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 care centres across the US
Roll-over study of ivacaftor in children with CF who have a CFTR gating mutation (Vertex VX-770-109) , protocol number NCT01946412 VERTEX VX-770-109
This study evaluated the long-term safety of ivacaftor (Kalydeco®) in children with CF. This study was for children who have a CFTR gating mutation and received at least one dose of ivacaftor in the VX11-770-108 study.
Age:
2 years to 5 years
Mutation(s):
One Copy F508del or No Copies F508del
FEV1% Predicted:
No FEV1 Limit
Number of Visits:
7
Length of Participation:
88 weeks
Open-label study of ivacaftor in people with CF who have a non-G551D CFTR gating mutation (Vertex VX-770-112) , protocol number NCT01707290 VERTEX VX-770-112
This study evaluated the long-term safety of ivacaftor (Kelydeco®) in participants who have a non-G551D CFTR gating mutation. This study was for people who had participated in one of the VX11-770-110, VX12-770111 or VX12-770-113 studies.
6 Years and Older
2 years
Mucociliary ClearanceEnrolling Location: Florida, USA
Study of BI 1265162, a mucus clearance drug, in teens and adults with CF. , protocol number NCT04059094 BI 1399-0003
This study will look at the safety and effectiveness of BI 1265162, a drug intended to improve the clearance of mucus from the lungs. This study is for people with CF ages 12 and older.
12 Years and Older
No Mutation Requirement
40 to 90%
5
7 weeks
ObservationalEnrolling Location: Multiple care centres across the US
Study to evaluate the effects of the triple-combination modulator, elexacaftor/texacaftor/ivacaftor (PROMISE) , protocol number NCT04038047 PROMISE
This observational study will measure the effects of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor, in people with CF. These drugs are intended to help CFTR protein function closer to normal. This study will look at how treatment with the triple-combination therapy affects people with CF across many different aspects of the disease.
Two Copies F508del or One Copy F508del
6
Restore CFTR FunctionEnrolling Location: Multiple care centres across the US
Phase 2 study of PTI-808 triple-combination therapy in adults with cystic fibrosis who have at least one copy of the F508del mutation. , protocol number NCT03251092 PTI-808
This study will look at the safety and tolerability of PTI-808, a modulator intended to help CFTR protein function closer to normal. PTI-808 will be tested in combination with two other modulators, PTI-801 and PTI-428.
18 Years and Older
Two copies F508del or One Copy F508del
9
10 weeks
Study of ABBV-3067 and ABBV-2222 in adults with cystic fibrosis who have two copies of the F508del mutation. , protocol number NCT03969888 AbbVie ABBV-3067
This study will look at the safety and effectiveness of ABBV-3067, a CFTR modulator intended to help CFTR protein function closer to normal. ABBV-3067 will be tested alone and in combination with another CFTR modulator, ABBV-2222. This study has two parts. In Part 1, participants will be randomly assigned to receive one of the following treatments: the study drug ABBV-3067 alone; both ABBV-3067 and ABBV-2222; or a placebo. In Part 2 of the study, some participants will receive both CFTR modulators ABBV-3067 and ABBV-2222, and some participants will receive a placebo. Researchers will test the effectiveness of ABBV-3067 alone and in combination with ABBV-2222 by measuring changes in lung function and sweat chloride. This study may require lung function tests and/or other measurements.
18 years and Older
Two Copies F508del
3 months
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.
10
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
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