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

• Status: Completed
• Trial end date: 2015-03-01
• Target enrollment: 0
• Participant gender: All

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Merck KGaA; Merck KGaA, Darmstadt, Germany

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