{
  "abstract": "In a first-in-human trial (NI006-101), the monoclonal antibody cliramitug, targeting misfolded transthyretin, demonstrated a favorable safety profile and time- and dose-dependent reductions in surrogate markers of cardiac amyloid burden over the course of 12 months in patients with amyloid transthyretin cardiomyopathy (ATTR-CM). Here we evaluate the long-term safety and efficacy of cliramitug in a subgroup of participants of the NI006-101 trial who continued treatment in a second open-label extension (OLE2) and further explore dose- and time-dependent effects on amyloid depletion, cardiac biomarkers and cardiac structure and function. Twenty-three participants (20 receiving background treatment with tafamidis, all male) entered OLE2 and received a median of 10 additional infusions, increasing the maximum total exposure to 24 infusions and extending the median follow-up to 29.3 months. Thirteen participants initially treated with ≤10 mg kg-1 were up-titrated to 30 mg kg-1 during OLE2. Treatment adherence was high (98%), with no treatment-related serious adverse events or discontinuations. Continued treatment and up-titration in participants with lower prior exposure led to further reductions in cardiac extracellular volume on MRI and tracer uptake on bisphosphonate scintigraphy. Improvements were also observed in NT-proBNP and troponin T levels, left ventricular relaxation, filling pressures and wall thickness. Increases in Kansas City Cardiomyopathy Questionnaire scores suggested potential quality-of-life benefits. Consistent with results from the original trial, cliramitug showed favorable long-term safety and further reductions in cardiac amyloid burden following up-titration to 30 mg kg-1. These time- and dose-dependent improvements across structural, functional and biomarker endpoints support the therapeutic potential of amyloid-depleting therapy with cliramitug in ATTR-CM. ClinicalTrials.gov: NCT04360434 .",
  "abstract_shown": true,
  "affiliations": [],
  "article_type": null,
  "arxiv": null,
  "author_total": 17,
  "authors": [
    {
      "name": "Peter C. Kahr"
    },
    {
      "name": "Fabian Aus Dem Siepen"
    },
    {
      "name": "Olivier Lairez"
    },
    {
      "name": "Erwan Donal"
    },
    {
      "name": "Thibaud Damy"
    },
    {
      "name": "Arnt V. Kristen"
    },
    {
      "name": "Cristina C. Quarta"
    },
    {
      "name": "Michele F. Mercuri"
    },
    {
      "name": "Sarah Keppner-Witter"
    },
    {
      "name": "Eva Herrmann-Keiner"
    },
    {
      "name": "Mia Tichy"
    },
    {
      "name": "Aubin Michalon"
    },
    {
      "name": "Jan Grimm"
    },
    {
      "name": "Roger M. Nitsch"
    },
    {
      "name": "Christoph Hock"
    },
    {
      "name": "Pablo Garcia-Pavia"
    },
    {
      "name": "Peter van der Meer"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/9nm2ghav05y0c.bib",
  "cited_by": 1,
  "doi": "10.1038/s41591-026-04487-3",
  "fulltext_url": null,
  "id": "9nm2ghav05y0c",
  "issns": [
    "1078-8956",
    "1744-7933"
  ],
  "landing_url": "https://hal.science/hal-05825700v1",
  "language": "en",
  "licence": "CC-BY-NC-ND-4.0",
  "licence_label": "CC BY-NC-ND 4.0",
  "licence_source": "article",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
    [
      "hal",
      "0.sdv"
    ],
    [
      "hal",
      "1.sdv.ib"
    ],
    [
      "openalex",
      "1312|Amyloidosis: Diagnosis, Treatment, Outcomes"
    ]
  ],
  "openalex": "W7166175789",
  "pdf_url": "https://hal.science/hal-05825700/document",
  "pmcid": null,
  "pmid": "42362866",
  "published": "2026-10-20",
  "publisher": "Nature Publishing Group",
  "ris": "https://research.draconitic.com/paper/9nm2ghav05y0c.ris",
  "sources": [
    {
      "first_seen": "2026-10-30T12:17:59Z",
      "native_id": "hal-05825700",
      "source": "hal",
      "url": "https://hal.science/hal-05825700v1"
    }
  ],
  "subjects": [
    {
      "field": "biology",
      "origin": "hal:1.sdv.ib",
      "subfield": "systems-synthetic"
    }
  ],
  "title": "Cliramitug for depletion of cardiac amyloid transthyretin: long-term follow-up of the NI006-101 trial",
  "title_text": "Cliramitug for depletion of cardiac amyloid transthyretin: long-term follow-up of the NI006-101 trial",
  "type": "peer-reviewed",
  "updated_at": "2026-10-30T12:17:59Z",
  "url": "https://research.draconitic.com/paper/9nm2ghav05y0c",
  "venue": "Nature Medicine",
  "versions": []
}