Bronchiectasis MedDRA version: 9.1 Level: LLT Classification code 10006445 Term: Bronchiectasis
Conditions
Interventions
Product Name: Inhaled mannitol
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: mannitol
CAS Number: 69658
Concentration unit: mg milligram(s)
Concentration type: equal
Concen
Sponsors
Pharmaxis Pharmaceuticals Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The subject must: 1. Have given written informed consent to participate in this study in accordance with local regulations 2. Have documented evidence of confirmed diagnosis of (non-CF) bronchiectasis by CT, HRCT or bronchogram 3. Be aged 18 – 80 years inclusive 4. Have FEV1 40 - 80% predicted* or =1.0L (according to NHANESIII predicted tables) 5. Documented history of at least 2 pulmonary exacerbations, each requiring antibiotic therapy, per year for the 2 years prior to study entry 6. Have chronic sputum production of >1 tablespoonful per day on the majority of days in the 3 months prior to visit 0 7. Have chronic chest congestion ( defined as chronic excessive accumulation of mucus in the lungs) 8. Be able to perform all the techniques necessary to measure lung function Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: The subject must NOT: 1. Be investigators, site personnel directly affiliated with this study, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted. 2. Have bronchiectasis as a consequence of cystic fibrosis or focal endobronchial lesion 3. Be considered “terminally ill” or listed for transplantation 4. Be using hypertonic saline in the 4 weeks prior to commencing treatment 5. Have previously used inhaled mannitol for more than a day 6. Have had a significant episode of hemoptysis (>60 mL) in the previous 6 months 7. Have had antibiotics in the 4 weeks prior to V1 except chronic macrolide therapy 8. Have active signs and symptoms of asthma * 9. Have smoked within the last 3 months or ex-smokers with greater than 20 pack years (number of pack years = (number of cigarettes per day / 20) x number of years smoked, e.g., 20 cigarettes per day for 20 years, or 40 cigarettes per day for 10 years). 10. Have had a myocardial infarction in the three months prior to enrolment 11. Have had a cerebral vascular accident in the three months prior to enrolment 12. Have had major ocular surgery in the three months prior to enrolment 13. Have had major abdominal, chest or brain surgery in the three months prior to enrolment 14. Have a known cerebral, aortic or abdominal aneurysm 15. Have actively treated Mycobacterium tuberculosis 16. Have actively treated nontuberculous mycobacterial infection 17. Have end stage interstitial lung disease 18. Have active malignancy including melanoma (other skin carcinomas and remissions > 2 years exempted) 19. Be breast feeding or pregnant, or plan to become pregnant while in the study 20. Be using an unreliable form of contraception (female subjects at risk of pregnancy only) 21. Be participating in another investigational drug study, parallel to, or within 4 weeks of study entry 22. Have a known intolerance to mannitol or ß2-agonists 23. Have uncontrolled hypertension – e.g. for adults: systolic BP > 190 and or diastolic BP > 100 24. Subject has a condition or is in a situation which in the Investigator’s opinion may put the subject at significant risk, may confound results or may interfere significantly with the patient’s participation in the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: • To show a significant difference in the rates of graded pulmonary exacerbations, in patients with bronchiectasis treated with inhaled mannitol compared to control • To show a significant difference in Quality of Life as measured by the St. Georges Respiratory Questionnaire (SGRQ) in patients treated with inhaled mannitol compared to control ;Secondary Objective: • To show a significant improvement in other graded exacerbation parameters (time to first exacerbation and duration of exacerbation) • To show a significant difference in antibiotic use prescribed for treated pulmonary exacerbations • To show a significant difference in sputum volume • To show a significant difference in exercise tolerance • To show a significant difference in lung function • To demonstrate the safety profile of inhaled mannitol compared to control in subjects with bronchiectasis by investigating adverse events, airway reactivity, hematology, clinical chemistry, sputum microbiology and vital signs • To compare health related cost benefits of inhaled mannitol in patients with bronchiectasis. • To investigate quality adjusted life years (QALYs) by treatment group using health status data collected from EuroQol and Health Utilities Index Questionnaire ;Primary end point(s): • Graded pulmonary exacerbation rates • St Georges Respiratory Questionnaire (SGRQ) scores | — |
Countries
United Kingdom
Outcome results
None listed