Once-weekly, investigational GH replacement safe, effective

Biller BM. J Clin Endocrinol Metab. 2011;doi:10.1210/jc.2010-2819.

Weekly growth hormone replacement with an investigational, sustained-release formulation significantly reduced fat mass after 6 months and was well tolerated by adults with growth hormone deficiency, researchers found.

A 6-month study was conducted to evaluate the safety and efficacy of LB03002, a recently developed recombinant human GH formulation. The long-acting GH was developed for once-weekly administration by subcutaneous injection.

The study included 152 adults with GH deficiency who were randomly assigned to LB03002 or placebo once a week for 26 weeks. The researchers assessed changes in body composition, insulin-like growth factor I, adverse events, glucose homeostasis and antibody development.

According to the results, adults assigned to the LB03002 formulation experienced a significant increase in IGF-I (P<.001), whereas those assigned placebo experienced no change.

Mean fat mass decreased by 1 kg in the LB03002 group (95% CI, –1.614 to –0.491) but increased by 0.5 kg in the placebo group (95% CI, –0.205 to 1.345); the treatment difference was 1.622 kg (95% CI, –2.527 to 0.717). The researchers said the change in fat mass with LB03002 was primarily related to decreased trunk fat. LB03002 was also associated with positive effects on lean body mass; it increased significantly in the GH group vs. placebo (least square mean difference, 1.393; 95% CI, 0.614-2.171).

The researchers found “no concerning safety issues” related to LB03002 during the 6-month study.

“This study has provided evidence that GH replacement in adult patients with GH [deficiency] can be successful with the once-weekly sustained-release formulation,” the researchers concluded.

Disclosure: The study was jointly funded by LG Life Sciences Ltd., Korea, and Biopartners GmbH, Switzerland.

GH has to be given daily by subcutaneous injection. Treatment periods are always for several years, both in childhood and in adulthood. Therefore, compliance is often poor, contributing to poor outcome.

LB03002 is a new, sustained-release formulation of GH consisting of microparticles containing GH incorporated into hyaluronate and dispersed in an oil base of medium-chain triglycerides. In a recently published double blind, placebo-controlled study of adults with GH deficiency, an international group of investigators showed comparable responses of subcutaneously injected sustained-release GH given once a week, in terms of body composition. Mean fat mass reduction was mainly due to decreased trunk fat and lean body mass was significantly increased vs. placebo, thereby fulfilling the primary endpoint of the study. Patients showed a 1-kg decrease in fat mass and a 2-kg increase in lean body mass. Lipid parameters were not statistically different. No concerning safety issues arose during the 6-month study.

These data show the clinical efficacy and utility of a sustained-release, once a week GH preparation in adults with GH deficiency.

– Paul Saenger, MD
Endocrine Today Editorial Board member

Disclosure: Dr. Saenger is an advisor for Sandoz, LG Life Sciences, Ltd. and Biopartners GmbH. He is also on the speaker’s bureau for TEVA Pharmaceuticals and Genentech.

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