
Secondary outcomes:
measure: Incidence of new infective events during 12 weeks before intravenous iron, compared with 12 weeks after intravenous iron
timeFrame: 16 weeks (plus 8 weeks of retrospective data collection from notes)
measure: Change in number of antibiotic days
timeFrame: 16 weeks
measure: Change in abundance of sputum Pseudomonas
timeFrame: 16 weeks
measure: Change in sputum microbiological diversity
timeFrame: 16 weeks
measure: Change in exercise capacity (shuttle walk test)
timeFrame: 16 weeks
measure: Change in lung function (FEV1)
timeFrame: 16 weeks
measure: Change in arterial oxygen saturation
timeFrame: 16 weeks
measure: Change in body mass index
timeFrame: 16 weeks
measure: Change in quality of life (CFQ-R questionnaire)
timeFrame: 16 weeks
measure: Change in quality of life (SF-36 questionnaire)
timeFrame: 16 weeks
measure: Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)
timeFrame: 16 weeks
measure: Percentage of eligible patients entering and completing the study
timeFrame: 16 weeks
measure: Percentage of patient in whom each outcome is successfully measured
timeFrame: 16 weeks
Other outcomes:
Locations:
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
Study officials:
affiliation: University of Oxford
name: Nick P Talbot, BMBCh DPhil
role: PRINCIPAL_INVESTIGATOR
References: