Effects of Somatropin (Recombinant Human Growth Hormone) on Body Composition and Bone Mineral Density in Adult Patients with Growth Hormone Deficiency: Interim Analysis of an Open-Label, Phase 3b, Multicenter Trial

Presentation Number: SUN 460
Date of Presentation: April 2nd, 2017

Mohita Kumar*1, Bradley Scott Miller2, Alberto M. Pereira3 and Nitin Agarwal4
1Ferring Pharmaceuticals Inc., Parsipanny, NJ, 2University of Minnesota Masonic Children's Hospital, Minneapolis, MN, 3Leiden University Medical Center, Leiden, Netherlands, 4Minnesota Epilepsy Group, P.A.; Children’s Minnesota, St. Paul, MN

Abstract

Growth hormone deficiency (GHD) in adults is associated with abnormal body composition and reduced bone mineral density (BMD). Somatropin is approved for the treatment of pediatric GHD in the US. This phase 3b, open-label, multicenter, observational, noncomparative study, with planned follow-up of 5 years, evaluated long-term efficacy and safety of somatropin in adults with GHD. This interim analysis included data from treatment up to 24 months to assess the effect of somatropin on body composition and BMD. Patients aged ≥18 years with GHD confirmed by insulin-like growth factor 1 (IGF-1; ≤2 standard deviations [SD] of normal range for age/gender) or blood glucose (<2 mmol [36.0 mg/dL] on insulin tolerance test) and peak GH concentration (<9 mE/L on insulin tolerance, arginine, clonidine, or GHRH test) were enrolled and stratified by treatment experience. Somatropin was administered daily using the Zoma-Jet™ needle-free injector. New patients (no somatropin treatment in previous 12 mo) were initiated on somatropin 0.17 mg/day, and this dose was titrated to the minimum dose required to achieve an IGF-1 level within normal range; switch patients (previously treated) continued their current somatropin dose. Endpoints included changes from baseline in IGF-1 SD score (SDS; primary endpoint), percent body fat, lean body mass, and BMD. Of 98 patients enrolled, 42 were new (mean age: 46.1 y; 54.8% male) and 56 were switch (mean age: 46.3 y; 50.0% male) patients; 89 (new: n=41; switch: n=48) patients who received treatment and had no major protocol violations were included in efficacy analyses. Somatropin significantly increased IGF-1 levels vs. baseline in new patients at all time points (P≤0.0007); over 24 months, IGF-1 increased from −1.85 to 0.47 SDS (change: 2.35 SDS; P<0.0001). IGF-1 SDS was above reference value in switch patients at baseline (+0.69) and not significantly increased at any timepoint (change at 24 mo: −0.12 SDS; P=NS). At 24 months, percent body fat decreased significantly vs. baseline in new (20.08% vs. 29.25%; −42.33%; P=0.0023) and switch patients (24.87% vs. 27.95%; −20.19%; P=0.0007) and mean lean body mass increased significantly vs. baseline in new (61.28 vs. 53.94 kg; +21.80%; P=0.0006) and switch patients (58.56 vs. 54.28 kg; +14.37%; P<0.0001). No significant changes were seen in BMD over 24 months in new or switch patients. Somatropin significantly improved body composition endpoints over 24 months in patients with adult GHD.

 

Disclosure: MK: Employee, Ferring Pharmaceuticals. BSM: Consultant, Ferring Pharmaceuticals. Nothing to Disclose: AMP, NA