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 , protocol number NCT03601637 VX16-809-122
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
ObservationalEnrolling Location: Multiple care centres across the US
Sweat chloride observational study (CHEC-OB-17) , protocol number NCT03350828 CHEC-OB-17
This study is taking place at multiple care centers across the U.S. It will look at sweat chloride concentration in people who are currently taking CFTR modulators. Researchers will test the effectiveness of CFTR modulators by measuring change in sweat chloride concentration. Sweat chloride measures that are collected in the study visit will be analyzed in combination with data from the CF Foundation Patient Registry. This study is for people with cystic fibrosis who are enrolled in the CF Foundation Patient Registry and have been taking a CFTR modulator for at least three months before enrolling. This study will consist of a single study visit and will require a sweat test.
4 months and older
No mutation requirement
1
1 Day
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.
12 years and older
40 - 90%
7
10 weeks
Nutritional-GIEnrolling Location: Multiple care centres across the US
OPTION: Study of AzurRx MS1819 in people with cystic fibrosis and exocrine pancreatic insufficiency who are 18 years and older , protocol number NCT03746483 AzurRX AZ-CF2001
This trial will look at the safety and effectiveness of the drug MS1819 as a pancreatic enzyme replacement therapy. Sponsor: AzurRx BioPharma, Inc
18 years and older
30% or greater
10
11 weeks
OtherActive, not recruiting Location: Europe/UK
A Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic Fibro , protocol number A Randomized, Double Blind, Dose-finding Study of Inhaled Oligosaccharide (OligoG) vs Placebo in Patients With Cystic Fibro NCT03698448
Randomized, double blind, placebo controlled study. The study has two parts: Dose-finding part, followed by longer term follow-up (6 months)
Not specified
Not Specified
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.
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
Mucociliary ClearanceEnrolling Location: Multiple care centres across the US
Cohort 3 of a study to evaluate inhaled QBW276 in adults with CF , protocol number Novartis QBW276 - Cohort 3
This randomized, placebo-controlledstudy is taking place at multiple care centers across the US. This study will look at the safety, effectiveness and tolerability of the inhaled drug QBW276.
40-100%
15
3 months
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
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
20
5 Years