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
Phase 3 study of long-term ivacaftor in babies who have a CFTR gating mutation , protocol number NCT03277196 VX-770-126
This open-label study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of long-term ivacaftor in babies who have a CFTR gating mutation.
Age:
0 days to 24 months
Mutation(s):
One copy F508del or no copies F508del
FEV1% Predicted:
No FEV1 limit
Number of Visits:
18
Length of Participation:
128 weeks
Anti-InflammatoryEnrolling Location: Multiple care centres across the US
Phase 2 study of lenabasum in people with CF ages 12 and older (Corbus JBT101-CF-002) , protocol number NCT03451045 JBT101-CF-002
This study is taking place at multiple care centers across the U.S. It will look at the safety and effectiveness of the anti-inflammatory drug lenabasum and will use a placebo control.
12 years and older
No mutation requirement
40-100%
10
32 weeks
Phase 2a study of Galapagos GLPG2222 in adults with CF , protocol number Galapagos GLPG2222-CL-202
This study will look at two different dose levels of GLPG2222. The study is placebo-controlled, which means that some participants will receive the study drug and others will receive a placebo. Participants will receive either the study drug or the placebo for 29 days and will be in the study for six to 10 weeks. Researchers will test the drug's safety and effectiveness by monitoring adverse events and other measures.
18 years and older
Two copies of F508del
40% or greater
5
10 weeks
ObservationalEnrolling Location: Multiple US sites
Rare Mutation Cell Collection (RARE) , protocol number NCT 03161808 NCT 03161808
This study is taking place at multiple care centres across the US. Researchers will collect and make available for study cells from people with rare CFTR mutations.
2 years and older
40-90%
8
30 days
Restore CFTR FunctionClosed to enrollment Location: USA, Australia, Europe
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor , protocol number NCT02549451 VX15-809-110
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
6-11 years and 12 years and older
Homozygous for the F508del-CFTR Mutation
Not Specified
Not specified
Mucociliary ClearanceEnrolling Location: Multiple care centres across the US
Phase 1Bstudy of inhaled AZD5634 in adults with CF , protocol number NCT02950805
This study is taking place at multiple care centres accross the US. It will look at the safety and tolerability of the inhaled drug AZD5634 in adults with CF.
18 - 60 years
4
4 months
Anti-InfectiveEnrolling Location: Multiple care centres across the US
Phase 3 study of inhaled vancomycin in adults and children 6 years and older with cystic fibrosis. , protocol number SAVARA SAV005
This study is taking place at multiple care centers across the U.S. It will look at the effectiveness of the inhaled drug vancomycin in adults and children 6 years and older with cystic fibrosis and positive cultures for methicillin-resistant Staphylococcus aureus (MRSA).
6 years and older
30-100%
12 months
Restore CFTR FunctionClosed to enrollment Location: Multicentre
A Phase 3 Study of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor , protocol number NCT02412111 VX14-661-109
This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.
Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
Nutritional-GICompleted with results Location: Multiple care centres across the US
RESULT: Study of liprotamase non-porcine enzymes , protocol number NCT03051490
This study evaluated the effectiveness of liprotamase, a non-pig-derived pancreatic enzyme replacement therapy (PERT), as compared to Pancreaze®, a pig-derived PERT.
7 years and older
17
30 weeks
Restore CFTR FunctionCompleted with results Location: Multiple US sites
Vertex Lumacaftor (VX-809) and Ivacaftor in children with CF aged 6 to 11 years and have two copies of the delF508 CFTR mutation. , protocol number VX13-809-011b
This was an open-label study designed to look at the safety of lumacaftor in combination with ivacaftor (Orkambi). This study was for younger children with CF who have two copies of the F508del CFTR mutation.
6-11 years
11
27 weeks