A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
|ClinicalTrials.gov Identifier: NCT01746862|
Recruitment Status : Completed
First Posted : December 11, 2012
Results First Posted : October 18, 2016
Last Update Posted : October 18, 2016
|Condition or disease||Intervention/treatment||Phase|
|Idiopathic Short Stature||Drug: Saizen®||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||90 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Randomized, Open-label, Two-arm Parallel Group, No Treatment Group-controlled, Multicenter Phase III Study to Evaluate the Safety and Efficacy of Saizen® 0.067 mg/kg/Day Subcutaneous Injection in Children With Idiopathic Short Stature|
|Study Start Date :||December 2012|
|Primary Completion Date :||August 2014|
|Study Completion Date :||March 2015|
|Experimental: Saizen Test Group||
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.
|Active Comparator: Saizen Control Group||
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.
- Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method [ Time Frame: Baseline, Month 6 ]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.
- Change From Baseline in Height Velocity at Month 12 [ Time Frame: Baseline, 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.
- 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 [ Time Frame: Baseline, Month 6, Month 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.
- 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 [ Time Frame: Baseline, Month 6, Month 12 ]
- Percentage of Adherence to Study Treatment [ Time Frame: 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group) ]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.
- Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs [ Time Frame: Baseline up to Month 13 ]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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01746862
|Korea, Republic of|
|Please contact Merck KGaA Communication Center for Recruiting Sites|
|Located in, Korea, Republic of|
|Study Director:||Medical Responsible||Merck Ltd.|