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.
ObservationalClosed to enrollment Location: USA
An Ocular Safety Study of Kalydeco-Treated Children 11 Years of Age or Younger with CF , protocol number Vertex VX-770-115
This trial is designed to evaluate the risk of of cataracts (lens opacities) and describe the best corrected distance vision (with glasses/contacts for those who wear them) of children with Cystic Fibrosis who are 11 years of age or younger at the time of ivacaftor (Kalydeco) treatment initiation and are receiving or planning to receive commercially-available ivacaftor (Kalydeco) in the US.
Age:
6 Years to 11 Years
Mutation(s):
One Copy F508del or No Copies F50del
FEV1% Predicted:
No FEV1 Limit
Number of Visits:
5
Length of Participation:
2 years
Restore CFTR FunctionCompleted with results Location: USA
Phase 3 study of lumacaftor and ivacaftor in which cystic fibrosis with advanced lung disease. , protocol number Vertex VX-809-106
This study is taking place at multiple care centers across the U.S. It will evaluate the safety of the drugs lumacaftor and ivacaftor in people with advanced lung disease.
12 Years and Older
Two copies F508del
Less than 40%
14
52 weeks
Restore CFTR FunctionClosed to enrollment Location: USA
Phase 3 study of lumarcaftor and ivacaftor in children with cystic fibrosis , protocol number Vertex VX-809-109
This study is taking place at multiple care centers across the U.S. It will evaulate the safety and effectiveness of the drugs lumacaftor and ivacaftor in children with cystic fibrosis.
70% to 105%
24 weeks
BehavioralCompleted with results Location: Multiple care centres across the US
Study of adherence to lumacaftor/ivacaftor using a smart pill bottle device. , protocol number NCT02823470 VERTEX VX-809-114
This study evaluated the impact of a smart pill bottle device on adherence to lumacaftor/ivacaftor (Orkambi®). This study was for people with CF who were not currently being treated with lumacaftor/ivacaftor (Orkambi®) prior to enrolling in the study.
16 Years and Older
Two Copies F508del
40% or greater
7
48 weeks
Restore CFTR FunctionEnrolling Location: Multiple US sites
Phase 2 study of CTP-656 in aduts with CF who are currently taking ivacaftor (Kalydeco) , protocol number NCT02971839 CP656.2001
This study will evaluate the safety and effectiveness of the drug, CTP-656 in adults with Cystic Fibrosis who have a gating mutation and are currently taking ivacaftor (KALYDECO)
18 years and older
One copy F508del or no copies F508del
60-90%
6
7 weeks
Restore CFTR FunctionEnrolling Location: Multiple care centres across the US
RESTORE-CF: Phase 1/2 study of MRT5005 in adults with cystic fibrosis (Parts A & B) , protocol number NCT03375047 -Translate Bio RESTORE-CF MRT5005-101
This first human study is taking place at multiple care centers across the US. It will look at the safety and tolerability of different doses of the nebulized drug MRT5005 in adults with CF.
No mutation requirement
50-90%
21
12 months
Phase 3 study of VX-445 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 (Vertex VX17-445-102) , protocol number NCT03525444 VX17-445-102
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-445 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
40-90%
10
32 weeks
Phase 3 study of VX-445 triple combination drug in people with CF ages 12 years and older who have two copies of the F508del mutation (Vertex VX17-445-103) , protocol number NCT03525548 VX17-445-103
This randomized, 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-445 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.
Two copies of F508del
40 - 90%
10 weeks