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

Merck KGaA (EMD Serono)

Status and phase

Completed

Phase 3

Conditions

Idiopathic Short Stature

Treatments

Drug: Saizen®

Study type

Interventional

Funder types

Industry

Identifiers

NCT01746862

EMR200104-533

Details and patient eligibility

About

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).

Enrollment

90 patients

Sex

All

Ages

5+ years old

Volunteers

No Healthy Volunteers

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