Juul et al 2023 | Somapacitan in children born small for gestational age: a multi-centre, open-label, controlled phase 2 study | Randomised, multi-centre, open-label, controlled phase 2 study comprising a 26-week main phase and a 4-year extension. | • Young children with GHD from the age of 2.7 to 11.0 years old. | 62 patients (37 for weekly GH, and 25 for daily GH) | participants received weekly Somapacitan 0.16 mg kg/week. N = 12 Somapacitan 0.20 mg kg/week. N = 13 Somapacitan 0.24 mg kg/week. N = 12 | Participants received Daily GH 0.035 mg/kg/day N = 12 GH 0.067 mg/kg/day N = 13 | 4-years |
Sävendahl et al 2022 | Effective GH Replacement With Once-weekly Somapacitan vs Daily GH in Children With GHD: 3-year Results From REAL 3 | A multicenter, randomized, controlled, phase 2 study for 156 weeks | • Young children with GHD from the age of 3 to 12 years old. | 57 patients (43 for weekly GH, and 14 for daily GH) | participants received weekly Somapacitan 0.04 mg kg/week. N = 14 Somapacitan 0.06 mg kg/week. N = 15 Somapacitan 0.16 mg kg/week. N = 14 | Participants received Daily GH 0.034 mg/kg/day N = 14 | 3-years |
Miller et al 2022 | Weekly Somapacitan is Effective and Well Tolerated in Children with GH Deficiency: The Randomized Phase 3 REAL4 Trial | REAL4 is a randomised, multinational, open-labeled, active-controlled parallel group phase 3 trial, comprising a 52-week main trial | • Young children with GHD from the age of 2.5 to 10 years old. | 200 patients (132 for weekly GH, and 68 for daily GH) | participants received weekly Somapacitan 0.16 mg kg/week. N = 132 | Participants received Daily GH 0.034 mg/kg/day N = 68 | 3-years |
Bright et al 2021 | Development of a Predictive Enrichment Marker for the Oral GH Secretagogue LUM-201 in Pediatric Growth Hormone Deficiency | A completed, randomized, placebo-controlled trial, multicentre, for 48 weeks. | • Young children with GHD from the age of 3 to 14 years old. | 66 patients (20 for weekly GH, and 46 for daily GH) | participants received weekly rhGH 0.3 mg/kg/week kg/week. N = 20 | Participants received Daily LUM-201 0.4 mg/kg/d N = 22 LUM-201 0.8 mg/kg/d N = 24 | 3-years |
Thorntonet et al 2021 | Weekly Lonapegsomatropin in Treatment-Naïve Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial | A randomized, open-label, active-controlled, multicenter, 52-week Phase 3 trial | • Young children with GHD from the age of 4 to 11 years old. | 161 patients (105 for weekly GH, and 56 for daily GH) | participants received weekly lonapegsomatropin 0.24 mg/kg/week. N = 105 | Participants received Daily somatropin 0.24 mg/kg/d N = 56 | 13 months |
Qiao et al 2019 | Use of PEGylated Recombinant Human Growth Hormone in Chinese Children with Growth Hormone Deficiency: A 24-Month Follow-Up Study | A single center, prospective, nonrandomized cohort study, China,for104 weeks. | • Young children with GHD from the age of 3 to 13 years old. | 98 patients (49 for weekly GH, and 49 for daily GH) | participants received weekly PEGylated 0.2 mg/kg/week. N = 49 | Participants received Daily rhGH 0.30 mg/kg/d N = 49 | 2 year and 2 months |
Hwang et al 2018 | Once-Weekly Administration of Sustained-Release Growth Hormone in Korean Prepubertal Children with Idiopathic Short Stature: A Randomized, Controlled Phase II Study | This, randomized, open-label, phase II study, at week 26. | • Young children with GHD from the age of 9 and 11 years old. | 42 patients (28 for weekly GH, and 14 for daily GH) | participants received weekly LB03002 0.5 mg/kg/week. N = 14 LB03002 0.7 mg/kg/week. N = 14 | Participants received Daily rhGH 0.37 mg/kg/week N = 14 | 6 months |
Zelinska et al 2017 | Long-Acting C-Terminal Peptide–Modified hGH (MOD-4023): Results of a Safety and Dose-Finding Study in GHD Children | A multicenter, open-label, randomized, controlled phase 2 study, for 48 weeks. | • Young children with GHD from the age of 3 and 11 years old. | 53 patients (42 for weekly GH, and 11 for daily GH) | participants received weekly MOD-4023 0.25 mg kg/week. N = 13 MOD-4023 0.48 mg kg/week. N = 15 MOD-4023 0.66 mg kg/week. N = 14 | Participants received Daily rhGH 0.24 mg/kg/week N = 11 | One a year |
