This is an open-label, multi-center, randomized, two-arm parallel, no-treatment group controlled (only for the first 6 months), Phase 3 study in children with ISS. The subjects will be treated with 0.067 milligram/kilogram/day (mg/kg/day) of Saizen®, weight base dose, for 12 months (12 months of treatment in the test group, and 6 months of no treatment and then 6 months of treatment in the control group).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
90
Subjects in the Saizen test group will receive Saizen (recombinant-human growth hormone \[r-hGH\]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months.
Subjects in the Saizen control group will receive no treatment for the first 6 months and thereafter will receive Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months.
Please contact Merck KGaA Communication Center for Recruiting Sites
Located in, South Korea
Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method
Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = (\[Baseline height minus height measurement obtained at least 6 months prior\] / 6) \* 12. Height velocity at Month 6 = (\[Month 6 height minus height measurement obtained at least 6 months prior\] / 6) \* 12.
Time frame: Baseline, Month 6
Change From Baseline in Height Velocity at Month 12
Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = (\[Baseline height minus height measurement obtained at least 12 months prior\] / 12) \* 12. Height velocity at Month 12 = (\[Month 12 height minus height measurement obtained at least 12 months prior\] / 12) \* 12.
Time frame: Baseline, Month 12
Change From Baseline in Height at Month 6 and 12
Time frame: Baseline, Month 6, Month 12
Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12
Height SDS was calculated as: Height SDS = (measured height - population mean) / population standard deviation, where mean and standard deviation were based on the Korean standard growth charts. SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) a subject's value was relative to the mean of the reference population. The scores were centred around zero. Negative score indicated a subject was smaller for their age/gender.
Time frame: Baseline, Month 6, Month 12
Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12
Time frame: Baseline, Month 6, Month 12
Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12
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Time frame: Baseline, Month 6, Month 12
Percentage of Adherence to Study Treatment
Percentage of adherence to study treatment (adherence rate) was defined as the actual number of received treatments divided by the scheduled number of treatments multiplied by 100. The adherence rate for 6 months was calculated from Baseline to 6 months for the Saizen Test Group and from Month 6 to Month 12 for the Saizen Control Group.
Time frame: 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group)
Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs
An adverse event (AE) was defined as any untoward medical occurrence which does not necessarily have a causal relationship with this the study drug. An AE was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. A serious adverse event (SAE) was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. For the Saizen Test Group, TEAEs were defined as events that occurred or worsened at or after the first administration of treatment and for the Saizen Control Group, TEAEs were defined as events that occurred or worsened at or after the randomization.
Time frame: Baseline up to Month 13