Safety and Efficacy of Omnitrope® (rhGH) in Short Children Born Small for Gestational Age (SGA)
- Registration Number
- NCT00537914
- Lead Sponsor
- Sandoz
- Brief Summary
This study is performed to investigate the long-term safety, in particular the diabetogenic potential and immunogenicity of rhGH therapy in short children born small for gestational age (SGA).
- Detailed Description
Prospective, open label, non-comparative, multicenter study. Short children born SGA were to be treated until they reached final height, but treatment could be discontinued earlier if medically indicated or if there was inadequate response to treatment.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 278
- Pre-pubertal (Tanner stage I) children born SGA Boys: 4 years of age or older Girls: 4 years of age or older
- Growth disturbance defined as current height SDS < -2.5 (and parental adjusted SDS <-1) for chronological age and sex according to country specific references.
- Birth weight and/or length below -2 standard deviations (SD) for gestational age
- Onset of puberty
- Closed epiphyses
- Diabetes mellitus type I or type II
- Fasting blood glucose greater than 100 mg/dl or greater than 5.6 mmol/l measured in venous blood sample
- Abnormal findings in Oral Glucose Tolerance Test (OGTT) defined by greater than 140 mg/dl or greater than 7.8 mmol/l after 120 minutes
- Known IGF-I level above +2SD for sex and age
- Acute critical illness
- Previous treatment with any hGH preparation
- Treatment with antidiabetic medication (e.g. metformin, insulin)
- Drug abuse, substance abuse, or alcohol abuse
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Omnitrope Omnitrope All enrolled patients received Omnitrope. The median daily dose varied between 0.0340 and 0.0351 mg/kg/day and the maximum range was 0.000 to 0.040 mg/kg/day over all visits.
- Primary Outcome Measures
Name Time Method Number of Participants With Development of Diabetes in Short Children Born SGA During Treatment throughout the study, approximately 13 years The development of diabetes in short children born SGA during treatment was evaluated based on the carbohydrate metabolism parameters FPG, HbA1c and OGTT (basal and 2-h plasma glucose). Only cases which were confirmed by the investigator were included.
- Secondary Outcome Measures
Name Time Method Mean Change in Height (H) (cm) From Baseline Baseline, 3 months, 1 year, 2 years, 5 years and 9 years Mean change in Height from baseline for all patients was reported.
Mean Change in Height Standard Deviation Score Over Time From Baseline Baseline, 3 months, 0.5 year, 9 months, 1 year, 1.25 years, 1.5 years, 1.75 years, 2 years, 3 years, 4 years, 5 years, 6 years, 7 years, 8 years, 9 years, 10 years, 11 years, 12 years, 12.5 years SDS reflects the deviation of a measured value from the mean value of normally growing children of the same sex and chronological age, expressed in units of the standard deviation (SD) of normally growing children of the same sex and chronological age. SDS was calculated according to the formula SDS=(X1-X2)/SD, where X1 is the measured value, X2 the mean value for the relevant chronological age and sex, and SD the reference standard deviation for the relevant sex and age. Refer to SAP page 14 for the formula and to Appendix B (H SDS) and D (HV SDS) for the applied reference Table.
In general, a negative SDS indicates that the value is below average or mean and a positive value means it is above the average or mean. The calculated mean change compared to baseline reflects the catch-up growth over time towards average normal growth starting from below average.Mean Change in Height Velocity (HV) (cm/Year) Over Time From Baseline Baseline, 3 months, 1 year, 2 years, 5 years and 9 years Mean change in Height velocity (HV) (cm/year) over time from baseline was reported.
Mean Change in Height Velocity Standard Deviation Score (HV SDS) Over Time From Baseline Baseline, 3 months, 0.5 year, 9 months, 1 year, 1.25 years, 1.5 years, 1.75 years, 2 years, 3 years, 4 years, 5 years, 6 years, 7 years, 8 years, 9 years, 10 years, 11 years, 12 years, 12.5 years SDS reflects the deviation of a measured value from the mean value of normally growing children of the same sex and chronological age, expressed in units of the standard deviation (SD) of normally growing children of the same sex and chronological age. SDS was calculated according to the formula SDS=(X1-X2)/SD, where X1 is the measured value, X2 the mean value for the relevant chronological age and sex, and SD the reference standard deviation for the relevant sex and age. Refer to SAP page 14 for the formula and to Appendix B (H SDS) and D (HV SDS) for the applied reference Table.
In general, a negative SDS indicates that the value is below average or mean and a positive value means it is above the average or mean. The calculated mean change compared to baseline reflects the initial high increase in height velocity which remains positive over years, but is decreasing over time.Mean Change in Serum IGF-1 Level (Nmol/L) From Baseline Baseline, 3 months, 1 year, 2 years, 5 years and 9 years Mean change in serum IGF-1 level (nmol/L) from baseline was reported.
Mean Change in IGFBP-3 Levels (Nmol/L) From Baseline Baseline, 3 months, 1 year, 2 years, 5 years and 9 years Mean change in IGFBP-3 levels (nmol/L) from baseline was reported.
Number of Participants With the Development of Anti-rhGH Antibodies During Omnitrope Treatment throughout the study, approximately 13 years Number of participants with the development of anti-rhGH antibodies with positive test result were reported.
Trial Locations
- Locations (1)
Sandoz Investigational Site
🇷🇴Iaşi, Romania