The morbidity and mortality associated with Cystic Fibrosis (CF) are the result of chronic
suppurative lung disease. The aggressive use of antibiotics is one of the mainstays of
treatment in CF, however, the problems of multiple drug resistance and adverse reactions are
major clinical issues.
Cysteamine is a licensed drug used in the treatment of cystinosis. In vitro work suggests
that cysteamine has properties of potential benefit in CF. Cysteamine is a potent mucolytic,
it disrupts biofilms, it is antimicrobial, and synergises with other antibiotic agents. CF is
characterised by malabsorption and it is not known whether cysteamine is absorbed in CF,
furthermore it is not known if cysteamine enters the bronchial secretions. It is not possible
to assume that the pharmacokinetics of cysteamine in patients with CF are the same as those
reported for cystinosis.
Objectives: to characterise the pharmacokinetic profile of cysteamine in people with CF, to
ascertain whether cysteamine enters the bronchial secretions and the tolerability of
cysteamine by patients with CF.
Method: a single centre, single group open label investigation of the tolerability and
pharmacokinetics of oral cysteamine (Cystagon) when administered to patients with Cystic
Fibrosis at the dose licensed for use in cystinosis.
Setting: adult CF clinic, Aberdeen Royal Infirmary.
Target population: 12 patients aged ≥18years with CF associated lung disease who are
clinically stable.
Intervention: Oral cysteamine (Cystagon) will be increased from 450mg od to 450mg qds over
three weeks, they will remain on 450mg qds for two weeks.
Assessment: face to face health outcome assessments will be carried out for all participants
at recruitment/baseline, 1, 2, 3, and 5 and 6 weeks. Serial blood cysteamine levels will be
measured in the first 24 hours after the first dose. Sputum cysteamine will be quantified
after two weeks of full dose cysteamine 450mg qds. Disease specific health status (CFQ-R)
will be assessed at baseline and after two weeks of full dose. At each assessment, lung
function (FEV1, FVC), adverse reactions and serious adverse events will be ascertained. Blood
samples will be taken for measurement of haematological and biochemical parameters. Sputum
samples at each assessment will be analysed for microbial load and spinnbarkeit.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
University of Aberdeen
Collaborators:
Cystic Fibrosis Trust NHS Grampian University of Huddersfield