Student and Faculty Publications
Publication Date
10-1-2023
Journal
Nature Medicine
Abstract
Fibrodysplasia ossificans progressiva (FOP) is a rare disease characterized by heterotopic ossification (HO) in connective tissues and painful flare-ups. In the phase 2 LUMINA-1 trial, adult patients with FOP were randomized to garetosmab, an activin A-blocking antibody (n = 20) or placebo (n = 24) in period 1 (28 weeks), followed by an open-label period 2 (28 weeks; n = 43). The primary end points were safety and for period 1, the activity and size of HO lesions. All patients experienced at least one treatment-emergent adverse event during period 1, notably epistaxis, madarosis and skin abscesses. Five deaths (5 of 44; 11.4%) occurred in the open-label period and, while considered unlikely to be related, causality cannot be ruled out. The primary efficacy end point in period 1 (total lesion activity by PET–CT) was not met (P = 0.0741). As the development of new HO lesions was suppressed in period 1, the primary efficacy end point in period 2 was prospectively changed to the number of new HO lesions versus period 1. No placebo patients crossing over to garetosmab developed new HO lesions (0% in period 2 versus 40.9% in period 1; P = 0.0027). Further investigation of garetosmab in FOP is ongoing.
Keywords
Adult, Humans, Myositis Ossificans, Positron Emission Tomography Computed Tomography, Ossification, Heterotopic, Connective tissue diseases, Musculoskeletal abnormalities, Clinical trials, Drug development, Musculoskeletal system
Included in
Bioinformatics Commons, Biomedical Informatics Commons, Dermatology Commons, Medical Sciences Commons, Musculoskeletal Diseases Commons, Oncology Commons, Skin and Connective Tissue Diseases Commons
Comments
ClinicalTrials.gov identifier NCT03188666.
Supplementary Materials
PMID: 37770652