Intravenous Iron in Adults With Cystic Fibrosis

Cystic Fibrosis Clinical Trial

Official title:

A Pilot Trial of Intravenous Iron for the Treatment of Iron Deficiency in Adult Patients With Cystic Fibrosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03632525
• Other study ID #: PID12800
• Status: Completed
• Phase: Phase 4
• Start date: February 22, 2019
• Est. completion date: October 26, 2021

Study information

• Verified date: September 2022
• Source: University of Oxford
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This pilot interventional cohort study will examine the effects of intravenous iron in adults with cystic fibrosis and iron deficiency.

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 information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: October 26, 2021
• Est. primary completion date: March 27, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia, Iron-Deficiency
• Cystic Fibrosis
• Fibrosis
• Iron-deficiency

Intervention

Drug:
• Ferric carboxymaltose
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).

Locations

Country	Name	City	State
United Kingdom	John Radcliffe Hospital	Oxford

Sponsors (1)

Lead Sponsor	Collaborator
University of Oxford

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)	8 weeks
Secondary	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.	16 weeks (plus 8 weeks of retrospective data collection from notes)
Secondary	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	16 weeks
Secondary	Change in abundance of sputum Pseudomonas	Assessed by quantitative PCR	16 weeks
Secondary	Change in sputum microbiological diversity	Assessed by microbiota analysis (16s rRNA gene sequencing)	16 weeks
Secondary	Change in exercise capacity (shuttle walk test)	Standardised and validated exercise test involving exercise at progressive intensity	16 weeks
Secondary	Change in lung function (FEV1)	Assessed by spirometry	16 weeks
Secondary	Change in arterial oxygen saturation	Assessed by non-invasive pulse oximetry	16 weeks
Secondary	Change in body mass index	Calculated by standard formula: BMI=weight/(height squared)	16 weeks
Secondary	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.	16 weeks
Secondary	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.	16 weeks
Secondary	Change in pulmonary artery pressure, assessed by echocardiography (exploratory outcome)	Assessed via changes in tricuspid regurgitant jet velocity	16 weeks
Secondary	Percentage of eligible patients entering and completing the study	Calculated based on number of eligible patients that enter and/or complete the study.	16 weeks
Secondary	Percentage of patient in whom each outcome is successfully measured	Calculated based on number of participating patients in whom each outcome is measured.	16 weeks