Table 1.
Summary of clinical trials for model analysis
| Trial number | Subjects treated with somapacitan and included in model analysis | Study design | Drug dose and administration | Sampling design (rich sampling)a | |
|---|---|---|---|---|---|
| PK samples per profile (15 min to 168 h) | IGF-I samples per profile (0–168 h) | ||||
| Healthy adults (NCT01514500) [14] | 73b | Phase I, randomized, placebo-controlled, double-blind study of single and multiple doses of somapacitan | Single dose only: 5 cohorts treated with a single dose of somapacitan (0.01c–0.32 mg/kg): 0.01 (n = 6), 0.04 (n = 6), 0.08 (n = 6), 0.16 (n = 6), 0.32 (n = 6) mg/kg (n = 30), or placebo (n = 10) for 4 weeks | 27 | 11 |
| Single and multiple dose: 4 cohorts treated with once-weekly doses of somapacitan: (0.02–0.24 mg/kg): 0.02 (n = 12), 0.08 (n = 12), 0.16 (n = 12), 0.24 (n = 13) mg/kg, or placebo (n = 4) for 4 weeks | 27 | 11 | |||
| Subjects with AGHD (NCT01706783) [15] | 26 | Phase I, randomized (3:1), open-label, active-controlled, dose-escalation trial of multiple, once-weekly doses of somapacitan, compared with daily GH | Single and multiple dose: 4 cohorts treated with once-weekly somapacitan (0.02–0.12 mg/kg): 0.02 (n = 7), 0.04 (n = 6), 0.08 (n = 6), and 0.12 (n = 7) mg/kg (n = 24), or daily injections of Norditropin® NordiFlex® (n = 8) for 4 weeks | 24 | 9 |
| Children with GHD (NCT01973244) [13] | 24 | Phase I, randomized (3:1), open-label, active-controlled, dose-escalation trial of single doses of once-weekly somapacitan vs. once-daily GH in children with GHD (0.03 mg/kg) (n = 8 each) for 7 days | Single dose only: 4 cohorts (n = 6) treated with a single dose of somapacitan (0.02–0.16 mg/kg): 0.02, 0.04, 0.08, and 0.16 mg/kg (n = 24) or once-daily Norditropin® SimpleXx® (0.03 mg/kg) (n = 8 each) for 7 days | 12 | 8 |
AGHD adult growth hormone deficiency, GH growth hormone, GHD growth hormone deficiency, IGF-I insulin-like growth factor-I, PD pharmacodynamic, PK pharmacokinetic
a Rich sampling provided complete PK/PD profiles for each patient’s profile according to the number of samples specified
b In this trial the cohort randomized for treatment with single doses of somapacitan was limited to Caucasian subjects, while the multiple-dose cohort included both Japanese and Caucasian subjects
c Subjects treated with somapacitan 0.01 mg/kg were not included in the present analysis