Luo et al 2017 | Long-acting PEGylated recombinant human growth hormone (Jintrolong) for children with growth hormone deficiency: phase II and phase III multicenter, randomized studies | Phase II and III, multicenter, open-label, randomized controlled trials. | • Young children with GHD from the age of 5 to 15 years old. | 97 patients (63 for weekly GH, and 34 for daily GH) | participants received weekly PEG-rhGH 0.1mg/kg/week. N = 32 PEG-rhGH 0.2mg/kg/week. N = 31 | Participants received Daily rhGH 0.25 mg/kg/week N = 34 | 6 months |
Chatelain et al 2017 | A Randomized Phase 2 Study of Long-Acting TransCon GH vs Daily GH in Childhood GH Deficiency | A multicenter, randomized, controlled, open-label for 26 weeks | • Young children with GHD from the age of 3 to 12 years old. | 57 patients (43 for weekly GH, and 14 for daily GH) | participants received weekly TransCon 0.14 mg / kg/week. N = 12 TransCon 0.21 mg kg/week. N = 14 TransCon 0.30 mg kg/week. N = 14 | Participants received Daily Genotropin 0.03mg/kg/day N = 13 | 6 months |
Battelino et al 2017 | Somapacitan, a once-weekly reversible albumin-binding GH derivative, in children with GH deficiency: a randomized dose-escalation trial | Phase 1, randomized, open-label, active-controlled, dose-escalation trial | • Young children with GHD from the age of 6 to 13 years old. | 32 patients (24 for weekly GH, and 8 for daily GH) | participants received weekly somapacitan 0.02 mg / kg/week. N = 6 somapacitan 0.04 mg kg/week. N = 6 somapacitan 0.08 mg kg/week. N = 6 somapacitan 0.16 mg kg/week. N = 6 | Participants received Daily Norditropin 0.03mg/kg/day N = 8 | 3 momths |
Hou et al 2015 | comparative pharmacokinetics and pharmacodynamics of a Pegylated recombinant human growth hormone and daily recombinant human growth hormone in growth hormonedeficient children | single-center, open-label, crossover Phase I trial. | Young children with GHD from the age of 3 to 16 years old. | 24 patients (12 for weekly GH, and 12 for daily GH) | participants received weekly Jintrolong 0.2 mg/kg/week. N = 12 | Participants received Daily rhGH 0.0286 mg/kg/d N = 12 | 3 months |
Khadilkar et al 2014 | 24-Month Use of Once-Weekly GH, LB03002, in Prepubertal Children with GH Deficiency | Phase III, 12-month, multinational, randomized, open-label, comparator-controlled trial, with a 12-month uncontrolled extension. | • Young children with GHD from the age of 3.0–12.4 years old. | 178 patients (91 for weekly GH, and 87 for daily GH) | participants received weekly LB03002 0.5 mg/kg/week. N = 91 | Participants received Daily rhGH 0.03 mg/kg/d N = 87 | One a year |
Hwang et al 2013 | Efficacy and safety of LB03002, a once-weekly sustained-release human GH for 12-month treatment in Korean children with GH deficiency | Phase III, 12-month, multinational, randomized, controlled trial, with a 12-month | • Young children with GHD from the age of 4–14 years old. | 60 patients (30 for weekly GH, and 30 for daily GH) | participants received weekly LB03002 0.5 mg/kg/week. N = 30 | Participants received Daily rhGH 0.21 mg/kg/d N = 30 | One a year |
Peter et al 2012 | Three-Year Efficacy and Safety of LB03002, a Once-Weekly Sustained-Release Growth Hormone (GH) Preparation, in Prepubertal Children with GH Deficiency (GHD) | A multicenter, randomized, controlled, open-label for 26 weeks | • Young children with GHD from the age of 3 to 12 years old. | 51 patients (39 for weekly GH, and 12 for daily GH) | participants received weekly LB03002 0.2 mg / kg/week. N = 13 LB03002 0.5mg kg/week. N = 13 LB03002 0.7mg kg/week. N = 13 | Participants received Daily Genotropin 0.003mg/kg/day N = 12 | |