The trial will compare a twice-monthly somavaratan dosing regimen for non-inferiority of treatment effect against daily injections of rhGH.
This study is designed as a pivotal study to compare the safety and efficacy of a selected dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open label study of 12 months duration. The primary endpoint is height velocity at 12 months.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
138
Somavaratan will be administered per dose and schedule specified in the arm description.
rhGH will be administered per dose and schedule specified in the arm description.
Annual Height Velocity
Height measured without shoes in triplicate by stadiometer. Annual height velocity was calculated as (height at Month 12 - height at Baseline)/(Month 12 Date - Baseline Date) \* 365.25, where height was expressed as centimeters (cm) so that height velocity is expressed as centimeters per year (cm/yr). Annual height velocity after 12 months continuous treatment with either somavaratan or daily rhGH has been reported. Missing data was imputed using last observation carried forward. Least square (LS) mean was calculated using analysis of covariance (ANCOVA) model.
Time frame: 12 months
Change From Baseline in Height Standard Deviation Score (SDS) at Month 12
Height SDS was determined using the Center for Disease Control (CDC) Clinical Growth Charts; 2000. The SD score was calculated as the participant's height value minus the mean divided by the standard deviation (SD). The mean and the SD vary depending on the age and sex of the participant. Mean change from baseline in height SDS at Month 12 is presented.
Time frame: Baseline, Month 12
Change From Baseline in Bone Age Relative to Chronological Age at Month 12, as Assessed by Central Reader
Bone age was assessed from a radiograph of the left hand and wrist by central reader.
Time frame: Baseline, Month 12
Change From Baseline in Body Mass Index (BMI) at Month 12
The BMI is a person's weight in kilograms (kg) divided by the square of height in meters.
Time frame: Baseline, Month 12
Change From Baseline in Body Weight at Month 12
Body weight measured in light clothing and without shoes.
Time frame: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor 1 (IGF-I) SDS at Month 12
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The SD score was calculated as the actual value of IGF-I minus mean reference value of IGF-1 divided by reference standard deviation of IGF-I. The mean and the SD vary depending on the age and sex of the participant. Change in IGF-I level (SD score) at Month 12 from Baseline was assessed. A higher score reflects a better outcome.
Time frame: Baseline, Month 12
Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP-3) at Month 12
Time frame: Baseline, Month 12
Number of Participants With Adverse Events (AEs)
An AE was defined as any untoward medical occurrence that develops or worsens in severity during the conduct of a clinical study and does not necessarily have a causal relationship to the study drug. SAEs included death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized the participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of serious and non-serious AEs regardless of causality is located in 'Reported Adverse Events module'.
Time frame: Baseline up to Month 12