A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
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).
Inclusion Criteria:

 -  Age greater than or equal to 5 years

 -  Pre-pubertal; testicular volume less than 4 milliliter (in males) and breast Stage 1
 (in females)

 -  The official records of height (for example records measured in hospitals or schools)
 during previous 6 months or more preceding inclusion in the study (self-measurement of
 the height at home will not be considered as a valid record)

 -  Height less than or equal to 3rd percentile compared to same sex, same age

 -  Peak serum growth hormone (GH) greater than 10 microgram per liter (mcg/L) in GH
 stimulation test (results of peak serum GH greater than 10 mcg/L in GH stimulation
 test within 1 year can be used instead)

 -  Naive to GH therapy

 -  Normal birth weight (that is greater than or equal to 3rd percentile when compared to
 same sex)

 -  Normal thyroid function

 -  Normal karyotype in girls

 -  Written informed consent from parent/guardian

 -  Written informed consent from the subject who speaks, understand, read, and write
 Korean

 -  Bone age less than 10 years in boys and less than 9 years in girls, whose difference
 between the bone and chronological age is no more than 3 years

 Exclusion Criteria:

 -  Puberty development (Tanner stage greater than or equal to 2)

 -  Skeletal dysplasia or abnormal body proportions

 -  Chronic systemic illness

 -  Dysmorphic syndrome

 -  Growth Hormone Deficiency

 -  Small for Gestational Age (SGA)

 -  Current medication for Attention deficit hyperactivity disorder (ADHD) or
 hyperactivity disorder

 -  Current medication with drugs that may influence secretion or action of growth hormone
 (such as estrogen, androgen, anabolic steroid, corticosteroid, thyroxine, aromatase
 inhibitors)

 -  Diabetes mellitus

 -  Kidney transplantation

 -  Acute critical illness, including complications following open heart surgery,
 abdominal surgery or multiple accidental trauma

 -  Acute respiratory failure

 -  Malignancy or previous therapy for malignancy

 -  Known hypersensitivity to somatotropin or any of its excipients including cresol or
 glycerol

 -  Closed epiphyses, progression or recurrence of an underlying intracranial tumor,
 chronic renal disease

 -  Endocrinologic or metabolic disorders such as Prader-Willi syndrome; Russel-Silver
 syndrome; Seckel syndrome; Down syndrome; Cushing syndrome; Noonan syndrome; short
 stature caused by other chromosomal abnormalities

 -  The disorders that explain short stature such as psychiatric disorders, nutritional
 disorders, and chronic debilitating diseases

 -  Participation in another clinical trial within the past 3 months

 -  Status of legal incapacity or limited legal capacity of the parents or legal guardian