Cystic fibrosis in children aged 6 to 17 years MedDRA version: 14.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Bronchitol
Product Name: Bronchitol
Product Code: N/A
Pharmaceutical Form: Inhalation powder, hard capsule
INN or Proposed I
Sponsors
Pharmaxis Ltd.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Personally provide, or have a legal guardian provide written informed consent to participate in the trial, according to local regulations; 2. rhDNase and maintenance antibiotic use is allowed but treatment must have been established at least 3months prior to screening. The subject must remain on rhDNase and / or maintenance antibiotics for the duration of the trial. The subject must not commence treatment with rhDNase or maintenance antibiotics during the trial; 3. Have a confirmed diagnosis of cystic fibrosis (sweat test result >= 60 mEq/L chloride and/or genotyping showing two identifiable mutations consistent with a diagnosis of cystic fibrosis); 4. Be aged >= 6 years and = 30% and = 8 years; and 6. Be able to perform all the techniques necessary to measure lung function. Are the trial subjects under 18? yes Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range 0 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Be using maintenance nebulised hypertonic saline Be considered “terminally ill”; eligible for lung transplantation, or have received a lung transplant previously; 2. Require home oxygen or assisted ventilation; 3. Have had an episode of massive haemoptysis defined as acute bleeding =240 ml in a 24-hour period and/or recurrent bleeding =100 ml/day over several days in the three-months prior to Screening (Visit 0); 4. Have a known intolerance to mannitol; 5. Be taking non-selective beta blockers; 6. In the three months prior to Screening (Visit 0) have had a myocardial infarction; a cerebral vascular accident; major ocular, abdominal, chest or brain surgery; 7. Have a known cerebral, aortic or abdominal aneurysm; 8. Be currently participating in, or have participated in another investigative drug trial within four weeks of Screening (Visit 0); 9. Be pregnant or breastfeeding, or plan to become pregnant whilst in the trial; 10. For females of childbearing potential (at the discretion of the investigator), be using an unreliable form of contraception; 11. Have a “failed” or “incomplete” mannitol tolerance test; or 12. Have any concomitant medical, psychiatric, or social condition that, in the Investigator’s opinion, would put the subject at significant risk, may confound the results or may significantly interfere with the subject’s participation in the trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the effect of eight weeks of twice-daily treatment with inhaled dry powder mannitol on lung function (FEV1) in subjects with CF who are aged six to seventeen years; Secondary Objective: • To determine the effect of inhaled mannitol on FVC; • To determine the effect of inhaled mannitol on FEF25-75 (exploratory objective) and • To assess the safety of inhaled mannitol. • To evaluate the difference in treatment induced sputum weight in patients treated with inhaled mannitol compared to those treated with placebo ;Primary end point(s): The absolute change from each baseline to week 8 of each treatment period in percentage of predicted FEV1.;Timepoint(s) of evaluation of this end point: Endpoint will be evaluated at the end of the trial. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change in percentage of predicted FVC between placebo and mannitol treatment • Change in percentage of predicted FEF25-75 between placebo and mannitol treatment (exploratory endpoint) • Adverse events, vital signs and physical examination • Treatment induced sputum weight ;Timepoint(s) of evaluation of this end point: Endpoints will be evaluated at the end of the trial. | — |
Countries
Argentina, Belgium, Canada, France, Italy, Netherlands, United Kingdom
Outcome results
None listed