{
  "abstract": "Introduction: Systemic sclerosis (SSc) is a rare, heterogeneous, autoimmune disease. It involves a complex interplay of immune dysregulation, vasculopathy and fibrosis. There are no widely approved drugs that target all three underlying mechanisms and modify the overall SSc disease course or prevent overall disease progression. Nerandomilast, a preferential phosphodiesterase 4B inhibitor, has demonstrated antifibrotic, immunomodulatory and vasculoprotective effects in models relevant to SSc. The phase III VERANDA-SSc trial (NCT07497087) will investigate the efficacy and safety of nerandomilast in patients with SSc.\n\nMethods and analysis: This double-blind, randomised, placebo-controlled trial will be conducted at ~235 sites across 45 countries. Approximately 450 patients with SSc will be randomised 1:1 to receive 18 mg nerandomilast two times a day or placebo in addition to standard-of-care therapy. The dose may be reduced to 9 mg two times a day or interrupted based on individual tolerability or if used concomitantly with strong CYP3A inhibitors. Treatment will continue for at least 52 weeks and until the planned number of primary endpoint events is reached.\n\nAdult patients must meet the 2013 American College of Rheumatology/European Alliance of Associations for Rheumatology classification criteria, have disease onset within 7 years and have active disease (key inclusion criteria permit the enrolment of patients with diffuse cutaneous or limited cutaneous SSc). Stable background immunosuppressive or nintedanib therapy is allowed.\n\nThe primary endpoint is a composite of time to the first occurrence of predefined SSc-related clinically meaningful disease progression or complications, or confirmed absolute decline in forced vital capacity % predicted ≥5% (for patients with interstitial lung disease at baseline), or relative increase in modified Rodnan skin score (mRSS) ≥25% together with an absolute increase in mRSS ≥5 points, or all-cause death. Safety, pharmacokinetics, organ manifestations and patient-reported outcomes will also be assessed.\n\nTrial registration number: NCT07497087.",
  "abstract_shown": true,
  "affiliations": [
    "Oslo University Hospital, Oslo, Norway",
    "Department of Rheumatology, University Hospital Zurich, University of Zurich, Zurich, Switzerland",
    "Rheumatology Department, Saint Joseph Hospital, Université Paris Cité, INSERM U1016, Paris, France",
    "NIHR Biomedical Research Centre and Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds, Leeds, UK",
    "UCL Medical School, London, UK",
    "Nippon Medical School, Tokyo, Japan",
    "University of Michigan, Ann Arbor, Michigan, USA",
    "UTHealth Houston, Houston, Texas, USA",
    "University of California, Los Angeles, California, USA",
    "Boehringer Ingelheim Pharmaceuticals, Inc, Ridgefield, Connecticut, USA",
    "Boehringer Ingelheim International GmbH, Ingelheim am Rhein, Germany",
    "Boehringer Ingelheim (Schweiz) GmbH, Basel, Switzerland"
  ],
  "article_type": "research article",
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  "author_total": 14,
  "authors": [
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      "name": "Anna-Maria Hoffmann-Vold",
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      "name": "Oliver Distler",
      "orcid": "0000-0002-0546-8310"
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      "name": "Yannick Allanore"
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      "name": "Francesco Del Galdo"
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      "name": "Christopher P Denton"
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      "name": "Masataka Kuwana"
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      "name": "Dinesh Khanna",
      "orcid": "0000-0003-1412-4453"
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      "name": "Zsuzsanna H McMahan",
      "orcid": "0000-0001-6461-8940"
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    {
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      "name": "Elizabeth R Volkmann"
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      "name": "Hao Li"
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      "name": "Mihaela Dumistracel"
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      "name": "Martina Gahlemann"
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      "name": "Susanne Stowasser"
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    {
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      "name": "Shervin Assassi"
    }
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  "bibtex": "https://research.draconitic.com/paper/4mhw3rtzghzef.bib",
  "cited_by": 0,
  "doi": "10.1136/jsrd-2026-000052",
  "fulltext_url": "https://pmc.ncbi.nlm.nih.gov/articles/PMC13735304/",
  "id": "4mhw3rtzghzef",
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  "language": "en",
  "licence": "CC-BY-NC-4.0",
  "licence_label": "CC BY-NC 4.0",
  "licence_source": "article",
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  "pdf_url": "https://jsrd.bmj.com/content/jsrd/11/2/e000052.full.pdf",
  "pmcid": "PMC13735304",
  "pmid": "42991098",
  "published": "2026-11-02",
  "publisher": "BMJ Publishing Group",
  "ris": "https://research.draconitic.com/paper/4mhw3rtzghzef.ris",
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  ],
  "title": "VERANDA-SSc: study design of a phase III, randomised, placebo-controlled trial evaluating the efficacy and safety of oral nerandomilast treatment in systemic sclerosis",
  "title_text": "VERANDA-SSc: study design of a phase III, randomised, placebo-controlled trial evaluating the efficacy and safety of oral nerandomilast treatment in systemic sclerosis",
  "type": "peer-reviewed",
  "updated_at": "2026-11-09T12:21:00Z",
  "url": "https://research.draconitic.com/paper/4mhw3rtzghzef",
  "venue": "Journal of Scleroderma and Related Disorders",
  "versions": []
}