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

Idiopathic Short Stature Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01746862
• Other study ID #: EMR200104-533
• Status: Completed
• Phase: Phase 3
• First received: November 30, 2012
• Last updated: June 17, 2016
• Start date: December 2012
• Est. completion date: March 2015

Study information

• Verified date: June 2016
• Source: Merck KGaA
• Contact: n/a
• Is FDA regulated: No
• Health authority: Korea: Food and Drug AdministrationKorea: Institutional Review Board
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 90
• Est. completion date: March 2015
• Est. primary completion date: August 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years and older

Eligibility

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

Study Design

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

Related Conditions & MeSH terms

• Dwarfism
• Idiopathic Short Stature

Intervention

Drug:
• Saizen®
Saizen® (Recombinant-human growth hormone [r-hGH]) will be administered subcutaneously as 6 days per week at a weight based dose of 0.067 mg/kg/day for 12 months in Saizen® treatment group and for 6 months in control group.

Locations

Country	Name	City	State
Korea, Republic of	Please contact Merck KGaA Communication Center for Recruiting Sites	Located in

Sponsors (1)

Lead Sponsor	Collaborator
Merck KGaA

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in growth velocity (centimeter/year [cm/yr]) at Month 6		Baseline, Month 6	No
Secondary	Change from baseline in growth velocity (cm/yr) at Month 12		Baseline, Month 12	No
Secondary	Changes from baseline in height (centimeter [cm]) at Month 6 and 12		Baseline, Month 6 and 12	No
Secondary	Changes from baseline in height standard deviation score (SDS) at Month 6 and 12		Baseline, Month 6 and 12	No
Secondary	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		Baseline, Month 3, 6, 9 and 12	No
Secondary	Percentage of participants who adhered to study treatment		Month 3, 6, 9 and 12	No
Secondary	Number of participants with adverse events (AEs)		Baseline up to Month 13	Yes