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

NCT01746862 | Completed Phase 3 Results

• 1 other identifier: EMR200104-533
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief 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).

Conditions

Idiopathic Short Stature

Keywords

Saizen®; Recombinant human growth hormone (r-hGH); Idiopathic short stature; Height velocity; Bone age

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method

  Baseline height is defined as the last available height measurement before randomization. Baseline height velocity = (\[Baseline height minus height measurement obtained at least 6 months prior\] / 6) \* 12. Height velocity at Month 6 = (\[Month 6 height minus height measurement obtained at least 6 months prior\] / 6) \* 12.

  Baseline, Month 6

Secondary Outcomes (7)

• Change From Baseline in Height Velocity at Month 12

  Baseline, Month 12

• Change From Baseline in Height at Month 6 and 12

  Baseline, Month 6, Month 12

• Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12

  Baseline, Month 6, Month 12

• Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12

  Baseline, Month 6, Month 12

• Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12

  Baseline, Month 6, Month 12

Study Arms (2)

Saizen Test Group

EXPERIMENTAL

Drug: Saizen®

Saizen Control Group

ACTIVE COMPARATOR

Drug: Saizen®

Interventions

Saizen® DRUG

Subjects in the Saizen test group will receive Saizen (recombinant-human growth hormone \[r-hGH\]) subcutaneously 6 days per week at a weight based dose of 0.067 milligram per kilogram of body weight per day (mg/kg/day) for 12 months.

Also known as: Somatropin, r-hGH

Saizen Test Group

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age greater than or equal to 5 years
• Pre-pubertal; testicular volume less than 4 milliliter (in males) and breast Stage 1 (in females)
• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Merck KGaA, Darmstadt, Germany: lead

Study Sites (1)

Please contact Merck KGaA Communication Center for Recruiting Sites

Located in, South Korea

Location

Related Publications (1)

• Chung WY, Yoo HW, Hwang JS, Ko CW, Kim HS, Jin DK, Lee KH, Han HS, Paranchothy P, Suh BK. Effect of Growth Hormone Therapy on Height Velocity in Korean Children with Idiopathic Short Stature: A Phase III Randomised Controlled Trial. Horm Res Paediatr. 2018;90(1):44-53. doi: 10.1159/000491016. Epub 2018 Aug 15.

  PMID: 30110706 DERIVED

MeSH Terms

Interventions

Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Merck KGaA Communication Center
• Organization: Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany

service@merckgroup.com

+49-6151-72-5200

Study Officials

• Medical Responsible

  Merck Ltd.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2012
• First Posted: December 11, 2012
• Study Start: December 1, 2012
• Primary Completion: August 1, 2014
• Study Completion: March 1, 2015
• Last Updated: October 18, 2016
• Results First Posted: October 18, 2016

Record last verified: 2016-08

Locations

KR (1)