TORPEDO-CF
Trial of Optimal Therapy for Pseudomonas Eradication in Cystic
Fibrosis
Chief Investigator
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Institution
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Dates
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Funding Stream
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Grant Ref
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Amount
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Dr Simon Langton-Hewer
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University Hospitals Bristol NHS Foundation Trust
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01/01/2010 - 31/12/2018
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NIHR Health Technology Assessment
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07/51/01
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£1,300,516.23
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Summary
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). Pseudomonasis 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.
How will it be done?
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 ofP.aeruginosainfection, 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).
The cost of each treatment arm will be estimated by the
collaborating health economics team. Quality of life will be
assessed with a CF-specific and generic measure (CFQ) and Utility
will be assessed using EQ-5D.
Project Timetable
The project will run over a total period of 60 months.The set up
phase of the trial will be six months, with expected recruitment
taking three years and a further 12 months for final follow up plus
six months for analysis and write up.
Links to further information
http://www.torpedo-cf.org.uk/
TORPEDO-CF
PROTOCOL Version: 9.0 21/10/2016
TORPEDO Final Analysis Statistical Analysis Plan v2
