SAXENDA was evaluated in a 56-week, double-blind, randomized, parallel group, placebo controlled multi-center trial in 251 pubertal pediatric patients aged 12 to 17 years, with BMI corresponding to 30 kg/m2 or greater for adults by international cut-off points [see Dosage and Administration (2.1)] and BMI of 95th percentile or greater for age and sex (NCT02918279). After a 12-week lifestyle run-in period, patients were randomized 1:1 to SAXENDA once-daily or placebo once-daily. The SAXENDA dose was titrated to 3 mg over a 4- to 8-week period based on tolerability as judged by the investigator. Escalation of the trial product was not allowed if the subject had a self-monitored plasma glucose (SMPG) < 56 mg/dL or < 70 mg/dL in the presence of symptoms of hypoglycemia during the week prior to or during the dose escalation visits. The proportion of patients who reached the 3 mg dose was 82.4%; for 8.8% of patients 2.4 mg was the maximum tolerated dose.

The mean age was 14.5 years: 40.6% of patients were male, 87.6% were White, 0.8% were Asian, 8% were Black or African American; 22.3% were of Hispanic or Latino ethnicity. The mean baseline body weight was 100.8 kg, and mean Body Mass Index (BMI) was 35.6 kg/m2.

The proportions of patients who discontinued study drug were 19.2% for the SAXENDA-treated group and 20.6% for the placebo-treated group; 10.4% of patients treated with SAXENDA and no patients treated with placebo discontinued treatment due to an adverse reaction [see Adverse Reactions (6.1)].

The primary endpoint was change in BMI SDS. At baseline, mean BMI SDS was 3.14 in the SAXENDA group and 3.20 in the placebo group. At week 56, treatment with SAXENDA resulted in statistically significant reduction in BMI SDS from baseline compared to placebo. The observed mean change in BMI SDS from baseline to week 56 was -0.23 in the SAXENDA group and -0.00 in the placebo group. The estimated treatment difference in BMI SDS reduction from baseline between SAXENDA and placebo was -0.22 with a 95% confidence interval of -0.37, -0.08; p=0.0022.

The time course of change in BMI SDS with SAXENDA and placebo from baseline through week 56 are depicted in Figure 5.

Figure 5. Change from Baseline in BMI SDS

Changes in weight and BMI with SAXENDA are shown in Table 10. Changes in waist circumference and cardiometabolic parameters with SAXENDA are shown in Table 11.

Table 10. Changes in Weight and BMI at Week 56 for Study 4 (Pediatric Patients Ages 12 to Less than 18)

Full Analysis Set. For body weight and BMI, baseline values are means, changes from baseline at week 56 are estimated means (least-squares) and treatment contrasts at week 56 are estimated treatment differences. Missing observations were imputed from the placebo arm based on a jump to reference multiple (x100) imputation approach.

Table 11. Mean Changes in Anthropometry and Cardiometabolic Parameters in Study 4 (Pediatric Patients Ages 12 to Less than 18)

Full Analysis Set. Baseline values are means, changes from baseline at week 56 are estimated means (least-squares) and treatment contrasts at week 56 are estimated treatment differences. Missing observations were imputed from the placebo arm based on a jump to reference multiple (x100) imputation approach.

    ** Baseline values are geometric means.