Objective: Growth hormone (GH) replacement therapy currently requires daily injections. This may cause poor compliance, inconvenience and distress for patients. CTP-modified hGH (MOD-4023) has been developed for once-weekly administration in growth hormone deficient (GHD) adults and children. The 18 and 24 month efficacy of once-weekly subcutaneous (SC) administration of MOD-4023 was evaluated in a Phase 2 study in children with GH deficiency.
Design and methods: A one year, randomized, controlled Phase 2 study was conducted in 53 pre-pubertal children with GHD receiving once-weekly SC injections of one of three MOD-4023 doses (0.25, 0.48, and 0.66 mg/kg per week) vs daily hGH (34 μg/kg/day). Forty-six subjects were rolled over to continue with the same MOD-4023 dose in an open-label extension (OLE), which will routinely assess growth until marketing approval. Height velocity (HV) results during the 2nd year of MOD-4023 treatment for 45 patients were compared to historical controls (1). IGF-1 and IGFBP-3 were monitored as well.
Results: The analysis included 2nd year height velocity data for 45 patients. All three doses of MOD-4023 demonstrated promising 2nd year growth, while the two higher doses of MOD-4023 resulted in better growth in comparison to the lower dose of MOD-4023 (0.25 mg/kg per week), and in line with reported age- and GHD severity-matched historical control (1).
Conclusions: The efficacy of single weekly administration of MOD-4023 for the treatment of pediatric GHD patients was further confirmed during the 2nd year of treatment as part of the OLE of a Phase 2 study. This further affirms that a single weekly injection of MOD-4023 has the potential to replace daily hGH injections in children with GHD and provides additional efficacy data to support dose selection for OPKOs upcoming Phase 3 trial.