enrollment-resources | Once-weekly SKYTROFA® (lonapegsomatropin-tcgd)

heiGHt was a 52-week, randomized, open-label, active-controlled, parallel-group phase 3 study of 161 treatment-naïve, prepubertal (Tanner stage I) pediatric patients with GHD aged 3 years and older. Patients were randomized in a 2:1 ratio to receive either SKYTROFA^® 0.24 mg/kg/week (n = 105) or daily somatropin using Genotropin^® 0.24 mg/kg/week (n = 56). The primary endpoint was AHV at 52 weeks.^1,2

fliGHt was a multicenter, phase 3, open-label, 26-week trial investigating the safety, tolerability, and efficacy of SKYTROFA administered once weekly in children with GHD. The trial included 3 treatment-naïve and 143 treatment-experienced patients previously treated with daily hGH for ≤ 130 weeks. Previous daily GH treatments included HumatroPen^®, Genotropin Pen^®, Saizen one.click^®, Omnitrope^® Pen, Genotropin GoQuick^®, Genotropin MiniQuick^®, Norditropin FlexPro^®, and Nutropin AQ^® NuSpin^®. The mean daily somatropin dose was 0.29 mg/kg/week upon entering the trial. Safety and tolerability were the primary endpoints. Patients aged 6 months to 3 years could be treatment naïve and were included only in the safety and tolerability analyses.³

SKYTROFA was studied in an open-label extension study of pediatric patients with GHD who had previously participated in phase 3 SKYTROFA trials. The study enrolled a total of 298 patients: 103 patients on SKYTROFA and 55 patients on daily somatropin from the heiGHt trial, and 140 patients on SKYTROFA from the fliGHt trial. The mean age at baseline of the enliGHten trial was 9.5 years for SKYTROFA-treated patients from the heiGHt trial, 9.5 years for daily somatropin-treated patients from the heiGHt trial, and 11.1 years for patients from the fliGHt trial. Long-term safety was the primary objective.⁴

Once-weekly SKYTROFA^® was studied in a multicenter, phase 3, open-label, 26-week trial investigating the safety, tolerability, and efficacy of SKYTROFA administered once weekly in children with pediatric GHD. The trial included 3 treatment-naïve and 143 treatment-experienced patients previously treated with daily hGH for ≤ 130 weeks. The mean daily somatropin dose was 0.29 mg/kg/week upon entering the trial. Safety and tolerability were the primary endpoints.¹

heiGHt was a 52-week, randomized, open-label, active-controlled, parallel-group phase 3 study of 161 treatment-naïve, prepubertal (Tanner stage I) pediatric patients with GHD aged 3 years and older. Patients were randomized in a 2:1 ratio to receive either SKYTROFA^® 0.24 mg/kg/week (n = 105) or daily somatropin using Genotropin^® 0.24 mg/kg/week (n = 56). The primary endpoint was AHV at 52 weeks.^1,2

Diagnosis of GHD (defined as peak GH ≤ 10 ng/mL confirmed via 2 different GH stimulation tests)
Treatment-naïve males and females (aged 3 to 12 or 3 to 11 years, respectively)
Tanner stage I
Height SDS ≤ -2.0
IGF-1 SDS ≤ -1.0
BMI within ± 2.0 SD of the mean
Bone age ≥ 6 months behind chronological age as determined by a blinded radiologist

Prior exposure to GH or IGF-1 therapies
History of malignant disease (clinically cured tumors permitted)
Contemporaneous malnourishment, idiopathic short stature, small for gestational age, or other non-GHD-related cause of short stature
Prior diagnosis of or receiving treatment for other major medical conditions in which the disease and/or treatment might affect longitudinal growth

