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.
ObservationalEnrolling Location: Multiple care centres across the US
Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part B) , protocol number NCT04509050
This two-part observational study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.
Age:
Less than 6 Years
Mutation(s):
No Mutation Requirement
FEV1% Predicted:
No FEV1 Limit
Number of Visits:
6
Length of Participation:
2 Years
Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN Part A) , protocol number NCT04509050
This two part study will look at the effects of CFTR modulators on growth in young children with CF. These drugs are intended to help CFTR protein function closer to normal.
0 Years to 4 Years
3 Years
OtherRecruiting Location: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation , protocol number NCT02725567
The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have a CF transmembrane conductance regulator (CFTR) gene gating mutation
Maximum 24 months of Age
One copy F508del
Not specified
OtherRecruiting Location: A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in subject 6 through 11 years of Age with cystic fibrosis and F/MF genotypes
A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in subject 6 through 11 years of Age with cystic fibrosis and F/MF genotypes , protocol number NCT04353817
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
6 Years through to 11 Years
Two copies of F508del
Study to evaluate the effects of CFTR modulators in infants and young children (BEGIN) , protocol number BEGIN NCT04509050
This two-part observational study will look at the effects of CFTR modulators on growth in infants and young children with CF. These drugs are intended to help CFTR protein function closer to normal.
0 Years to 6 Years
Mucociliary ClearanceRecruiting Location: New Zealand
Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis , protocol number AROENaC1001 NCT04375514
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of single doses of ARO-ENaC in healthy adult volunteers; and to evaluate the safety, tolerability, PK and efficacy of multiple doses of ARO-ENaC in patients with pulmonary cystic fibrosis.
18 Years to 55 Years
Any
between 40 and 90 percent-predicted
15
4 months
Anti-InflammatoryNot yet recruiting Location: Brisbane
A double blind, randomized, placebo controlled, cross-over trial investigating the effect of High Amylose Maize Starch (HAMS) supplementation on fecal microbiological and inflammatory outcomes in individuals with Cystic Fibrosis and Healthy Volunteers , protocol number ACTRN12618000283279
Cystic Fibrosis (CF) is associated with a significant increase in gut dysbiosis. Exposure to multiple courses of antibiotics as well as an inherently inflammatory gut contributes to this. We have shown that there are significant differences between the CF gut and healthy volunteers in these respects. High amylose maize starch (HAMS) is a pre-biotic food stuff produced in such a way as to encourage the production of beneficial, anti-inflammatory compounds. In laboratory culture, we have shown that HAMS increases the production of these compounds. What is not clear however is whether this laboratory data translates to a clinical benefit. This study will attempt to understand the impact of HAMS supplementation on the bacteria in the gut and the ability of the gut to make anti-inflammatory compounds, in adult patients with CF as well as in healthy control subjects.
18 Years and Older
Nutritional-GIEnrolling Location: Ohio, USA
OPTION 2: Study of AzurRX MS1819 in enteric capsules in adults with cystic fibrosis and exocrine pancreatic insufficiency (AzurRX AZ-CF2002) , protocol number NCT04375878
This study will look at the safety and effectiveness of the drug MS1819 in enteric capsules as a pancreatic enzyme replacement therapy (PERT).
30% or greater
10
8 weeks
OtherEnrolling Location: Multiple care centres across the US
SIMPLIFY: Study to evaluate stopping inhaled hypertonic saline or dornase alfa in people with CF who are taking the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor , protocol number SIMPLIFY-IP-19 NCT04378153
This study will test the effects and safety of stopping inhaled hypertonic saline or dornase alfa (Pulmozyme®) in teens and adults with CF who are also taking the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor (Trikafta®). Trikafta® is intended to help CFTR function closer to normal, resulting in better clearance of mucus from the lungs. Inhaled hypertonic saline and dornase alfa are intended to thin airway surface liquid and improve clearance of mucus from the lungs. They are considered to be relatively burdensome therapies, so this study will look at the impact of stopping them in people who are also taking Trikafta®.
12 Years and Older
60% or greater
4
10 weeks
Anti-InflammatoryActive, not recruiting Location: Montreal, Toronto and Vancouver
Phase 1b study to evaluate CB-280 in adults with cystic fibrosis and chronic Pseudomonas aeruginosa. , protocol number Calithera CX-280-202 NCT04279769
Patients will receive either CB-280 twice daily orally for 14 days or a placebo
18 Years and older
40% and 90%
2 weeks