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

Condition(s) targeted: Idiopathic Short Stature

Intervention: Saizen® (Drug)

Phase: Phase 3

Status: Completed

Sponsored by: Merck KGaA

Official(s) and/or principal investigator(s):
Medical Responsible, Study Director, Affiliation: Merck Ltd.

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).

Official title: A Randomized, Open-label, Two-arm Parallel Group, No Treatment Group-controlled, Multicenter Phase III Study to Evaluate the Safety and Efficacy of Saizen® 0.067 mg/kg/Day Subcutaneous Injection in Children With Idiopathic Short Stature

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Change from baseline in growth velocity (centimeter/year [cm/yr]) at Month 6

Secondary outcome:

Change from baseline in growth velocity (cm/yr) at Month 12

Changes from baseline in height (centimeter [cm]) at Month 6 and 12

Changes from baseline in height standard deviation score (SDS) at Month 6 and 12

Changes from baseline in serum concentration of insulin-like growth factor-I (IGF-I) and insulin like growth factor binding protein-3 (IGFBP-3) at Month 6 and 12

Percentage of participants who adhered to study treatment

Number of participants with adverse events (AEs)

Minimum age: 5 Years. Maximum age: N/A. Gender(s): Both.

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

Please contact Merck KGaA Communication Center for Recruiting Sites, Located in, Korea, Republic of

Starting date: January 2013
Last updated: July 20, 2015