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First Study of Oral Cysteamine in Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02212431
Recruitment Status : Completed
First Posted : August 8, 2014
Last Update Posted : November 5, 2015
Cystic Fibrosis Trust
NHS Grampian
University of Huddersfield
Information provided by (Responsible Party):
University of Aberdeen

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Cysteamine Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: An Open Label Investigation of the Tolerability and Pharmacokinetics of Oral Cysteamine in Adults With Cystic Fibrosis.
Study Start Date : August 2014
Actual Primary Completion Date : March 2015
Actual Study Completion Date : April 2015

Arm Intervention/treatment
Experimental: Cysteamine

Dosing will be in accordance with licensed use of cysteamine for cystinosis, i.e. 450mg qds.

Dose will be escalated:

450mg od for one week 450mg bd for one week 450mg tds for one week 450mg qds for two weeks

Drug: Cysteamine
Dose will be increased weekly from 450mg od to 450mg bd, to 450mg tds to 450mg qds, will remain on highest dose for 2 weeks
Other Name: Cystagon 150mg capsules

Primary Outcome Measures :
  1. Elimination rate constant [k] [ Time Frame: 24 hours ]
    Blood cysteamine prior to the initial dose on day 1 and 0.5, 1, 1.5, 2, 2.5, 3, 4, 6, 8, 10, and 24 hours post-dose

Secondary Outcome Measures :
  1. Concentration of cysteamine in sputum at week 5 [ Time Frame: 3 hours after final dose ]

Other Outcome Measures:
  1. Change in weight from baseline at week 5 [ Time Frame: 5 weeks ]
  2. Number of Participants with Serious and Non-Serious Adverse Events [ Time Frame: 6 weeks ]
  3. Change in FEV1, FVC from baseline at week 5 [ Time Frame: 5 weeks ]
  4. Change in disease related health status from baseline at week 5 measured by CFQ-R [ Time Frame: 5 weeks ]
  5. Change in haematological and biochemical indices from baseline at week 5 [ Time Frame: 5 weeks ]
  6. change in sputum microbiology from baseline at week 5 [ Time Frame: 5 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • CF related suppurative lung disease who expectorate sputum,
  • Clinically stable for >4 weeks,
  • Aged ≥18 years,
  • Weight >50kg,
  • Female participants of child bearing potential should be using a reliable form of contraception.

Exclusion Criteria:

  • Hypersensitivity to the active substance, any form of cysteamine, or to any of the excipients.
  • Hypersensitivity to penicillamine.
  • Lung, liver transplant, on active transplant list.
  • For women, current pregnancy or breast-feeding, or planned pregnancy during the study.
  • Any other significant disease/disorder which, in the investigator's opinion, either puts the patient at risk because of study participation or may influence the results of the study or the patient's ability to participate in the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02212431

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United Kingdom
Aberdeen Royal Infirmary
Aberdeen, United Kingdom, AB25 2ZN
Sponsors and Collaborators
University of Aberdeen
Cystic Fibrosis Trust
NHS Grampian
University of Huddersfield
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Principal Investigator: Graham Devereux, MD University of Aberdeen

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Responsible Party: University of Aberdeen Identifier: NCT02212431    
Other Study ID Numbers: 3/001/14
2014-000284-40 ( EudraCT Number )
First Posted: August 8, 2014    Key Record Dates
Last Update Posted: November 5, 2015
Last Verified: November 2015
Keywords provided by University of Aberdeen:
Cystic Fibrosis
Additional relevant MeSH terms:
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Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Cystine Depleting Agents
Molecular Mechanisms of Pharmacological Action