Cystic Fibrosis MedDRA version: 19.0 Level: LLT Classification code 10011764 Term: Cystic fibrosis NOS System Organ Class: 100000004850
Conditions
Interventions
Trade Name: Bronchitol
Product Name: Bronchitol
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed INN: Mannitol
Other descriptive name: MANNITOL
Concentration unit: mg milligram(s)
Sponsors
Pharmaxis Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: The subject must meet all of the following criteria: 1. Have given written informed consent to participate in this trial in accordance with local regulations; 2. Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value = 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype); 3. Be aged at least 18 years old; 4. Have FEV1 > 40 % and =65 years) yes F.1.3.1 Number of subjects for this age range 22
Exclusion criteria
Exclusion criteria: The subject must NOT meet any of the following criteria: 1. Be investigators, site personnel directly affiliated with this trial, or 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 eligible for lung transplantation; 3. Have had a lung transplant; 4. Be using maintenance nebulized hypertonic saline in the 2 weeks prior to visit 1; 5. Have had a significant episode of hemoptysis (> 60 mL) in the three months prior to Visit 0; 6. Have had a myocardial infarction in the three months prior to Visit 0; 7. Have had a cerebral vascular accident in the three months prior to Visit 0; 8. Have had major ocular surgery in the three months prior to Visit 0; 9. Have had major abdominal, chest or brain surgery in the three months prior to Visit 0; 10. Have a known cerebral, aortic or abdominal aneurysm; 11. Be breast feeding or pregnant, or plan to become pregnant while in the trial; 12. Be using an unreliable form of contraception (female subjects at risk of pregnancy only); 13. Be participating in another investigative drug trial, parallel to, or within 4 weeks of screening (Visit 0); 14. Have a known allergy to mannitol; 15. Be using non-selective oral beta blockers; 16. Have uncontrolled hypertension –i.e. 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 subject’s participation in the trial;or 18. Have a failed or incomplete mannitol tolerance test (MTT) (as evaluated in Section 8.1.1.1).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control (50mg b.i.d) for improving lung function as measured by mean change from baseline FEV1 (mL) over the 26-week treatment period in adult subjects with cystic fibrosis (CF).;Secondary Objective: 1. To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control for improving lung function as measured by mean change from baseline FVC (mL) over the 26-week treatment period in adult subjects with CF; 2. To determine whether inhaled mannitol (400 mg b.i.d.) is superior to control in increasing the time to first pulmonary exacerbation over the 26-week treatment period in adult subjects with CF; 3. To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for reducing the number of days on antibiotics (oral, inhaled or IV) due to pulmonary exacerbation 4. To determine whether in adult subjects with CF, inhaled mannitol (400 mg b.i.d.) is superior to control for decreasing the number of days in hospital due to pulmonary exacerbation; and 5. To determine whether inhaled mannitol (400 mg b.i.d.) decreases the rate of pulmonary exacerbations over the 26-week treatment period compared to control in adult subjects with CF. ;Primary end point(s): The primary endpoint is the mean change in FEV1 (mL) from baseline (Visit 1) over the 26-week treatment period (to Visit 4).;Timepoint(s) of evaluation of this end point: visit 1 and visit 4 | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: Throughout the 26 week treatment period;Secondary end point(s): • Mean change from baseline FVC (mL) over the 26-week treatment period; • Time to first pulmonary exacerbation over the 26-week treatment period; • Rate of pulmonary exacerbations over the 26-week treatment period; • Number of days in hospital due to pulmonary exacerbation; • The incidence of pulmonary exacerbations; • Number of days on antibiotics (oral, inhaled or IV) due to pulmonary exacerbation; • Ease of expectoration measured using a visual analogue scale; and • CFQ-R respiratory domain score. | — |
Countries
Argentina, Belgium, Bulgaria, Canada, Czech Republic, France, Greece, Hungary, Israel, Italy, Mexico, Poland, Romania, Russian Federation, Slovakia, South Africa, Spain, Sweden, Ukraine, United Kingdom, United States
Contacts
Public ContactBrett Charlton
Pharmaxis Limited
Outcome results
None listed