ESPE2024 Poster Category 2 Late Breaking (107 abstracts)
Up to 5 years of once-weekly somatrogon treatment in pediatric patients with growth hormone deficiency: results from an open-label extension of a global Phase 3 study
Lawrence Silverman 1 , Joel Steelman 2 , John Choe 3 , Renata Stawerska 4,5 , Cheri Deal 6 , Michael Wajnrajch 7,8 , Marc Thomas 9 , Daria La Torre 10 , Moshe Phillip 11 , Carrie Taylor 7 , Rong Wang 12 & Jose Cara 7
1Goryeb Children’s Hospital, Atlantic Health System, Morristown, USA. 2Cook Children’s Medical Center, Fort Worth, USA. 3OPKO Health Inc, Miami, USA. 4Department of Endocrinology and Metabolic Diseases, Polish Mother’s Memorial Hospital - Research Institute, Lodz, Poland. 5Department of Paediatric Endocrinology, Medical University of Lodz, Lodz, Poland. 6Centre de recherché CHU Ste-Justine, Université de Montréal, Montréal, Canada. 7Pfizer Inc, New York, USA. 8New York University, Grossman School of Medicine, New York, USA. 9Pfizer Ltd, Tadworth, United Kingdom. 10Pfizer SRL, Rome, Italy. 11Schneider Children’s Medical Center of Israel; Sackler Faculty of Medicine, Tel-Aviv University, Petah Tikva, Israel. 12Pfizer Inc, Groton, USA
Objective: Somatrogon, a long-acting recombinant human growth hormone (rhGH) is approved as a once-weekly treatment for pediatric growth hormone deficiency (pGHD). This abstract describes the long-term efficacy and safety of somatrogon in patients with pGHD following up to 5 years of treatment in a global, Phase 3 study (NCT02968004).
Methods: In the main study period, patients were randomized to receive either once-weekly somatrogon (0.66 mg/kg/week) or once-daily somatropin (0.24 mg/kg/week) for 12 months. After the main study period, patients were eligible to enter an open-label extension (OLE), during which somatrogon-treated patients continued to receive somatrogon and somatropin-treated patients switched to somatrogon (0.66 mg/kg/week). Data through May 2024 are presented; analyses of these data are descriptive, with no formal hypothesis testing performed.
Results: Of the 222 patients (somatrogo n = 108, somatropi n = 114) who completed the 12-month main study, 212 (somatrogo n = 104; somatropi n = 108) entered the OLE and received somatrogon. At the end of the main study, somatrogon- and somatropin-treated patients had similar mean height velocity (HV) (somatrogon: 10.18 [SD: 2.42] cm/year; somatropin: 9.68 [SD: 2.46] cm/year) and gain in height SDS (0.93 vs 0.84). At the end of OLE Y1, Y2, Y3, and Y4, mean (SD) annualized HV was 8.13 (1.84), 7.88 (1.82), 7.03 (1.75) and 6.63 (1.80) cm/year, respectively. Mean height SDS continued to increase during the OLE, with values of -1.41 (0.90), -0.95 (0.84), -0.72 (0.89), and -0.43 (0.85) observed at the end of OLE Y1, Y2, Y3, and Y4, respectively. The cumulative mean change in height SDS (from baseline) was 1.94 at the end of OLE Y4. Mean (SD) IGF-1 SDS at the end of the main study period was 0.65 (1.32) and -0.69 (1.09) in somatrogon- and somatropin-treated patients respectively; in the OLE, mean (SD) IGF-1 SDS was 1.19 (1.23), 1.27 (1.22), 1.36 (1.18), and 1.45 (1.13) at the end of OLE Y1, Y2, Y3, and Y4, respectively. In the main study, adverse events (AEs) were reported in 86.2% and 84.3% of somatrogon- and somatropin-treated patients, respectively. During OLE Y1, Y2, Y3, and Y4, AEs were reported in 72.2%, 69.4%, 71.8%, and 56.2% of patients, respectively; most AEs were mild to moderate in intensity.
Conclusion: Following up to 5 years of somatrogon treatment, children with pGHD continued to demonstrate catch up growth, with a mean increase in height SDS (from baseline) of 1.94 observed in OLE Y4.
Clinicaltrials.gov: NCT02968004
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