Cystic Fibrosis MedDRA version: 9.1 Level: LLT Classification code 10011762 Term: Cystic fibrosis
Conditions
Interventions
Product Name: Inhaled dry powder mannitol
Product Code: IDPM
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: Mannitol
CAS Number: 69658
Pharmaceutical form of the placebo: I
Sponsors
Pharmaxis Pharmaceuticals Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Subjects may be included in the study if all of the following criteria are met. The subject must: 1. Have given written informed consent to participate in this study in accordance with local regulations 2. Have a confirmed diagnosis of cystic fibrosis 3. Be aged >= 6 years 4. Have FEV1 >=30 % and =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects are excluded from participating in this study if one or more of the following criteria are met. The subject must NOT: 1. Be investigators, site personnel directly affiliated with this study, and their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted. 2. Be considered “terminally ill” or listed for lung transplantation 3. Have had a lung transplant 4. Be using nebulised hypertonic saline concurrently or in the 2 weeks prior to visit 1 5. Have had a significant episode of haemoptysis (>60 mL) in the three months prior to enrolment 6. Have had a myocardial infarction in the three months prior to enrolment 7. Have had a cerebral vascular accident in the three months prior to enrolment 8. Have had major ocular surgery in the three months prior to enrolment 9. Have had major abdominal, chest or brain surgery in the three months prior to enrolment 10. Have a known cerebral, aortic or abdominal aneurysm 11. Be breast feeding or pregnant, or plan to become pregnant while in the study 12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only) 13. Be participating in another investigative drug study, parallel to, or within 4 weeks of study entry 14. Have a known allergy to mannitol 15. Be using beta blockers 16. Have uncontrolled hypertension – systolic BP > 190 and or diastolic BP > 100 17. Have a condition or be 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 18. Be ‘Aridol-MTT test positive’.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the effect of IDPM compared to control on FEV1 in patients with CF.;Primary end point(s): Change in absolute FEV1 (forced expiratory volume in 1 second).;Secondary Objective: To determine the effect of IDPM compared to control on FEV1 in patients with CF on existing RhDNase treatment. (key objective) To assess whether IDPM treatment: • Reduces pulmonary exacerbations in those taking RhDNase as a sub-group and in the total cohort (key objective) • Improves quality of life (key objective) • Reduces days on IV antibiotics, rescue oral or inhaled antibiotics • Reduces days in hospital due to pulmonary exacerbations • Improves other measures of lung function • Demonstrates an appropriate safety profile (adverse events, haematology, biochemistry, change in bronchodilator response, sputum microbiology, physical examination) • Reduces hospital and community care costs. | — |
Countries
Ireland, United Kingdom
Outcome results
None listed