Safety and Efficacy of Omnitrope® (rhGH) in Short Children Born Small for Gestational Age (SGA)

Sandoz278 enrolled

Overview

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).

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

278

Conditions

Small for Gestational Age

Interventions

OmnitropeDRUG

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.

Eligibility

Sex: ALLMin age: 4 Years

Medical Language ↔ Plain English

Inclusion Criteria: * 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 Exclusion Criteria: * 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

Locations (31)

Outcomes

Primary Outcomes

Number of Participants With Development of Diabetes in Short Children Born SGA During Treatment

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.

Time frame: throughout the study, approximately 13 years

Secondary Outcomes

Mean Change in Height (H) (cm) From Baseline

Mean change in Height from baseline for all patients was reported.

Time frame: Baseline, 3 months, 1 year, 2 years, 5 years and 9 years

Mean Change in Height Standard Deviation Score Over Time From Baseline

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.

Time frame: 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

Mean Change in Height Velocity (HV) (cm/Year) Over Time From Baseline

Mean change in Height velocity (HV) (cm/year) over time from baseline was reported.

Time frame: Baseline, 3 months, 1 year, 2 years, 5 years and 9 years

Data from ClinicalTrials.gov

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Sandoz Investigational Site

Edegem, Antwerpen, Belgium

Sandoz Investigational Site

Prague, Prague, Czechia

Sandoz Investigational Site

Hradec Králové, Czechia

Sandoz Investigational Site

Ústí nad Labem, Czechia

Sandoz Investigational Site

Tbilisi, Georgia

Sandoz Investigational Site

Munich, Bavaria, Germany

Sandoz Investigational Site

Sankt Augustin, North Rhine-Westphalia, Germany

Sandoz Investigational Site

Szeged, Csongrád megye, Hungary

Sandoz Investigational Site

Budapest, Hungary

Sandoz Investigational Site

Budapest, Hungary

...and 21 more locations

Mean Change in Height Velocity Standard Deviation Score (HV SDS) Over Time From Baseline

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.