FORMaT is an adaptive, multi-arm, platform clinical trial designed to find out which combination of medications best treat Mycobacterium abscessus (MABS) chest infections. The treatment of MABS chest infections is complicated and the patient is required to take multiple medications which are often poorly tolerated.
The combination of medications used to treat MABS chest infections are also very expensive. However, the evidence for what medication combinations should be used is not well understood. When to start these medications is also not well understood. As such, the FORMaT trial has been designed as an adaptive, multi-arm, platform clinical trial and brings together internationally recognized experts and clinical leaders to solve this treatment challenge.
The FORMaT trial has been developed using an adaptive, platform clinical trial design. The trial platform provides a framework that can be used to continuously evaluate different treatment protocols.
Recruitment commenced on 2nd March 2020 prior to the COVID-19 pandemic. Recruitment was halted due to COVID-19 restrictions and as a result, there were significant delays to trial set up. Normal recruitment resumed when pandemic restrictions were lifted in the first quarter of 2022. Since then, and with inclusion of new sites, recruitment has accelerated, and FORMaT is approaching the first interim analysis milestone. As platform trials continue in an iterative fashion until the answers to research questions are achieved, there is currently no end date.
The FORMaT trial set up phase is now complete, and recruitment to the current trial iteration is progressing well. There are 17 sites open and recruiting in Australia with a further 4 sites to open in Australia in 2025. There are 4 international sites open (in Denmark and Singapore) with a further 22 sites (in Taiwan, Israel and the UK) planned to open in 2025. Seven sites in Belgium are in the trial set up phase and are planned for opening in 2026.
This set up phase has been supported through the funding obtained in grants from the Australia Government (Medical Research Future Fund), charitable donations and the U.S. Cystic Fibrosis Foundation (CFF). The initial round of funding for FORMaT has successfully supported FORMaT reaching the study milestones of set up and recruitment to the first planned interim analysis.
As such, FORMaT is now poised to expand with the addition of new treatment arms with novel therapies for MABS-PD in a clinical trial for the first time.
MABS are a group of rapid-growing, multi-drug resistant non-tuberculous mycobacteria (NTM) that can cause infections in humans, including lung infections. People who have underlying lung conditions such as cystic fibrosis (CF) or bronchiectasis are more likely to get MABS lung infections, however it can also affect people who do not have any pre-existing lung problems. The number of people who get MABS lung infections is small, but these infections are becoming more common, potentially due to climate change and management of potable water.
Some MABS lung infections can resolve without needing any treatment whereas other MABS lung infections can result in MABS lung disease. MABS lung disease is a chronic lung condition caused by the MABS infection, and it can significantly impact on the person’s quality of life. Currently, there are no guidelines for when to start treating MABS lung infections. The treatment regimens currently used can be administered to the patient for up to two years and are often associated with significant side-effects. The long treatment regimen does not always result in the MABS lung infection being cleared from the lungs. These challenges demonstrate that there is an urgent need to find effective and well tolerated treatment regimens to rid the lungs of MABS infection.