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 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.
Age:
18 years and older
Mutation(s):
Two copies of F508del
FEV1% Predicted:
40 - 90%
Number of Visits:
10
Length of Participation:
74 days
Mucociliary ClearanceCompleted with results Location: USA
Phase 3 study of inhaled mannitol in adults with cystic fibrosis , protocol number NCT02134353 DPM-CF-303
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 FunctionClosed to enrollment Location: Multicentre
A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2222 in Ivacaftor-treated Subjects With Cystic Fibrosis Harbouring One F508del CFTR Mutation and a Second Gating (Class III) Mutation , protocol number NCT03045523 GLPG2222-CL-201
This clinical study is a phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel group study to evaluate two doses of orally administered GLPG2222 in adult subjects with a confirmed diagnosis of CF harbouring one F508del CFTR mutation and a second gating (class III) mutation and on stable treatment with ivacaftor. Up to 35 evaluable subjects are planned to be included in the study. Eligible subjects must be on stable treatment with physician prescribed ivacaftor (Kalydeco®) for at least 28 days at the baseline visit. They will be randomized in a 2:2:1 ratio to receive one of two active doses of GLPG2222 (150 mg q.d. or 300 mg q.d.) or placebo q.d. administered for 29 days. Subjects will be in the study for a minimum of 6 weeks and a maximum of 10 weeks, from screening until the follow-up visit.
One F508del mutation on one allele in the CFTR gene, a gating (class III) mutation (one of the following: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R) on the 2nd allele in the
(FEV1) ≥ 40% of predicted normal for age, gender and height at screening
Not specified
Not Specified
ObservationalEnrolling Location: Multiple US sites
PREDICT: NTM observational study (NTM-OB-17) , protocol number NCT02073409 NTM-OB-17
This study is taking place at multiple care centers across the U.S. It will evaluate the current standard of diagnosing nontuberculous mycobacteria (NTM) in people with CF.
10 years and older
No mutation requirement
No FEV1 limit
20
5 Years
Restore CFTR FunctionRecruiting Location: Multiple care centres across the US & VIC, Australia
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.
0 days to 24 months
One copy F508del or no copies F508del
18
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
40-100%
32 weeks
Restore CFTR FunctionCompleted with results Location: Multiple care centres across the US
Phase 2a study of Galapagos GLPG2222 in adults with CF , protocol number NCT03119649 Galapagos GLPG2222-CL-202
This study evaluated the safety and effectiveness of the CFTR modulator GLPG2222. This study was for people with CF who have two copies of the F508del mutation.
40% or greater
10 weeks
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
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