Characterization of flare-ups and impact of garetosmab in adults with fibrodysplasia ossificans progressiva: a plain language summary of a post hoc analysis of the LUMINA-1 trial

Plain Language SummaryWhat is this summary about?Fibrodysplasia ossificans progressiva, or FOP, is a very rare disease in which new heterotopic bone forms in places where bone is not usually found. This is known as heterotopic ossification, or HO. This summary is an analysis from a study called LUMINA-1 that investigated a medicine called garetosmab in adults with FOP.What were the results?LUMINA-1 was a global study that included 44 adults with FOP. The analysis looked at flare-ups, which are episodes of localized swelling, pain, and/or warmth that occur in soft tissue. In Period 1 of the study, people were given a treatment into a vein; 20 of these participants received garetosmab and 24 received placebo. A placebo is a ‘dummy’ treatment that looks and feels like a real treatment, but has no active ingredients. Placebos are often used in clinical trials to assess the effects of a new drug.This analysis looked at the 24 participants receiving placebo who had never received any garetosmab treatment. Watching how people on a placebo progress is similar to observing how the disease or condition develops naturally. At Week 28, 71% (17/24) of the participants receiving placebo reported at least one new flare-up with 34 new flare-ups reported. After Period 1, all participants continued Periods 2 and 3 when everyone received garetosmab treatment. In Period 2, all participants received garetosmab for 28 weeks in an ‘open-label design’, meaning doctors and participants knew the treatment. Period 3 extended this open-label phase for at least another 20 weeks (up to Week 76). Fewer participants reported flare-ups while on garetosmab (35%) than during placebo (71%).Fewer people that received garetosmab (35%) reported flare-ups compared with people receiving placebo (71%). The results of this study suggest that new heterotopic bone formation does not always happen at the same location as the flare-up, but flare-ups are often a sign that people with FOP may have more active disease.What do the results mean?The analysis added to our understanding of flare-ups in FOP. Most people who did not receive treatment for FOP experienced a flare-up over a 28-week period. Treatment with garetosmab also showed it can help reduce the number, severity, and duration of flare-ups in people with FOP.How to say…Fibrodysplasia ossificans progressiva: fi-bro-dis-PLAY-see-ah os-SIF-eh-cans pro-gres-SEE-vahGaretosmab: ga-re-TOS-mabHeterotopic ossification: he-te-ro-TOP-ic os-if-eh-KAY-shnHeterotopic bone: Bone that forms in soft tissues where bone does not normally exist such as skeletal muscles, tendons, ligaments, and the thin lining of connective tissue.This 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.