the type of treatment or therapy being studied. A therapy could range from a medication addressing a particular characteristic of CF to a device or activity e.g. exercise
the different stages involved in the development of a new medication. Phase I focuses on initial safety in people. Phase 2 evaluates safety, correct dose and early signs of whether the medication works. Phase 3 is the stage before medication licensing and looks at safety and medication effectiveness. Phase 4 evaluates longer term use of a medication after it has been licensed for use
An open-label, pilot study to assess safety, tolerability, pharmacokinetics and effects of inhaled PC945 in the pre-emptive treatment of Aspergillus fumigatus colonisation in lung transplant recipients
The safety, tolerability and effects of taking inhaled PC945 (study drug) for early (‘pre-emptive’) treatment of airway infections by a fungus called Aspergillus will be studied in patients who have received a lung transplant. The amount of study drug that gets into the bloodstream and lungs will also be studied. As the study is open-label, participants will know what treatment they receive.
There are two study phases:
1. Surveillance Phase (first 12-weeks post-transplant): participants will receive preventative antifungal treatment, attend hospital visits and have bronchoscopies according to local Standard of Care (SOC). Participants who do not develop a fungal infection will participate in the non-intervention Surveillance Phase only.
2. Treatment Phase (16-weeks including treatment and follow-up): Participants who develop a fungal lung infection will receive either:
- Pre-emptive PC945 treatment for 28 days (Pre-Emptive Treatment phase) if diagnosed with an early Aspergillus infection without symptoms/disease, with an optional further 8 weeks of PC945 (Extended Treatment Phase), if required,
- 'Non-intervention' group: SOC antifungal treatment and management, if the Aspergillus infection is causing symptoms/disease.
Approximately 30 participants will be recruited into the Surveillance Phase from UK sites to identify 10 participants who are eligible for PC945 treatment.
All participants will have at least 2 study visits in the Surveillance Phase. Participants in the PC945 Pre-emptive Treatment Phase will have 5 study visits and will be contacted by telephone on up to 3 occasions. Participants in the 8-week Extended Treatment Phase will have 2 additional visits. Study visits and bronchoscopies will be timed to coincide with usual SOC practice where possible; participants may have additional bronchoscopy visits depending on local SOC.
Trial Reference Number
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the name of the treatment or therapy being researched
PC945 5mg once daily, PC945, nebulized
the number of participants who need to be recruited for the trial in the UK
Last edited date
26 November 2018
CF sponsor type