Patient baseline demographics²*

	SKYTROFA^® 0.24 mg hGH/kg/week	Daily somatropin^† 0.24 mg hGH/kg/week	Total
	(n = 105)	(n = 56)	(N = 161)
Male, n (%)	86 (82)	46 (82)	132 (82)
White, n (%)	100 (95.2)	52 (92.9)	152 (94.4)
Chronological age (year)	8.5 ± 2.7	8.5 ± 2.8	8.5 ± 2.7
Bone age (year)	5.8 ± 2.6	6.0 ± 2.7	5.9 ± 2.6
Bone age/chronological age (ratio)	0.69 ± 0.16	0.70 ± 0.14	0.69 ± 0.15
Weight (kg)	21.0 ± 6.5	21.2 ± 6.7	21.1 ± 6.6
BMI (kg/m²)	16.1 ± 1.8	16.5 ± 2.2	16.2 ± 1.9
Height (cm)	112.9 ± 14.1	112.2 ± 15.3	112.7 ± 14.5
Height (SDS)	–2.89 ± 0.85	–3.00 ± 0.90	–2.93 ± 0.87
Historical growth rate (cm/year)^‡	3.9 ± 2.0	3.9 ± 1.7	3.9 ± 1.9
IGF-1 SDS	–2.08 ± 0.88	–1.96 ± 0.98	–2.04 ± 0.92

* Plus-minus values are means ± SD.

^† Genotropin^®.

^‡ Historical growth rates based on best available medical records (SKYTROFA [n = 94] and daily somatropin^† [n = 54]).

fliGHt was a multicenter, phase 3, open-label, 26-week trial investigating the safety, tolerability, and efficacy of SKYTROFA^® administered once weekly in children with GHD. The trial included 3 treatment-naïve and 143 treatment-experienced patients previously treated with daily hGH for ≤ 130 weeks. Previous daily GH treatments included HumatroPen^®, Genotropin Pen^®, Saizen one.click^®, Omnitrope^® Pen, Genotropin GoQuick^®, Genotropin MiniQuick^®, Norditropin FlexPro^®, and Nutropin AQ^® NuSpin^®. The mean daily somatropin dose was 0.29 mg/kg/week upon entering the trial. Safety and tolerability were the primary endpoints.¹ Patients aged 6 months to 3 years could be treatment naïve and were included only in the safety and tolerability analyses.

Investigator-determined pediatric GHD with supporting biochemical and auxological criteria
Treatment-experienced patients aged 6 months to 17 years
Tanner stage < V
Open epiphyses
Treated with commercially available daily hGH therapy ≥ 0.20 mg/kg/week for 13 to 130 weeks

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, mean (SD)	–0.3 (1.1)	–0.24 (1.06)
Height SDS, mean (SD)	–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, mean (SD)	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)

enliGHten was an open-label extension study of pediatric patients with GHD who had previously participated in phase 3 SKYTROFA^® trials. The study enrolled a total of 298 patients: 103 patients on SKYTROFA and 55 patients on daily somatropin from the heiGHt trial, and 140 patients on SKYTROFA from the fliGHt trial. The mean age at baseline of the enliGHten trial was 9.5 years for SKYTROFA-treated patients from the heiGHt trial, 9.5 years for daily somatropin-treated patients from the heiGHt trial, and 11.1 years for patients from the fliGHt trial. Treatment completion status was evaluated throughout the study, and the results were stratified accordingly. Long-term safety was the primary objective.²

Patient baseline characteristics²

	Full analysis set	Treatment completers*
	(N = 298)	(n = 81)^†
Age, years, mean (SD)	10.3 (3.4)	13.3 (1.8)
Sex, male, n (%)	235 (78.9)	66 (81.5)
Height SDS, mean (SD)	–1.6 (0.9)	–1.6 (0.7)
IGF‑1 SDS, mean (SD)	1.0 (1.3)	0.8 (1.5)
Tanner stage, n (%)
Stage I	214 (71.8)	22 (30.1)
Stage II	40 (13.4)	17 (23.3)
Stage III	25 (8.4)	28 (38.4)
Stage IV	16 (5.4)	6 (8.2)
Stage V	3 (1.0)	0

* Baseline indicates last nonmissing measurement before the first dose of lonapegsomatropin-tcgd.²

^† Patients completed SKYTROFA^® treatment (mean treatment duration 3.2 years) because the physician deemed that treatment for pediatric GHD was no longer necessary.²

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