Title: Torpedo-CF: Trial of optimal therapy for pseudomonas eradication in cystic fibrosis
Funder: Department of Health and Social Care

Abstract: Design Multi-centre parallel group, randomised controlled trial comparing ten days of intravenous therapy to three months of oral therapy. Setting Multicentre (Paediatric and Adult centres) UK study coordinated via the Medicines for Children Research Network Clinical Trials Unit Target Population All cystic fibrosis patients who have isolated P.aeruginosa and fulfil the inclusion criteria from participating centres will be considered eligible to take part in the trial. Health Technologies Being Assessed This trial aims to examine whether ten days intravenous ceftazidime with tobramycin is superior to oral ciprofloxacin Both treatment arms will receive three months of nebulised colistin in conjunction to the randomised treatment. Measurement of cost and outcomes The primary outcome measure will be successful eradication of P.aeruginosa infection at three months post randomisation, and remaining infection free through to 15 months post randomisation. Secondary outcomes will include time to recuurence of P.aeruginosa infection, time to new P.aeruginosa infection, lung function, growth and nutritional status, number of pulmonary exacerbations, admission to hospital, number of days spent as inpatient, quality of life, utility, adverse events, reinfection with a different strain of Pseudomonas, other sputum/cough microbiology, candida infection, cost per patient, incremental cost effectiveness ratio, carer burden (as measured by number of days missed from school or work).

Lay abstract: Children and adults with cystic fibrosis are prone to chest infections and these infections can cause long term damage to the lungs, which can affect the health and long term survival of the patient. There are a number of different organisms that cause these infections, one of the most important of which is known as Pseudomonas aeruginosa (there is no lay term for this organism). Pseudomonas is treated with oral (by mouth), intravenous (directly into the blood) and nebulised antibiotics (inhaled as an aerosol), usually in combination. Sometimes doctors choose to treat the patient in the community with three months of oral and at least three months of nebulised antibiotics and sometimes choose to admit the patient to hospital and treat for 10-14 days with intravenous treatment as well as continuing the same nebulised treatment for three months. Despite there being substantial differences to the patient and family in terms of what these two treatment choices offer, at present it is not possible for medical teams to say if intravenous treatment is better than oral treatment in terms of their ability to totally clear the infection from the body. This study addresses this question and will also address the question of cost of each form of treatment. Cost will be measured in terms of direct costs of the treatment including cost of medication and of giving the medication (including the cost of hospital admission where appropriate) and in terms of indirect costs such as time missed from school and work and the relative amount of inconvenience experienced by the patient and family for each form of treatment.

Research activity codes: 6.1 PHARMACEUTICALS
Health categories: CONGENITAL DISORDERS;INFECTION