a phase 3 study to evaluate the safety and efficacy of saizen® in children with idiopathic short stature (iss)

Conditions: Idiopathic Short Stature

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

Keywords: saizen® recombinant human growth hormone (r-hgh) idiopathic short stature height velocity bone age