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Rare Endocrine Disorders

Author:

Battelino T, Højby Rasmussen M, De Schepper J, Zuckerman-Levin N, Gucev Z, Sävendahl L on behalf of the NN8640-4042 Study Group

 

Citation:

Battelino T, Rasmussen MH, De Schepper J, et al. Somapacitan, a once-weekly reversible albumin-binding GH derivative, in children with GH deficiency: A randomized dose-escalation trial. Clin Endocrinol (Oxf). 2017;87(4):350-358.

 

DOI:

https://doi.org/10.1111/cen.13409

 

Date of Publication:

4th October 2017

 

Abstract:

Objective: To evaluate the safety, local tolerability, pharmacodynamics and pharmacokinetics of escalating single doses of once-weekly somapacitan, a reversible, albumin-binding GH derivative, vs once-daily GH in children with GH deficiency (GHD).

 

Design: Phase 1, randomized, open-label, active-controlled, dose-escalation trial (NCT01973244).

 

Patients: Thirty-two prepubertal GH-treated children with GHD were sequentially randomized 3:1 within each of four cohorts to a single dose of somapacitan (0.02, 0.04, 0.08 and 0.16 mg/kg; n=6 each), or once-daily Norditropin® SimpleXx® (0.03 mg/kg; n=2 each) for 7 days.

 

Measurements: Pharmacokinetic and pharmacodynamic profiles were assessed.

 

Results: Adverse events were all mild, and there were no apparent treatment-dependent patterns in type or frequency. Four mild transient injection site reactions were reported in three of 24 children treated with somapacitan. No antisomapacitan/anti-human growth hormone (hGH) antibodies were detected. Mean serum concentrations of somapacitan increased in a dose-dependent but nonlinear manner: maximum concentration ranged from 21.8 ng/mL (0.02 mg/kg dose) to 458.4 ng/mL (0.16 mg/kg dose). IGF-I and IGFBP-3, and change from baseline in IGF-I standard deviation score (SDS) and IGFBP-3 SDS, increased dose dependently; greatest changes in SDS values were seen for 0.16 mg/kg. IGF-I SDS values were between −2 and +2 SDS, except for peak IGF-I SDS with 0.08 mg/kg somapacitan. Postdosing, IGF-I SDS remained above baseline levels for at least 1 week.

 

Conclusions: Single doses of once-weekly somapacitan (0.02-0.16mg/kg) were well tolerated in children with GHD, with IGF-I profiles supporting a once-weekly treatment profile. No clinically significant safety/tolerability signals or immunogenicity concerns were identified.