Safety and Efficacy of Bronchitol in Bronchiectasis

Condition(s) targeted: Bronchiectasis

Intervention: Mannitol (Drug); placebo (Drug)

Phase: Phase 3

Status: Active, not recruiting

Sponsored by: Pharmaxis

Official(s) and/or principal investigator(s):
Brett Charlton, Study Director, Affiliation: Pharmaxis Ltd Australia

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.

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 design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study

Primary outcome:

24 hour sputum clearance

Quality of Life SGRQ

Secondary outcome:

bronchiectasis symptoms

cough severity

exercise capacity

lung function, including gas transfer

antibiotic use

bronchial wall thickening and inflammation

peripheral airway function

adverse events

haematology, biochemistry,

sputum microbiology

Minimum age: 15 Years. Maximum age: 80 Years. Gender(s): Both.

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

Greenlane Hospital, Auckland, New Zealand

Middlemore Hospital, Auckland, New Zealand

Norfolk and Norwich University Hospital, Norwich, United Kingdom

Papworth Hospital, Cambridge, United Kingdom

North West Lung Centre, Wythshawe Hospital, Manchester, United Kingdom

Glenfield Hospital, Leicester, United Kingdom

Canberra Hospital, Canberra, Australian Capital Territory, Australia

John Hunter Hospital, Newcastle, New South Wales, Australia

Royal Prince Alfred Hospital, Camperdown Sydney, New South Wales, Australia

Royal North Shore Hospital, St Leonards, New South Wales, Australia

Bankstown Hospital, Sydney, New South Wales, Australia

St George Hospital, Kogarah, New South Wales, Australia

Belfast City Hospital, Belfast, Northern Ireland, United Kingdom

Cairns Base Hospital, Cairns, Queensland, Australia

Mater Adult Hospital, Brisbane, Queensland, Australia

Burnside War Memorial Hospital, Toorak Gardens, Adelaide, South Australia, Australia

Royal Adelaide Hospital, Adelaide, South Australia, Australia

Repatriation General Hospital, Adelaide, South Australia, Australia

The Alfred Hospital, Melbourne, Victoria, Australia

Peninsula Health Frankston Hospital, Frankston, Victoria, Australia

Royal Perth Hospital, Perth, Western Australia, Australia

AARI Sir Charles Gairdner Hospital, Perth, Western Australia, Australia

Starting date: March 2006
Ending date: August 2008
Last updated: November 7, 2007