{
  "abstract": "Introduction Mycobacteroides abscessus (MABS) is within the non-tuberculous mycobacteria family. It inhabits soil and water, exhibits multi-antibiotic resistance and causes opportunistic lung infections, which may progress to symptomatic MABS-pulmonary disease (MABS-PD) associated with substantial morbidity, increased healthcare utilisation, impaired quality of life and increased mortality. Treatment regimens for MABS-PD are highly variable, not evidence-based and involve complex, expensive drug combinations administered for prolonged periods (>12 months) with frequent adverse effects and treatment failure. There is an urgent need for safe, efficacious and cost-effective MABS-PD therapy. Here, we describe the Master Protocol for the Finding the Optimal Regimen for Mycobacteroides abscessus Treatment (FORMaT) trial. FORMaT aims to determine the most effective and best tolerated treatment for MABS-PD as defined by MABS clearance from respiratory samples with good treatment tolerance.Methods and analysis FOR MaT is an international multicentre, adaptive platform trial evaluating treatment combinations for MABS-PD. Participants are randomised multiple times during the trial, with assessment of the primary outcome of clearance of MABS infection with good treatment tolerance. Initially, therapies recommended in international consensus guidelines are being tested. Data obtained will eliminate therapies lacking efficacy or causing unacceptable toxicity. Novel treatments can then be added and tested against previously determined optimal approaches, leading in an iterative fashion to improved microbiological clearance and health outcomes. In parallel, an Observational cohort and several integrated and discovery studies are embedded in FORMaT to identify biomarkers of MABS-PD and MABS clearance, clinical and radiographic treatment response, drug pharmacokinetics and Mycobacteroides genomics and resistome.Ethics and dissemination The FOR MaT Master Protocol and related documents are approved by regulatory authorities in each participating jurisdiction and/or site. Results will be published in peer-reviewed journals and presented at scientific meetings. De-identified, aggregated data will be shared on an approved online platform.Trial registration numbers NCT04310930, ANZCTR12618001831279, 2020-000050-10, ISRCTN67303903.",
  "authors": [
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Centre for Children’s Health Research, Brisbane, Queensland, Australia"
      ],
      "name": "Tiffany Jong"
    },
    {
      "affiliations": [
        "Sunshine Coast University Hospital, Sunshine Coast, Queensland, Australia",
        "Sunshine Coast Health Institute, Birtinya, Queensland, Australia",
        "Centre for Bioinnovation, University of the Sunshine Coast, Sippy Downs, Queensland, Australia"
      ],
      "name": "Timothy Baird"
    },
    {
      "affiliations": [
        "Nottingham Respiratory Biomedical Research Centre, University of Nottingham, Nottingham, UK"
      ],
      "name": "Helen Louise Barr"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "The Prince Charles Hospital Department of Thoracic Medicine, Chermside, Queensland, Australia"
      ],
      "name": "Scott Bell"
    },
    {
      "affiliations": [
        "Newcastle University Faculty of Medical Sciences, Newcastle upon Tyne, UK"
      ],
      "name": "Theophile Bigirumurame"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Centre for Children’s Health Research, Brisbane, Queensland, Australia"
      ],
      "name": "Kara Brady"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "The Prince Charles Hospital Department of Thoracic Medicine, Chermside, Queensland, Australia"
      ],
      "name": "Andrew Burke"
    },
    {
      "affiliations": [
        "Griffith University Centre for Applied Health Economics, Southport, Queensland, Australia"
      ],
      "name": "Joshua Byrnes"
    },
    {
      "affiliations": [
        "Department of Pediatrics, Division of Respiratory Medicine and Allergy, Erasmus MC University Medical Center Rotterdam, Rotterdam, The Netherlands",
        "Department of Radiology and Nuclear Medicine, Erasmus MC University Medical Center Rotterdam, Rotterdam, The Netherlands"
      ],
      "name": "Daan Caudri"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Queensland Children’s Hospital, Brisbane, Queensland, Australia"
      ],
      "name": "Julia E Clark"
    },
    {
      "affiliations": [
        "The University of Melbourne Department of Microbiology and Immunology, Parkville, Victoria, Australia"
      ],
      "name": "Lachlan J M Coin"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "The University of Queensland Greenslopes Clinical Unit, Greenslopes, Queensland, Australia"
      ],
      "name": "Felicia Goh"
    },
    {
      "affiliations": [
        "Menzies Health Institute Queensland, Griffith University School of Medicine and Dentistry, Gold Coast, Queensland, Australia"
      ],
