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 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
Study to evaluate VX-561 in adults 18 years and older with cystic fibrosis , protocol number VERTEX VX-561-101
This study will look at the safety and effectiveness of the drug VX-561, a drug intended to help CFTR function closer to normal. This study is randomized and placebo-controlled. This means that some participants will receive one of four dose levels of the study drug, some participants will receive ivacaftor, and some participants will receive placebo. In this study, researchers will test the effectiveness of VX-561 by monitoring lung function. They will also monitor sweat chloride and adverse events. This study may require lung function tests, blood samples, and/or other measures.
One Copy F508del or No Copies F508del
40 to 100%
8
16 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 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
OtherEnrolling Location: Monash Health - Melbourne
Cystic Fibrosis Related Diabetes (CFRD) Model of care: Optimising Patient Centred Healthcare Delivery in the Inpatient and Outpatient Setting , protocol number RES-18-0000-183 Monash Health Ref RES-18-0000-183 Monash Health Ref
Cystic Fibrosis Related Diabetes Clinical Care Do you have cystic fibrosis related diabetes (CFRD)? Do you feel that clinical services managing CFRD could be improved upon? We want to hear your thoughts on how clinical care for CFRD can be optimised. The purpose of this research is to determine the requirements of adult patients with CFRD. We wish to assess barriers and enablers to provision of optimal care of adults with CFRD with a view to ultimately improving clinical outcomes for all people with CFRD. If you would like to participate, please follow the link to an anonymous survey that should take about 15 minutes to complete. Click here to complete CFRD survey This research has been initiated by the researchers at Monash Medical Centre in Melbourne, Victoria. This project has been approved by Monash Health Human Research Ethics Committee.
Not Specified
Not specified
1
approx. 30 mins
OtherEnrolling Location: NSW - Australia
A Phase 3 multi-centre randomised placebo-controlled study of azithromycin in the primary prevention of radiologically-defined bronchiectasis in infants with cystic fibrosis (COMBATCF). , protocol number NCT01270074 ACTRN12610001072000
The general aim of this project is to conduct a randomized double-blind, placebo-controlled clinical trial of azithromycin to determine whether treatment from infancy is safe and will prevent the onset of bronchiectasis. One hundred and thirty infants will be recruited from CF clinics in Australia and New Zealand and treated from 3 months to three years of age. The primary outcome will be the proportion with radiologically-defined bronchiectasis at 3 years of age. Safety and mechanistic evaluations will also be undertaken.
6 Weeks to 6 Months
0
Mucociliary ClearanceEnrolling Location: Australian Sites: Adelaide, Brisbane, Melbourne, Newcastle, Sydney Westmead, Sydney Randwick, Perth
Saline Hypertonic in pre-schoolers + CT (SHIP-CT) , protocol number NCT02950883 & ACTRN12615001067561p SHIP002 (SHIPCT)
The purpose of this study is to assess whether inhalation of 7% hypertonic saline (HS) twice daily for 48 weeks reduces structural lung disease as assessed by computed tomography (CT) in comparison with inhalation of 0.9% isotonic saline (IS) in preschool children (ages 3 to 6) with cystic fibrosis.
3-5 years
A documented genotype with two disease-causing mutations in the CFTR gene
6
54 weeks
Randomised controlled trial to be determine whether once-daily insulin detemir slows decline in weight and lung functions in patients with cystic fibrosis and early insulin deficiency. , protocol number NCT01100892 ACTRN13611000068965
Cystic fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and eventually diabetes. The CF-IDEA trial (Cystic Fibrosis - Insulin Deficiency, Early Action) will determine whether starting insulin treatment before the onset of diabetes (earlier than current practice) will improve the health of children with CF by improving body weight and lung function.
5 Years to 19 Years
Restore CFTR FunctionCompleted with results Location: USA & Australia
Phase 3 study of Ataluren in people with CF who have a nonsense mutation and are not taking aminoglycosides. , protocol number NCT02139306 PTC124-GD-021-CF
This study is taking place at multiple care centers across the U.S. It looked at the effectiveness and safety of the oral drug ataluren in people with CF who have a nonsense mutation of cystic fibrosis. This study used a placebo control.
6 Years and Older
60 to 90%
56 weeks
Restore CFTR FunctionEnrolling Location: Multiple care centres across the US & Australia
A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis Who Have an F508del-CFTR Mutation , protocol number NCT02565914 VX14-661-110
This is a Phase 3, multicenter, open-label, 2-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in Studies NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111, and NCT02508207. The study is designed to evaluate the safety and efficacy of long term treatment of VX-661 in combination with ivacaftor.
12 years and older
Restore CFTR FunctionEnrolling Location: Multicentre - Australia 1. Prince Charles Hospital, 2. Lady Cilento, 3. SCGH
Phase 3 study of VX-659 triple combination drug in people with CF 12 years and older who have one copy of the F508del mutation and one copy of a minimal function mutation (VX17-659-102) , protocol number NCT03447249 VX17-659-102
This randomized, placebo-controlled study will be taking place at multiple care centers across the U.S. It will evaluate the effectiveness, safety, and effect on the body of the drug VX-659 in combination with tezacaftor and ivacaftor. It is for people with cystic fibrosis ages 12 and older with one copy of the F508del CFTR mutation and one copy of a minimal function mutation
One copy F508del
40 - 90%
32 weeks