the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use
A pilot trial of intravenous iron for the treatment of iron deficiency in adult patients with cystic fibrosis
Cystic fibrosis (CF) is an inherited condition affecting over 10,000 patients in the UK, which leads to recurrent chest infections and poor absorption of nutrients from the gut. In the past, patients with CF died in childhood, but most now survive into adulthood. However, they require frequent contact with healthcare services, expensive and time-consuming treatments, and have poor health-related quality of life.
Adults with cystic fibrosis often have low iron levels (iron deficiency). This is partly due to poor absorption of iron from the gut, and partly to the trapping of iron within cells of the immune system during periods of infection. Unfortunately, iron tablets are often ineffective in this setting, and may cause significant side effects in patients with cystic fibrosis.
In other patient groups, intravenous iron is used routinely to correct iron deficiency. In these patients it has been shown to be safe, and to improve energy levels, exercise tolerance, cognitive function and quality of life, even in the absence of anaemia. Recent research suggests that iron may be particularly beneficial in patients with heart and lung disease.
Despite these possible benefits, IV iron is rarely used in patients with CF, due in part to concerns about encouraging the growth of bacterial in the lungs. Iron deficiency therefore often goes untreated. However, since no clinical trials have examined the use of IV iron in CF patients to date, the risks and benefits remain uncertain.
We plan to undertake a small pilot study examining the effects of IV iron in 20 adults with CF and low iron levels. We will primarily assess whether intravenous iron is safe and effective at treating iron deficiency in this group, but will also study various other clinical parameters, to guide the design of future larger studies.
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the name of the treatment or therapy being researched
the number of participants who need to be recruited for the trial in the UK
Last edited date
18 March 2019
University of Oxford
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