Vaman Khadilkar1, Klavdia A Radjuk, Elena Bolshova, Rajesh Khadgawat, Mohamed El Kholy, Meena Desai, Valentina Peterkova, Veronica Mericq, Jürgen Kratzsch, E Christine Siepl, Dieter Martin, Prema Lopez, Hyi-Jeong Ji, Yoon Ju Bae, Jin Hwa Lee, Paul H Saenger. 1. Department of Paediatric Endocrinology (V.K.), Jehangir Hospital, Pune 411004, India; Second Children's Hospital (K.A.R.), Minsk 220020, Belarus; Institute of Endocrinology and Metabolism (E.B.), Academy of Medical Sciences, Kiev 04114, Ukraine; Department of Endocrinology (R.K.), All India Institute of Medial Sciences, New Delhi 110029, India; Department of Pediatrics (M.E.K.), Ain Shams University, Cairo 12311, Egypt; Sir Hurkisondas Nurrotumdas Hospital and Research Centre (M.D.), Mumbai 400004, India; Endocrinological Scientific Center (V.P.), Russian Academy of Medical Sciences, Moscow 117036, Russia; Institute of Maternal and Child Research (V.M.), Santiago de Chile 8389100, Chile; Institute for Laboratory Medicine (J.K.), Leipzig 04103, Germany; Biopartners GmbH (E.C.S., D.M., P.L.), Baar 6340, Switzerland; LG Life Sciences Ltd (H.-J.J., Y.J.B., J.H.L.), Seoul 110-783, South Korea; and Department of Pediatrics (P.H.S.), Winthrop University Hospital, Mineola, New York 11501.
Abstract
BACKGROUND: Sustained-release GH formulations may provide a strategy for improving treatment compliance and persistence in GH-deficient patients. OBJECTIVE: The aim of the study was to examine efficacy and safety of LB03002, a sustained-release GH formulation for once-weekly administration. DESIGN: We conducted a phase III, 12-month, multinational, randomized, open-label, comparator-controlled trial with a 12-month uncontrolled extension. PATIENTS: Prepubertal GH treatment-naive GH-deficient children (mean age, 7.8 y) participated in the study. INTERVENTION: We administered once-weekly LB03002 (n=91) or daily GH (n=87) for 1 year, followed by once-weekly LB03002 for all patients for another year (LB03002 throughout, n=87; switched to LB03002, n=80). OUTCOME MEASURES: Height, height velocity (HV), IGF-1, GH antibodies, andadverse events were determined throughout. Primary analysis was noninferiority of LB03002 vs daily GH at 1 year by analysis of covariance. RESULTS: Mean±SD HV during year 1 was 11.63±2.60 cm/y with LB03002, and 11.97±3.09 cm/y with daily GH, with increases from baseline of 8.94±2.91 and 9.04±3.19 cm/y, respectively. The least square mean HV difference for LB03002 - daily GH was -0.43 cm/y (99% confidence interval, -1.45 to 0.60 cm/y). Mean HV also remained above baseline in year 2 (8.33±1.92 cm/y in the LB03002 throughout group, and 7.28±2.34 cm/y in the switched to LB03002 group). Injection site reactions occurred more frequently in LB03002-treated patients but were considered mild to moderate in >90% of cases. CONCLUSIONS:Growth response with once-weekly LB03002 in GH-deficient children is comparable to that with daily GH, achieving expected growth rates for 24 months. Once-weekly LB03002 is a strong candidate for long-term GH replacement in GH-deficient children.
RCT Entities:
BACKGROUND: Sustained-release GH formulations may provide a strategy for improving treatment compliance and persistence in GH-deficientpatients. OBJECTIVE: The aim of the study was to examine efficacy and safety of LB03002, a sustained-release GH formulation for once-weekly administration. DESIGN: We conducted a phase III, 12-month, multinational, randomized, open-label, comparator-controlled trial with a 12-month uncontrolled extension. PATIENTS: Prepubertal GH treatment-naive GH-deficientchildren (mean age, 7.8 y) participated in the study. INTERVENTION: We administered once-weekly LB03002 (n=91) or daily GH (n=87) for 1 year, followed by once-weekly LB03002 for all patients for another year (LB03002 throughout, n=87; switched to LB03002, n=80). OUTCOME MEASURES: Height, height velocity (HV), IGF-1, GH antibodies, and adverse events were determined throughout. Primary analysis was noninferiority of LB03002 vs daily GH at 1 year by analysis of covariance. RESULTS: Mean±SD HV during year 1 was 11.63±2.60 cm/y with LB03002, and 11.97±3.09 cm/y with daily GH, with increases from baseline of 8.94±2.91 and 9.04±3.19 cm/y, respectively. The least square mean HV difference for LB03002 - daily GH was -0.43 cm/y (99% confidence interval, -1.45 to 0.60 cm/y). Mean HV also remained above baseline in year 2 (8.33±1.92 cm/y in the LB03002 throughout group, and 7.28±2.34 cm/y in the switched to LB03002 group). Injection site reactions occurred more frequently in LB03002-treated patients but were considered mild to moderate in >90% of cases. CONCLUSIONS: Growth response with once-weekly LB03002 in GH-deficientchildren is comparable to that with daily GH, achieving expected growth rates for 24 months. Once-weekly LB03002 is a strong candidate for long-term GH replacement in GH-deficientchildren.
Authors: Aristides K Maniatis; Samuel J Casella; Ulhas M Nadgir; Paul L Hofman; Paul Saenger; Elena D Chertock; Elena M Aghajanova; Maria Korpal-Szczyrska; Elpis Vlachopapadopoulou; Oleg Malievskiy; Tetyana Chaychenko; Marco Cappa; Wenjie Song; Meng Mao; Per Holse Mygind; Alden R Smith; Steven D Chessler; Allison S Komirenko; Michael Beckert; Aimee D Shu; Paul S Thornton Journal: J Clin Endocrinol Metab Date: 2022-06-16 Impact factor: 6.134
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Authors: Sochung Chung; Jae-Ho Yoo; Jin Ho Choi; Young-Jun Rhie; Hyun-Wook Chae; Jae Hyun Kim; Il Tae Hwang; Choong Ho Shin; Eun Young Kim; Kee-Hyoung Lee Journal: Ann Pediatr Endocrinol Metab Date: 2018-03-22