      "name": "Keith Grimwood"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Centre for Children’s Health Research, Brisbane, Queensland, Australia"
      ],
      "name": "Daniel Hicks"
    },
    {
      "affiliations": [
        "Murdoch Children’s Research Institute Clinical Epidemiology and Biostatistics Unit, Parkville, Victoria, Australia"
      ],
      "name": "Kaushala Jayawardana"
    },
    {
      "affiliations": [
        "Murdoch Children’s Research Institute Clinical Epidemiology and Biostatistics Unit, Parkville, Victoria, Australia"
      ],
      "name": "Sri Joshi"
    },
    {
      "affiliations": [
        "Murdoch Children’s Research Institute Clinical Epidemiology and Biostatistics Unit, Parkville, Victoria, Australia",
        "The University of Melbourne Department of Paediatrics, Parkville, Victoria, Australia"
      ],
      "name": "Katherine Lee"
    },
    {
      "affiliations": [
        "Rigshospitalet, Kobenhavn, Denmark"
      ],
      "name": "Tavs Qvist"
    },
    {
      "affiliations": [
        "The Prince Charles Hospital Department of Thoracic Medicine, Chermside, Queensland, Australia",
        "QIMR Berghofer Medical Research Institute, Herston, Queensland, Australia"
      ],
      "name": "David Reid"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Centre for Children’s Health Research, Brisbane, Queensland, Australia"
      ],
      "name": "Megan Rice"
    },
    {
      "affiliations": [
        "The University of Queensland Centre for Clinical Research, Herston, Queensland, Australia",
        "Herston Infectious Diseases Institute (HeIDI), Metro North Hospital and Health Service, Herston, Queensland, Australia",
        "Departments of Pharmacy and Intensive Care Medicine, Royal Brisbane and Women’s Hospital, Herston, Queensland, Australia",
        "Division of Anesthesia Critical Care and Emergency and Pain Medicine, University Hospital Centre Nimes, Nimes, France"
      ],
      "name": "Jason A Roberts"
    },
    {
      "affiliations": [
        "Flinders University, Adelaide, South Australia, Australia",
        "South Australian Health and Medical Research Institute Limited, Adelaide, South Australia, Australia"
      ],
      "name": "Geraint Rogers"
    },
    {
      "affiliations": [
        "Wal-Yan Respiratory Research Centre, The Kids Research Institute Australia, Nedlands, Perth, Australia"
      ],
      "name": "Claire Shackleton"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Queensland Children’s Hospital, Brisbane, Queensland, Australia"
      ],
      "name": "Peter D Sly"
    },
    {
      "affiliations": [
        "Queen’s University Belfast School of Medicine Dentistry and Biomedical Sciences, Belfast, UK"
      ],
      "name": "Alan R Smyth"
    },
    {
      "affiliations": [
        "Murdoch Children’s Research Institute Clinical Epidemiology and Biostatistics Unit, Parkville, Victoria, Australia"
      ],
      "name": "Luke Stevens"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Centre for Children’s Health Research, Brisbane, Queensland, Australia"
      ],
      "name": "Rebecca Stockwell"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia"
      ],
      "name": "Abdullah Tarique"
    },
    {
      "affiliations": [
        "Infection and Immunity, South Australian Health and Medical Research Institute Limited, Adelaide, South Australia, Australia"
      ],
      "name": "Steven Taylor"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "The Prince Charles Hospital Department of Thoracic Medicine, Chermside, Queensland, Australia",
        "The University of Queensland Greenslopes Clinical Unit, Greenslopes, Queensland, Australia"
      ],
      "name": "Rachel Thomson"
    },
    {
      "affiliations": [
        "Department of Pediatrics, Division of Respiratory Medicine and Allergy, Erasmus MC University Medical Center Rotterdam, Rotterdam, The Netherlands",
        "Department of Radiology and Nuclear Medicine, Erasmus MC University Medical Center Rotterdam, Rotterdam, The Netherlands",
        "Thirona BV, Nijmegen, The Netherlands"
      ],
      "name": "Harm A W M Tiddens"
    },
    {
      "affiliations": [
        "Murdoch Children’s Research Institute Clinical Epidemiology and Biostatistics Unit, Parkville, Victoria, Australia"
      ],
      "name": "Xiao Fang Wang"
    },
    {
      "affiliations": [
        "Population Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK",
        "MRC Biostatistics Unit, University of Cambridge, Cambridge, UK"
      ],
      "name": "James Wason"
    },
    {
      "affiliations": [
        "The University of Queensland, Brisbane, Queensland, Australia",
        "Queensland Children’s Hospital, Brisbane, Queensland, Australia"
      ],
      "name": "Claire Wainwright"
    }
  ],
  "title": "Finding the optimal regimen for Mycobacteroides abscessus treatment (FORMaT) in people with Mycobacteroides abscessus pulmonary disease: a multicentre, randomised, multi-arm, adaptive platform trial",
  "uid": "63830ea0-2091-50a7-8be5-8686c41fdd51"
}