Clinical trial NCT00277537: Safety and Efficacy of Bronchitol in Bronchiectasis
Official title: A Phase III Multicenter, Randomised, Parallel, Placebo-Controlled, Double-Blind Study to Investigate the Safety and Efficacy of Treatment With Bronchitol (Dry Powder Mannitol) in the Symptomatic Treatment of Bronchiectasis.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Bronchiectasis
Keywords: Mannitol
Lead sponsor: Syntara
Sponsor class: INDUSTRY
First posted: 2006-01-16
Enrollment: 354
Sex: ALL
Minimum age (years): 15
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Study will assess the safety and effectiveness of 12 week treatment with the study medication, Bronchitol, in subjects with bronchiectasis (a lung disease where patients have trapped, and often infected, thick, sticky mucus). Past studies have shown Bronchitol inhalation may help to facilitate the clearance of mucus by altering its rheology (making it less thick and sticky), thereby enhancing the shift of stagnant mucus from the lungs. On completion of the double blind phase, subjects will have the opportunity to participate in a 52 week open label phase.


Eligibility criteria: Inclusion Criteria:

* Non cystic fibrosis bronchiectasis
* Have FEV1 50% - 80% predicted and ≥1.0L
* Have chronic sputum production of \>10 mL per day on the majority of days in the 3 months prior to study entry

Exclusion Criteria:

* Be using hypertonic saline or mucolytic pharmacological agents concurrently or in the 4 weeks prior to study entry
* Have airway hyperresponsiveness as defined by a positive Aridol challenge

Primary outcomes:
measure: 24 hour sputum clearance
timeFrame: 24 hours / 12 weeks
measure: Quality of Life SGRQ
timeFrame: 12 weeks
Secondary outcomes:
measure: bronchiectasis symptoms
timeFrame: 12 weeks
measure: cough severity
timeFrame: 12 weeks
measure: exercise capacity
timeFrame: 12 weeks
measure: lung function, including gas transfer
timeFrame: 12 weeks
measure: antibiotic use
timeFrame: 12 weeks
measure: bronchial wall thickening and inflammation
timeFrame: 12 weeks
measure: adverse events
timeFrame: 12 weeks / 12 months
measure: haematology, biochemistry,
timeFrame: 12 weeks / 12 months
measure: sputum microbiology quantitative and qualitative
timeFrame: 12 weeks / 12 months
Other outcomes:
Locations:
city: Canberra
country: Australia
facility: Canberra Hospital
state: Australian Capital Territory
city: Camperdown Sydney
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Kogarah
country: Australia
facility: St George Hospital
state: New South Wales
city: Newcastle
country: Australia
facility: John Hunter Hospital
state: New South Wales
city: St Leonards
country: Australia
facility: Royal North Shore Hospital
state: New South Wales
city: Sydney
country: Australia
facility: Bankstown Hospital
state: New South Wales
city: Brisbane
country: Australia
facility: Mater Adult Hospital
state: Queensland
city: Cairns
country: Australia
facility: Cairns Base Hospital
state: Queensland
city: Adelaide
country: Australia
facility: Repatriation General Hospital
state: South Australia
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Toorak Gardens, Adelaide
country: Australia
facility: Burnside War Memorial Hospital
state: South Australia
city: Frankston
country: Australia
facility: Peninsula Health Frankston Hospital
state: Victoria
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Perth
country: Australia
facility: AARI Sir Charles Gairdner Hospital
state: Western Australia
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: Auckland
country: New Zealand
facility: Greenlane Hospital
city: Auckland
country: New Zealand
facility: Middlemore Hospital
city: Belfast
country: United Kingdom
facility: Belfast City Hospital
state: Northern Ireland
city: Cambridge
country: United Kingdom
facility: Papworth Hospital
city: Leicester
country: United Kingdom
facility: Glenfield Hospital
city: Manchester
country: United Kingdom
facility: North West Lung Centre, Wythshawe Hospital
city: Norwich
country: United Kingdom
facility: Norfolk and Norwich University Hospital
Study officials:
affiliation: Pharmaxis Ltd Australia
name: Brett Charlton
role: STUDY_DIRECTOR
References:
citation: Bilton D, Daviskas E, Anderson SD, Kolbe J, King G, Stirling RG, Thompson BR, Milne D, Charlton B; B301 Investigators. Phase 3 randomized study of the efficacy and safety of inhaled dry powder mannitol for the symptomatic treatment of non-cystic fibrosis bronchiectasis. Chest. 2013 Jul;144(1):215-225. doi: 10.1378/chest.12-1763.