Finding the right dose of posaconazole for children & young people

with Cystic Fibrosis and Aspergillus infection

What is cASPerCF?

cASPerCF is the name of a study that will test an antifungal drug called posaconazole in children and young people with Cystic Fibrosis who have Aspergillus infection.

The study is being led and coordinated by the MRC Centre for Medical Mycology at the University of Exeter, United Kingdom, in collaboration with the Children’s Hospital Bambino Gesù in Rome, Italy, which acts as the sponsor of the study. Other institutes and organisations involved in the investigational team and in the development of the study are the Radboud University Medical Centre, Nijmegen, the Netherlands, the European Cystic Fibrosis Society, and the Consorzio per Valutazioni Biologiche e Farmacologiche (CVBF) in Italy.

Children and young people aged 8-17 years old with Cystic Fibrosis across Europe will be recruited into this study.


What is Aspergillus infection?

Aspergillus is a fungus commonly found in our environment. The tiny Aspergillus spores which are transported by air, can enter the lungs during breathing. Normally, this will not cause any problem, but in people with underlying lung disease, it can lead to infection. Not all Aspergillus infections are the same. Sometimes, Aspergillus can be present in the lungs of people with Cystic Fibrosis and no problems are noticed. However, Aspergillus may cause more inflammation in the lungs and, sometimes this can develop into a problem called allergic bronchopulmonary aspergillosis (ABPA) which can lead to patients becoming wheezy and feeling chest tightness. Other times, people with Cystic Fibrosis can develop Aspergillus bronchitis, which has similar symptoms to other types of chest infections.


How can we tell if somebody has Aspergillus infection?

Aspergillus usually shows up in sputum samples if there is an infection, and there are special blood tests that can also help doctors to know if a child or young person with Cystic Fibrosis has an Aspergillus infection. Those tests measure antibodies against Aspergillus and tell us that the immune cells have noticed Aspergillus in the lungs.


Why is this study needed?

Over half of people with Cystic Fibrosis have Aspergillus in their lungs and sometimes this can cause problems with breathing and lung function to deteriorate. We do not know the best way to manage Aspergillus infections in children and young people, and different Cystic Fibrosis centers treat Aspergillus infection in different ways.


What will the study tell us that is new?

The cASPerCF study will test a medication called Posaconazole. This medicine is already used to treat Aspergillus infection in adults and is also used “off-label” (outside the licensed indication) in children. Several studies have demonstrated that posaconazole is effective, safe and well tolerated in children and young people with blood disorders. However, dosing, safety, and efficacy in children and young people with Cystic Fibrosis have not been extensively investigated. We will study which is the best dose to give children and young people with Cystic Fibrosis and Aspergillus infection. The study will tell us which dose of posaconazole works best to help clear Aspergillus infection, reduce inflammation and improve lung function.


Follow the cASPerCF trial on Twitter @cASPerCFstudy