A Phase 2, Six-Month Safety and Efficacy Study of TransCon hGH Compared to Daily hGH in Pre-Pubertal Children with Growth Hormone Deficiency (GHD)

This study demonstrates the safety and efficacy of TransCon hGH in children with GHD over a treatment period of six months. Safety and efficacy (annualized height velocity), as well as PK and PD data of 53 GHD patients treated over a six-month period with TransCon hGH or daily hGH will be presented. All TransCon hGH doses demonstrated an excellent safety (comparable to daily hGH) and local tolerability profile (only mild and sporadic reactions comparable to daily hGH / no nodule formation and lypodystrophy) and an excellent growth within the expected ranges - mean annualized height velocities ranging between 11.9 cm to 13.9 cm for the different dose levels of TransCon hGH compared to 11.6 cm mean annualized height velocity for daily hGH treatment

Gene expression signatures predict response to therapy with growth hormone.

From PubMed via Jisc Publications RouterHistory: received 2020-05-10, revised 2021-03-17, accepted 2021-04-23Publication status: aheadofprintRecombinant human growth hormone (r-hGH) is used as a therapeutic agent for disorders of growth including growth hormone deficiency (GHD) and Turner syndrome (TS). Treatment is costly and current methods to model response are inexact. GHD (n = 71) and TS patients (n = 43) were recruited to study response to r-hGH over 5 years. Analysis was performed using 1219 genetic markers and baseline (pre-treatment) blood transcriptome. Random forest was used to determine predictive value of transcriptomic data associated with growth response. No genetic marker passed the stringency criteria for prediction. However, we identified an identical set of genes in both GHD and TS whose expression could be used to classify therapeutic response to r-hGH with a high accuracy (AUC > 0.9). Combining transcriptomic markers with clinical phenotype was shown to significantly reduce predictive error. This work could be translated into a single genomic test linked to a prediction algorithm to improve clinical management. Trial registration numbers: NCT00256126 and NCT00699855