Oral Cysteamine in Adult Patients with Cystic Fibrosis (CF)
Research type
Research Study
Full title
A Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study Investigating the Optimal Dose Regimen, Efficacy, and Safety of Adding Oral Cysteamine in Adult Patients with Cystic Fibrosis (CF) Being Treated for an Exacerbation of CF-associated Lung Disease
IRAS ID
195249
Contact name
Graham Devereux
Contact email
Sponsor organisation
NovaBiotics, Ltd
Eudract number
2015-004986-99
Duration of Study in the UK
0 years, 6 months, 11 days
Research summary
Cystic Fibrosis (CF) is the commonest fatal inherited disease in Caucasian populations of European origin. In CF the lungs are chronically infected with bacteria that cause chronic symptoms and acute periods of worsening infection known as exacerbations.
Cysteamine is a licensed drug used in the treatment of cystinosis for >20 years. Laboratory studies suggest that cysteamine may be a beneficial add-on to conventional antibiotic treatment in CF. We have shown that oral cysteamine is absorbed by adults with CF and that concentrations in the lung are about four times that in the blood. We now wish to find out what is the best dose of cysteamine to use in people with CF when they have an infective exacerbation. In addition we wish to find out the best Patient Reported Outcome Measure (PROM) for use in future studies of CF exacerbations.
Patients with CF experiencing an exacerbation will receive their usual treatments but in addition will be randomized to receive either cysteamine or placebo as add-on therapy.
120 patients with CF will be randomized in a 1:1:1:1:1:1 ratio to the following total daily dose groups of cysteamine or placebo: 450 mg as one dose: 450 mg as 3 doses of 150 mg: 900 mg as 2 doses of 450 mg: 900 mg as 3 doses of 300 mg: 1,350 mg as 3 doses of 450 mg: placebo.
The study is double-blinded and multicentre.
During the treatment period, patients will take study drug daily for 14 days and will be reviewed on Days 7, 14, and 21. The following will be assessed: sputum samples for bacterial load, lung function (FEV1), weight, markers of inflammation (CRP, blood white cell count), and four available PROM questionnaires. Safety will be assessed by patient reported adverse events, laboratory blood and urine tests and physical examinations.REC name
East of Scotland Research Ethics Service REC 2
REC reference
16/ES/0100
Date of REC Opinion
22 Sep 2016
REC opinion
Further Information Favourable Opinion