fliGHt was a multicenter, phase 3, open-label trial that investigated safety, tolerability, and efficacy of SKYTROFA® in children with pediatric GHD1*
STUDY DESIGNS
*Safety and tolerability were the primary endpoints.1
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
*
Both events were assessed by the investigator as unrelated to the study drug1
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
*Values in graph are observed means.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
†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
*
Based on an analysis of covariance (ANCOVA) model that included: sex, baseline age, peak stimulated GH levels (log transformed), height SDS minus average parental height SDS, prior somatropin dose level (log transformed), and duration (log transformed).1
