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Garetosmab in fibrodysplasia ossificans progressiva: a randomized, double-blind, placebo-controlled phase 2 trial. 2023
Maja Di Rocco, and
Eduardo Forleo-Neto, and
Robert J Pignolo, and
Richard Keen, and
Philippe Orcel, and
Thomas Funck-Brentano, and
Christian Roux, and
Sami Kolta, and
Annalisa Madeo, and
Judith S Bubbear, and
Jacek Tabarkiewicz, and
Małgorzata Szczepanek, and
Javier Bachiller-Corral, and
Angela M Cheung, and
Kathryn M Dahir, and
Esmée Botman, and
Pieter G Raijmakers, and
Mona Al Mukaddam, and
Lianne Tile, and
Cynthia Portal-Celhay, and
Neena Sarkar, and
Peijie Hou, and
Bret J Musser, and
Anita Boyapati, and
Kusha Mohammadi, and
Scott J Mellis, and
Andrew J Rankin, and
Aris N Economides, and
Dinko Gonzalez Trotter, and
Gary A Herman, and
Sarah J O'Meara, and
Richard DelGizzi, and
David M Weinreich, and
George D Yancopoulos, and
E Marelise W Eekhoff, and
Frederick S Kaplan
Department of Pediatrics, Unit of Rare Diseases, IRCCS Istituto Giannina Gaslini, Genoa, Italy.
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. ClinicalTrials.gov identifier NCT03188666 .
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