fliGHt was a multicenter, phase 3, open-label trial that investigated safety, tolerability, and efficacy of SKYTROFA® in children with pediatric GHD1*


Key inclusion criteria1:
- Investigator-determined pediatric GHD with supporting biochemical and auxological criteria
- Treatment-experienced patients aged 6 months to 17 years
- Tanner stage < 5
- Open epiphyses
- Treated with commercially available daily hGH therapy ≥ 0.20 mg/kg/week for 13 to 130 weeks
Patients were excluded based on the following1:
- Body weight < 5.5 kg or > 80 kg
- History of malignant disease
- Major medical conditions or clinically significant abnormality likely to affect growth or the evaluation of growth
- Poorly controlled diabetes mellitus or diabetic complications
A broad range of patients enrolled in the 26-week clinical trial1
Baseline demographics and clinical characteristics |
Total enrolled | Previously treated |
---|---|---|
N = 146 | n = 143 | |
Male, n (%) | 110 (75.3) | 109 (76.2) |
White, n (%) | 124 (84.9) | 121 (84.6) |
Hispanic or Latino, n (%) | 10 (6.8) | 10 (7.0) |
Mean chronological age (year) | 10.6 | 10.8 |
BMI (SDS) | –0.3 ± 1.1 | –0.24 ± 1.06 |
Height (SDS) | –1.42 ± 0.84 | –1.40 ± 0.83 |
Tanner stage, n (%) | ||
I | 95 (65.1) | 92 (64.3) |
II | 14 (9.6) | 14 (9.8) |
III | 30 (20.5) | 30 (21.0) |
IV | 7 (4.8) | 7 (4.9) |
IGF-1 SDS | 0.85 ± 1.29 | 0.91 ± 1.25 |
Daily somatropin dose at trial enrollment (mg hGH/kg/week) |
n/a | 0.29 (0.05) |
Daily somatropin dose duration since treatment initiation (years) |
n/a | 1.14 (0.73) |
Safety and tolerability in children who switched from daily hGH1
- Treatment-emergent AEs reported by patients taking SKYTROFA were consistent with those reported by treatment-naïve patients with pediatric GHD studied in the heiGHt trial1
Adverse events
- Most patients experienced AEs that were mild (45%) or moderate (12%) in severity, and 2 SAEs were reported in 1 patient1*
- Most common AEs were pyrexia (12%), nasopharyngitis (10%), upper respiratory tract infection (10%), headache (8%), and oropharyngeal pain (5%)1
Tolerability
- Once-weekly injections of SKYTROFA were well tolerated1
- Throughout the trial, the majority of patients (> 95%) did not experience bruising, swelling, or itching1
Growth rate data through week 26 after switching to SKYTROFA
Height SDS from GHD diagnosis to week 26 of the fliGHt trial1*†
Reprinted with permission from S. Karger AG, Basel.1
†Average parental height SDS = –0.27 (based on N = 143 at fliGHt baseline).1
‡The mean duration was 1.14 years for prior daily somatropin treatment.1
§The mean (SD) most recent height SDS before diagnosis = –2.1 (0.9) (n = 135).1
- The change in height SDS from baseline through 26 weeks was +0.251
- Mean (SD) height SDS at baseline was –1.40 (0.83)1
- Mean AHV was 8.72 cm/year throughout the 6-month fliGHt trial1
Continued growth was observed across all subgroups1
AHV* (cm/year)
Height SDS*, Change From Baseline
Reprinted with permission from S. Karger AG, Basel.1