Somapacitan, a once-weekly reversible albumin-binding GH derivative, in children with GH deficiency: A randomized dose-escalation trial

the NN8640-4042 Study Group

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32 Scopus citations


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 (0.02-0.16 mg/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.

Original languageAmerican English
Pages (from-to)350-358
Number of pages9
JournalClinical Endocrinology
Issue number4
StatePublished - Oct 2017

Bibliographical note

Funding Information:
The authors would like to thank Bo Grønlund, Mette Suntum (statistician), Knud Vad and Nina Worm White of Novo Nordisk for review of the manuscript. Assistance in finalizing the manuscript for submission was provided by Grace Townshend and Richard McDonald of Watermeadow Medical, funded by Novo Nordisk A/S.

Funding Information:
TB is a board member of Novo Nordisk, Sanofi, Eli Lilly Medtronic and Bayer Health Care, and a consultant for Spring. His institution received research grant support, with receipt of travel and accommodation expenses in some cases, from Abbott, Medtronic, Novo Nordisk, GluSense, Sanofi, Sandoz and Diamyd. He has received honoraria for participating on the speaker’s bureaux of Eli Lilly, Bayer, Novo Nordisk, Medtronic, Sanofi and Roche, and owns stocks of DreamMed. MHR is an employee of Novo Nordisk A/S. JDS has received consultation fees from Ferring, Novo Nordisk and Pfizer. NZ-L and ZG have received consultation fees from Novo Nordisk. LS has received consultation fees from Ferring, Novo Nordisk, Merck, Pfizer and Sandoz, and research grants from Merck, Novo Nordisk and Pfizer.

Publisher Copyright:
© 2017 The Authors. Clinical Endocrinology Published by John Wiley & Sons Ltd


  • IGF-I
  • growth hormone
  • growth hormone deficiency
  • long-acting growth hormone


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