title: 24-month use of once-weekly GH, LB03002, in prepubertal children with GH deficiency.
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.
  
  
  METHODS
  We conducted a phase III, 12-month, multinational, randomized, open-label, comparator-controlled trial with a 12-month uncontrolled extension.
  
  
  METHODS
  Prepubertal GH treatment-naive GH-deficient children (mean age, 7.8 y) participated in the study.
  
  
  METHODS
  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).
  
  
  METHODS
  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-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.
authors.affiliation: 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.
authors.forename: Vaman
authors.initials: V
authors.lastname: Khadilkar
authors.forename: Klavdia A
authors.initials: KA
authors.lastname: Radjuk
authors.forename: Elena
authors.initials: E
authors.lastname: Bolshova
authors.forename: Rajesh
authors.initials: R
authors.lastname: Khadgawat
authors.forename: Mohamed
authors.initials: M
authors.lastname: El Kholy
authors.forename: Meena
authors.initials: M
authors.lastname: Desai
authors.forename: Valentina
authors.initials: V
authors.lastname: Peterkova
authors.forename: Veronica
authors.initials: V
authors.lastname: Mericq
authors.forename: Jürgen
authors.initials: J
authors.lastname: Kratzsch
authors.forename: E Christine
authors.initials: EC
authors.lastname: Siepl
authors.forename: Dieter
authors.initials: D
authors.lastname: Martin
authors.forename: Prema
authors.initials: P
authors.lastname: Lopez
authors.forename: Hyi-Jeong
authors.initials: HJ
authors.lastname: Ji
authors.forename: Yoon Ju
authors.initials: YJ
authors.lastname: Bae
authors.forename: Jin Hwa
authors.initials: JH
authors.lastname: Lee
authors.forename: Paul H
authors.initials: PH
authors.lastname: Saenger
mesh_terms: D000906:Antibodies / Q000097:blood; D001827:Body Height / Q000187:drug effects; D002648:Child; D002675:Child, Preschool; D003692:Delayed-Action Preparations; D004334:Drug Administration Schedule; D005260:Female; D006130:Growth Disorders / Q000097:blood / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000008:administration & dosage* / Q000009:adverse effects / Q000172:deficiency; D006801:Humans; D008297:Male; D011627:Puberty; D013997:Time Factors
chemical_list: D000906:Antibodies; D003692:Delayed-Action Preparations; D019382:Human Growth Hormone