Cystic Fibrosis organisations in Australia provide support and services to people with Cystic Fibrosis (CF) and their carers and families. This is complemented by a commitment to research and a quality improvement program focussing on improved clinical care for people with CF.
Every four days a baby is born in Australia with cystic fibrosis (CF) and more than one million Australians are carriers of cystic fibrosis. Cystic Fibrosis Australia (CFA) is committed to improving clinical practice and patient outcomes through its quality improvement programmes and research with the aim of extending life expectancy from 37 to 50 years by 2025.
Cystic Fibrosis is a recessive genetic condition. It primarily affects the lungs and digestive system because of a malfunction in the exocrine system, responsible for producing saliva, sweat, tears and mucus.
In addition to working for a cure, Cystic Fibrosis Australia also provides support and advocacy to improve the lives of people with cystic fibrosis. Get involved by raising awareness about CF, participating in a fundraising event or volunteering.
Cystic Fibrosis Australia has established a consistent approach to advocacy across Australia and is now a subject matter expert for government, industry and the media.
The Australian Cystic Fibrosis Research Trust (ACFRT) is managed by Cystic Fibrosis Australia (CFA). Since 1989 it has funded more than 300 projects valued at over $6,000,000.
Visit the media room to browse through number of resources including media representatives, press releases and reports.
Clinical trials are listed below.
Restore CFTR FunctionCompleted with results Location: Multiple US sites & Australia
Phase 2 study of VX-440 combination drug in people with cystic fibrosis. , protocol number NCT02951182 VX15-440-101
This study is taking place at multiple care centres accross the US. It will look at the safety and effectiveness of the drug VX-440 in combination with ivacaftor and/or tezacaftor (VX-661)
Age:
18 years and older
Mutation(s):
homozygous or hetrozygous F508del
FEV1% Predicted:
40 - 90%
Number of Visits:
11
Length of Participation:
57
ObservationalEnrolling Location: Multiple care centres across the US
GI symptoms observational study (GALAXY) , protocol number NCT03801993 GALAXY-OB-18
This study will collect information on gastrointestinal (GI) symptoms that people with CF experience.
2 years and older
No mutation requirement
No FEV1 limit
1
29 days
OtherEnrolling Location: USA
Study of SPI-1005 in people with CF ages 18 and older (Sound Pharma SPI-3005-501.2) , protocol number NCT02819856 SPI-3005-501.2
This study will test the safety and tolerability of SPI-1005, a drug intended to prevent and treat hearing loss caused by aminoglycosides.
40-100%
6
49 days
Restore CFTR FunctionEnrolling Location: Multiple care centres across the US
Phase 3 study of lumacaftor/ivacaftor (Orkambi®) in babies with two copies of the F508del CFTR mutation , protocol number NCT03601637 VX16-809-122
This study is taking place at multiple care centers across the U.S. It will evaluate the safety of the drug lumacaftor/ivacaftor (Orkambi®) and its effect on the body. It is for babies ages 1 to less than 2 years old who have cystic fibrosis and two copies of the F508del CFTR mutation.
1-2 years
Two copies of F508del
38
Sweat chloride observational study (CHEC-OB-17) , protocol number NCT03350828 CHEC-OB-17
This study is taking place at multiple care centers across the U.S. It will look at sweat chloride concentration in people who are currently taking CFTR modulators. Researchers will test the effectiveness of CFTR modulators by measuring change in sweat chloride concentration. Sweat chloride measures that are collected in the study visit will be analyzed in combination with data from the CF Foundation Patient Registry. This study is for people with cystic fibrosis who are enrolled in the CF Foundation Patient Registry and have been taking a CFTR modulator for at least three months before enrolling. This study will consist of a single study visit and will require a sweat test.
4 months and older
1 Day
Restore CFTR FunctionEnrolling Location: Multiple US sites
Phase 3 study of VX-659 triple combination drug in people with CF ages 12 years and older who have two copies of the F508del mutation (VX17-659-103) , protocol number NCT03460990 VX17-659-103
This randomized, placebo-controlled study is taking place at multiple care centers across the U.S. It will look at the effectiveness and safety of the drug VX-659 in combination with ivacaftor and tezacaftor. It is for people with CF ages 12 and older who have two copies of the F508del CFTR mutation.
12 years and older
7
10 weeks
Nutritional-GICompleted with results Location: Multiple care centres across the US
OPTION: Study of AzurRx MS1819 in people with cystic fibrosis (CF) and exocrine pancreatic insufficiency who are 18 years and older (AzurRX AZ-CF2001) , protocol number NCT03746483 AzurRX AZ-CF2001
This study evaluated the safety and effectiveness of the drug MS1819 as a pancreatic enzyme replacement therapy (PERT). MS1819 is a non-porcine (not pig-derived) lipase-only enzyme for people with CF who have exocrine pancreatic insufficiency (EPI).
30% or greater
10
11 weeks
OtherActive, not recruiting Location: Europe/UK
A Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic Fibro , protocol number A Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic Fibro NCT03698448
Randomized, double blind, placebo controlled study. The study has two parts: Dose-finding part, followed by longer term follow-up (6 months)
Not specified
Not Specified
Restore CFTR FunctionCompleted with results Location: Multiple care centres across the US
Phase 2 study of PTI-428 drug in people with CF ages 18 and older who have two copies of the F508del CFTR mutation (Proteostasis PTI-428-06) , protocol number NCT03591094 PTI-428-06
This study evaluated the safety and tolerability of the drug PTI-428. This study was for people with CF ages 18 and older who have two copies of the F508del CFTR mutation and were already taking tezacaftor/ivacaftor (Symdeko®). A key goal of the study was to determine if PTI-428 could increase the amount of CFTR protein produced in people who were already taking tezacaftor/ivacaftor (Symdeko®).
74 days
Mucociliary ClearanceCompleted with results Location: USA
Phase 3 study of inhaled mannitol in adults with cystic fibrosis , protocol number NCT02134353 DPM-CF-303
This study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of the inhaled drug mannitol and will use a placebo control.
18 Years and Older
No Mutation Requirements
40 to 90%
5