Orally inhaled heparin in patients with cystic fibrosis (CF)
- Conditions
- Cystic fibrosisNutritional, Metabolic, Endocrine
- Registration Number
- ISRCTN78613729
- Lead Sponsor
- Vectura Limited (UK)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 64
Amendment as of 20/04/2010:
Point one below has been amended as follows:
1. Male or female, aged 16 years or older
Current information as of 21/09/2009:
1. Male or female, aged 18 years or older
2. Non-smoker
3. Written informed consent obtained prior to any trial specific procedures
4. Confirmed diagnosis of CF lung disease (i.e., respiratory clinical symptoms and positive sweat test or disease inducing mutations) by CF expert/investigator
5. Forced expiratory volume in one second (FEV1) at 40 - 90% of predicted value for age, sex and height at screening and baseline
6. FEV1 value at Baseline is within +/-15% of value at screening
7. Regular mucus production due to CF
8. Ease of sputum expectoration as defined by VAS score of = 80 mm
9. Inflammatory markers above upper limit of normal range.
10. Adequate contraceptive measures.
11. Able to comply with all protocol requirements
12. Able to use inhalation device.
Amended as of 17/04/2009:
Please note that point 5 of the below criteria has been amended to read:
5. Forced expiratory volume in one second (FEV1) at 40 - 90% of predicted value for age, sex and height at screening and baseline
Initial information at time of registration:
1. Male or female, aged 18 years or older
2. Non-smoker
3. Written informed consent obtained prior to any trial specific procedures
4. Confirmed diagnosis of CF lung disease (i.e., respiratory clinical symptoms and positive sweat test or disease inducing mutations) by CF expert/investigator
5. Forced expiratory volume in one second (FEV1) at 40 - 80% of predicted value for age, sex and height during six months prior to screening
6. FEV1 within 10% of best value during six months prior to screening
7. Regular mucus production due to CF
Initial information at time of registration:
To be eligible for inclusion into this trial, each patient must not violate any one of the following exclusion criteria at the time of screening, at the time of assessment or as specifically described below:
1. Any contraindication to Monoparin® considered clinically relevant
2. Increased bleeding risk
3. History of heparin-induced thrombocytopaenia
4. Patients with bleeding diathesis
5. Evidence of portal hypertension (e.g., hypersplenism or known grade III/IV oesophageal varices)
6. Clinically significant liver disease
7. Pregnancy at screening, or lactation
8. Previous thoracic or scheduled major surgery during trial
9. Any regular anticoagulant therapy (e.g., warfarin, aspirin) in the two weeks prior to screening
10. Modification of medication to treat respiratory disease between screening and baseline (Day 1)
Added 17/04/2009:
11. Diagnosis or history of aspergilloma
Added 21/09/2009:
12. Clinically significant serious disease or organ system disease not currently controlled / stable on present therapy
13. Planned hospitalisations which could interfere with trial compliance
14. Unable for any other reason to satisfactorily comply with the protocol (e.g., attendance for trial visits, treatment or assessments)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Safety and tolerability. <br><br>Timepoints:<br>Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.
- Secondary Outcome Measures
Name Time Method Assessment of:<br>1. Sputum properties (i.e., rheological viscoelasticity/physicochemical measurement parameters)<br>2. Sputum inflammatory markers<br>3. Exhaled breath condensate pH<br>4. Blood plasma inflammatory markers<br>5. Blood coagulation <br>6. Visual Analogue Scale (VAS) parameters <br>7. Sputum microbiology <br>8. Pulmonary function parameters including FEV1 and forced vital capacity (FVC)<br>9. Response to the Cystic Fibrosis Questionnaire<br><br>Timepoints:<br>Five visits to the trial centre are included: screening, baseline, week 2, week 4 and, for follow-up, week 6.