ECEESPE2025 Rapid Communications Rapid Communications 4: Pituitary, Neuroendocrinology and Puberty Part 1 (6 abstracts)
Efficacy and safety of once-weekly somatrogon in adults with growth hormone deficiency: a phase 3 study
Vera Popovic 1 , Ionela Baciu 2 , Aleksandra Gilis-Januszewska 3 , Kevin CJ Yuen 4,5 , Maria Fleseriu 6 , Christina Wang 7 , Susan M Webb 8 , Felipe Casanueva 9 , Carmen Emanuela Georgescu 10 , Gili Hart 11 , Daria La Torre 12 , Allison Manners 13 , Carrie Taylor 14 , Ahuva Bar-Ilan 11 , Martin Carlsson 15 , John Choe 16 , Martin Bidlingmaier 17 , Christian J. Strasburger 18 & Beverly M.K. Biller 19
1Medical Faculty, University of Belgrade, Belgrade, Serbia; 2C.I. Parhon National Institute of Endocrinology, C. Davila University of Medicine and Pharmacy, Bucharest, Romania; 3Department of Endocrinology, Jagiellonian University, Medical College, Krakow, Poland; 4Department of Neuroendocrinology and Neurosurgery, Swedish Neuroscience Institute, Seattle, United States; 5CURRENT: Departments of Neuroendocrinology and Neurosurgery, Barrow Neurological Institute, University of Arizona College of Medicine and Creighton School of Medicine, Phoenix, United States; 6Pituitary Center, Departments of Medicine and Neurological Surgery, Oregon Health & Science University, Portland, United States; 7Clinical and Translational Science Institute and Division of Endocrinology, Department of Medicine, the Lundquist Institute at Harbor-UCLA Medical Center, Torrance, United States; 8Department of Endocrinology/Medicine, Hospital S Pau & Univ Autonoma Barcelona, Research Center for Pituitary Diseases, Institut de Recerca Sant Pau (IIB-Sant Pau) and CIBERER Unit 747, ISCIII, Barcelona, Spain; 9Santiago de Compostela University (USC), CIBERobn, IDIS, Complejo Hospitalario Universitario de Santiago, Santiago de Compostela, Spain; 10Department of Endocrinology, Iuliu Hatieganu University of Medicine and Pharmacy Cluj-Napoca & Cluj County Emergency Hospital, Cluj-Napoca, Romania; 11OPKO Biologics, Kiryat Gat, Israel; 12Pfizer srl, Rome, Italy; 13OPKO Pharmaceuticals LLC, Miami, United States; 14Pfizer Inc, Collegeville, United States; 15Pfizer Inc, New York, United States; 16OPKO Health Inc, Miami, United States; 17Endocrine Laboratory, Medizinische Klinik und Poliklinik IV, LMU Klinikum, Munich, Germany; 18Department of Medicine for Endocrinology & Metabolism, Charite Universitaetsmedizin, Berlin, Germany; 19Neuroendocrine & Pituitary Tumor Clinical Center, Massachusetts General Hospital, Boston, United States
JOINT356
Objective: To evaluate the efficacy and safety of once-weekly somatrogon, a long-acting recombinant human growth hormone, in adults with growth hormone deficiency (GHD) (NCT01909479). Somatrogon has been approved in many countries for treatment of pediatric GHD.
Methods: This randomized, double-blind, placebo-controlled Phase 3 study enrolled adults with GHD across 52 sites in multiple countries. The study included a 26-week double-blind period (Period 1), a 26-week open-label extension (OLE; Period 2) (Main Study=Periods 1 and 2, lasting up to 60 weeks, including a 2–8-week washout period), and a multi-year long-term OLE (Period 3) to evaluate long-term safety. In Period 1, patients were randomized 2:1 to somatrogon or placebo, with dosing based on gender, age, and estrogen therapy. During Periods 2 and 3, all patients received somatrogon. The primary endpoint was change (baseline–Week 26) in trunk fat mass (FM). Secondary endpoints included changes (baseline–Weeks 26 and 52) in total FM, lean body mass (LBM), percentage change in trunk FM, and trunk FM (baseline–Week 52 only). A post-hoc supplemental efficacy analysis evaluated changes (baseline–Week 26) in percent trunk FM relative to the total trunk mass (FM + LBM), trunk LBM, and appendicular skeletal muscle mass. Safety assessments included adverse events (AEs), vital signs, and laboratory evaluations.
Results: In Period 1, 389 patients were screened, 202 randomized and 198 received treatment (somatrogon:133; placebo:65). Change in trunk FM from baseline to Week 26 (primary endpoint) was not significantly different between somatrogon vs placebo (-0.37 vs 0.03 kg; P=0.0821). Change in LBM from baseline to Week 26 was significantly higher in somatrogon vs placebo (1.35 vs 0.02 kg; P<0.0001). The somatrogon group showed significant improvements in percentage change in trunk FM (baseline-Week 26) and the 3 supplemental endpoints (baseline-Week 26). Mean IGF-1 SDS at baseline was ≤-2.6 in both groups; IGF-1 SDS normalized following initiation of somatrogon in either Period 1 or 2. The incidence of all-causality AEs was similar between patients originally randomized to somatrogon vs placebo (Period 1: 63.9% vs 69.2%; Period 2: 56.4% vs 60.0%; Period 3: 64.9% vs 60.0%); most AEs were mild to moderate in severity. The most frequently reported AEs (somatrogon vs placebo) were injection site pain (9.0% vs 13.8%) in Period 1, and nasopharyngitis in Periods 2 (7.5% vs 6.2%) and 3 (11.7% vs 10.0%).
Conclusions: Once-weekly somatrogon treatment significantly improved most body composition parameters and was well tolerated in adults with GHD.
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