Assessment of different types of bone imaging in patients with fibrodysplasia ossificans progressiva: a plain language summary from additional analysis of the LUMINA-1 Study

Plain Language SummaryWhat is this summary about?This is a plain language summary of additional results from a clinical research study called LUMINA-1, looking at how well two types of imaging scans worked. LUMINA-1 studied an investigational medicine called garetosmab in adults with fibrodysplasia ossificans progressiva, or FOP. FOP is a very rare genetic disease that is characterized by new bone forming in places where it should not be found. This is known as heterotopic ossification, or HO. These new bone lesions (new heterotopic bone growth) can form in soft tissues such as muscles, tendons, ligaments, and the thin lining of connective tissue, causing stiffness. This new bone should not be removed by surgery because there is a risk that the bone lesions might have formed near nerves and blood vessels or because surgery itself can trigger more bone growth. Therefore, it builds up and accumulates. This build-up can make it difficult and painful for people with FOP to move. Most people with FOP need to use a wheelchair by early adulthood, and some have a higher risk of dying earlier than expected.What were the results?LUMINA-1 was a global study of 44 adults with FOP who received garetosmab or placebo every 4 weeks. Garetosmab is a new drug being investigated for the treatment of FOP. Placebo is a dummy treatment that looks and feels like the test treatment being investigated, but there are no active ingredients in it. The effects of a new drug are often tested in clinical trials versus a placebo. In this study, neither the doctor nor the participant knew which treatment the participant received. This study showed that garetosmab stopped new bone lesions from forming.This plain language summary describes another analysis from the LUMINA-1 study, looking at how well two types of imaging scans worked. The two imaging scans are called 18F-labeled sodium fluoride positron emission tomography (PET) and X-ray computed tomography (CT). These scans are used to find and assess the size of new bone lesions in people with FOP.Researchers thought that using both PET and CT scans might be better than using only CT scans to find and assess new bone lesions, because they believed that using both scans would be able to show small or immature bone lesions that CT-only scans may miss. They also thought that PET scans could show how fast new bone lesions were forming and growing. Hence, they compared the use of PET plus CT scans with the use of CT-only scans at the beginning of the study, 8 weeks after starting treatment, and 28 weeks after starting treatment with either placebo or garetosmab. The researchers found that the PET plus CT scans and CT-only scans gave very similar results for finding and assessing the size of new bone lesions in both the placebo group and the garetosmab group.What do the results mean?Using CT-only scans might be a suitable way of finding and assessing new bone lesions in people with FOP. CT scans are easier to use in clinical practice, are more readily available, and are a simpler procedure for patients to undergo than the combination of PET plus CT scans. These results suggest that, although PET scans can provide important insights in certain clinical and research settings, CT-only scans may be a useful tool in future research investigating how potential new medicines and treatments affect HO in people with FOP.How to say (download PDF and double click sound icon to play sound)…Fibrodysplasia ossificans progressiva: fi-bro-dis-play-see-ah os-sif-eh-cans pro-gres-see-vahGaretosmab: ga-re-tos-mabHeterotopic bone: he-te-ro-TOP-ic boneThis is an abstract of the Plain Language Summary of Publication article.View the full Plain Language Summary PDF of this article to read the full-textLink to original article hereTrial registration: ClinicalTrials.gov identifier: NCT03188666.Keywords: Computed tomographyFibrodysplasia ossificans progressivaGaretosmabHeterotopic ossificationPositron emission tomographyLay summaryPlain language summaryThis article is related to:The effects of garetosmab in people with fibrodysplasia ossificans progressiva (FOP): A plain language summary of the LUMINA-1 study Author contributionsDinko González Trotter: Writing–review & editing, Methodology, Formal analysis, Conceptualization. Jennifer McGinniss: Writing–review & editing, Writing–original draft, Methodology, Formal analysis, Data curation, Conceptualization. Kusha Mohammadi: Writing–review & editing, Methodology, Formal analysis, Conceptualization. Michelle Davis: Writing–review & editing, Methodology, Formal analysis, Conceptualization. Bret J. Musser: Writing–review & editing, Methodology, Formal analysis, Conceptualization. Gary A. Herman: Writing–review & editing, Methodology, Formal analysis, Conceptualization. Scott Mellis: Writing–review & editing, Methodology, Formal analysis, Conceptualization. Aris N. Economides: Writing–review & editing, Methodology, Formal analysis, Conceptualization.Disclosure statementDGT, JM, KM, BJM, GAH, SM, and ANE are employees and stockholders of Regeneron Pharmaceuticals, Inc. MD is a non-paid member of the Rare Bone Disease Alliance Steering Committee. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.Medical writing support under the direction of the authors was provided by Samantha Cockburn of Alpha (a division of Prime, Knutsford, UK) according to Good Publication Practice guidelines (https://www.acpjournals.org/doi/10.7326/M22-1460) and funded by Regeneron Pharmaceuticals, Inc.Patient reviewers on this PLSP have received honorarium from Future Rare Diseases for their review work but have no other relevant financial relationships to disclose.Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.AcknowledgementsThe authors thank the patients and their families for their participation in this study, as well as the healthcare providers and investigators who treated these patients and made this study possible.Additional informationFundingThis manuscript was funded by Regeneron Pharmaceuticals, Inc., which was involved in the study design, data collection and analysis, decision to publish, and preparation of this manuscript.