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 lumacaftor/ivacaftor (Orkambi®) in babies with two copies of the F508del CFTR mutation (Vertex VX-16-809-122) , protocol number NCT03601637 VX-16-809-122
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.
Age:
1-2 years
Mutation(s):
Two copies of F508del
FEV1% Predicted:
No FEV1 limit
Number of Visits:
11
Length of Participation:
38
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 randomized, placebo-controlled study is taking place at multiple care centers across the U.S. It will look at the safety and tolerability of the drug PTI-428 and how it is processed by the body. It is for people with CF ages 18 and older who have two copies of the F508del CFTR mutation and are already taking tezacaftor/ivacaftor.
18 years and older
40 - 90%
10
74 days
ObservationalEnrolling Location: Queensland University of Technology Contact: Jeremy Russell - phone:07 3069 7552 email: j21.russell@hdr.qut.edu.au. To get started, go to: https://rebrand.ly/HYPRS-CF
Helping Young People Reduce the Stress of Cystic Fibrosis Study , protocol number The HYPRS-CF Study 1800000294 (UHREC Reference number)
Researchers at QUT are conducting a national, online research study with young people with Cystic Fibrosis (CF). The goal of this study is to learn how young people think and feel about the stress of CF and whether a social robot could possibly help them cope with this stress.
10-18 years
No mutation requirement
1
Not Specified
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
12 years and older
One copy F508del
32 weeks
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.
7
10 weeks
Mucociliary ClearanceCompleted with results Location: USA
Phase 3 study of inhaled mannitol in adults with cystic fibrosis , protocol number DPM-CF-303 NCT02134353
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
6
Restore CFTR FunctionEnrolling Location: Australia - QLD
A Study to Evaluate Safety, PK and PD of FDL169 in Cystic Fibrosis Subjects , protocol number FDL169-2015-04 NCT03093714
A Randomized, Double-Blind, Placebo-Controlled, Parallel Study to Evaluate Safety, Pharmacokinetics (PK) and Pharmacodynamics(PD) of FDL169 in Cystic Fibrosis (CF) Subjects Homozygous for the F508del-CFTR Mutation
18 Years to 85 Years
Homozygous for the F508del-CFTR mutation.
(FEV1) >40% of predicted normal for age, sex and height.
Not specified
Mucociliary ClearanceEnrolling Location: Australian Sites: Adelaide, Brisbane, Melbourne, Newcastle, Sydney Westmead, Sydney Randwick, Perth
Saline Hypertonic in pre-schoolers + CT (SHIP-CT) , protocol number SHIP002 (SHIPCT) NCT02950883 & ACTRN12615001067561p
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
54 weeks
Restore CFTR FunctionClosed to enrollment Location: LOCATION: Perth Linear Clinical Research, Contact: Jennifer Seabourne CONTACT: Greenteam@linear.org.au. Brisbane-Q-pharm CONTACT: Sharon Rankine Phone: 0439 664 900,; Email: s.rankine@qpharm.com.au
A Study to Assess the Safety, Tolerability and PK Profile of FDL176 in Healthy and CF Participants , protocol number FDL176-2016-01 NCT03173573
This is a 5-part study. Part 1 is a double blind, placebo-controlled, dose escalation, first-in-human study to assess the safety, tolerability and PK profiles following single oral administration of FDL176 to healthy male participants. Part 2 is a single dose, open-label study in healthy male participants to determine the effect of food on the PK profile of FDL176. Part 3 is a single dose, double blind, placebo-controlled study in healthy female participants to assess the PK, safety and tolerability profiles of FDL176. Part 4 is a randomised, double-blind, placebo-controlled, dose-escalation study to assess the safety, tolerability and PK profiles following multiple oral administrations of FDL176 to healthy male and female participants. Part 5 is a single dose, open-label study in male and female participants with CF to determine the PK profile of FDL176.
18 Years to 55 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 PTC124-GD-021-CF NCT02139306
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
One Copy F508del or No Copies F508del
60 to 90%
56 weeks