A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
Condition: Idiopathic Short Stature
MeSH terms: Dwarfism
Intervention: Saizen®; Saizen®
Intervention description: 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.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 90.0
Sex: All
Minimum age: 5 Years
Healthy volunteers: No
Keywords: Saizen®; Recombinant human growth hormone (r-hGH); Idiopathic short stature; Height velocity; Bone age

Summary:

      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).
    

Eligibility criteria:

        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