Finding the Optimal Regimen for Mycobacterium Abscessus Treatment (FORMaT)
Mycobacterium abscessus (MABS) is a group of rapid-growing, multi-drug resistant non-tuberculous mycobacteria (NTM) causing infections in humans. MABS pulmonary disease (MABS-PD) can result in significant morbidity, increased healthcare utilisation, accelerated lung function decline, impaired quality of life, more challenging lung transplantation, and increased mortality. While the overall numbers affected is small, the prevalence of infections is increasing worldwide. The Finding the Optimal Regimen for Mycobacterium abscessus Treatment (FORMaT) trial aims to produce high quality evidence for the best treatment regimens to maximise health outcomes and minimise toxicity and treatment burden, as well as developing biomarkers (serology, gene expression signatures, and radiology) to guide decisions for starting treatment and measuring disease severity in patients with MABS PD.
• Positive MABS-PD diagnosis meeting all three American Thoracic Society clinical, radiological and microbiological diagnostic criteria for MABS-PD. Defined as:
‣ Clinical: Pulmonary symptoms and exclusion of other diagnoses.
⁃ Radiological: Nodular or cavitary opacities on chest radiograph or a chest high-resolution computed tomography (HRCT) scan showing multifocal bronchiectasis with multiple small nodules.
⁃ Microbiological: MABS positive culture results from at least two separate expectorated sputum samples.
• or Positive culture results from at least one bronchial wash or lavage. or Transbronchial or other lung biopsy with mycobacterial histopathologic features (granulomatous inflammation or acid-fast bacilli (AFB)) and positive culture for NTM or biopsy showing mycobacterial histopathologic features (granulomatous inflammation or AFB) and one or more sputum or bronchial washes that are culture positive for NTM.
• Screening samples must be collected within the timeframes stated in the relevant appendix.
• Male or female participants of any age.
• Participant has not received treatment for MABS-PD in the 12 months preceding assessment of eligibility or as specified in the relevant appendix (this includes drugs prescribed for the treatment of other mycobacteria and/or other indications that may have activity against MABS, as specified in the FORMaT Prohibited Drug List Standard Operating Procedure (SOP)).
• Informed consent signed by participant or parent/legal guardian if participant is under 18 years of age.
• Ability to comply with study visits, therapies and study procedures as judged by the site investigator.
∙ To be eligible to participate in the Observational Cohort the following criteria must be met:
• Male and female participants of any age with at least one positive respiratory culture for MABS.
• Informed consent signed by participant or parent/legal guardian if participant is under 18 years of age.
• Ability to comply with study visits and study procedures as judged by the site investigator.
∙ Appendix specific sub-studies and integrated studies Appendix specific sub-studies and integrated studies may have additional eligibility criteria which are described in each of the relevant appendices.