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 FunctionEnrolling Location: Multiple care centres across the US
Study to evaluate VX-121 in adults 18 years and older with cystic fibrosis , protocol number NCT03912233 VERTEX VX-18-121-101
This study will look at the safety, tolerability, and effectiveness of the drug VX-121, a drug intended to help CFTR function closer to normal. This study will take place in two parts. Part 1 is randomized and placebo-controlled. This means that some participants will receive the study drug in combination with tezacaftor and VX-561, and some participants will receive placebo. Part 2 is a randomized, active-comparator trial. This means that some participants will receive the study drug in combination with tezacaftor and VX-561, and some participants will receive placebo in combination with tezacaftor and ivacaftor. In this study, researchers will test the safety and tolerability by monitoring for adverse events. They will test for effectiveness by measuring lung function. They will also monitor sweat chloride and how much of the drug stays in the body. This study may require sweat tests, lung function tests, blood samples and/or other measures.
Age:
18 years and Older
Mutation(s):
Two Copies F508del or One Copy F508del
FEV1% Predicted:
40 to 90%
Number of Visits:
10
Length of Participation:
17 weeks
Anti-InfectiveEnrolling Location: Multiple care centres across the US
Study to evaluate inhaled molgramostim in adults 18 years and older with cystic fibrosis (Savara SAV008-02) , protocol number NCT03597347 SAVARA SAV008-02
This study will look at the effectiveness of the inhaled drug molgramostim, a drug intended to treat infections in the lung. This study is open-label with no control. This means that all participants will receive the study drug. In this study, researchers will test the effectiveness of inhaled molgramostim by monitoring NTM cultures. They will also monitor lung function, BMI and quality of life. This study may require sputum samples, lung function tests and/or other measures.
18 years and older
No mutation requirement
30% or greater
19
72 weeks
Restore CFTR FunctionCompleted with results Location: USA & Australia
Phase 3 study of inacaftor in babies who have a CFTR gating mutation , protocol number NCT02725567 VX15-770-124
This two-part, open label study is taking place at multiple care centers across the US. It will look at the safety and effectiveness of ivacaftor, as well as how the body processes the drug, in babies who have a CFTR gating mutation.
0 Months to 24 Months
One Copy F508del or No Copies F508del
No FEV1 Limit
8
24 weeks
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)
homozygous or hetrozygous F508del
40 - 90%
11
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 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
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-GIEnrolling Location: Multiple care centres across the US
OPTION: Study of AzurRx MS1819 in people with cystic fibrosis and exocrine pancreatic insufficiency who are 18 years and older , protocol number NCT03746483 AzurRX AZ-CF2001
This trial will look at the safety and effectiveness of the drug MS1819 as a pancreatic enzyme replacement therapy. Sponsor: AzurRx BioPharma, Inc
11 weeks