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

This study is currently recruiting participants.
Verified February 2014 by Merck KGaA
Sponsor:
Information provided by (Responsible Party):
Merck KGaA
ClinicalTrials.gov Identifier:
NCT01746862
First received: November 30, 2012
Last updated: February 7, 2014
Last verified: February 2014
  Purpose

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).


Condition Intervention Phase
Idiopathic Short Stature
Drug: Saizen®
Phase 3

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

Resource links provided by NLM:


Further study details as provided by Merck KGaA:

Primary Outcome Measures:
  • Change from baseline in growth velocity (centimeter/year [cm/yr]) at Month 6 [ Time Frame: Baseline, Month 6 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from baseline in growth velocity (cm/yr) at Month 12 [ Time Frame: Baseline, Month 12 ] [ Designated as safety issue: No ]
  • Changes from baseline in height (centimeter [cm]) at Month 6 and 12 [ Time Frame: Baseline, Month 6 and 12 ] [ Designated as safety issue: No ]
  • Changes from baseline in height standard deviation score (SDS) at Month 6 and 12 [ Time Frame: Baseline, Month 6 and 12 ] [ Designated as safety issue: No ]
  • 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 [ Time Frame: Baseline, Month 3, 6, 9 and 12 ] [ Designated as safety issue: No ]
  • Percentage of participants who adhered to study treatment [ Time Frame: Month 3, 6, 9 and 12 ] [ Designated as safety issue: No ]
  • Number of participants with adverse events (AEs) [ Time Frame: Baseline up to Month 13 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 87
Study Start Date: January 2013
Estimated Study Completion Date: June 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Saizen® treatment group
Saizen® 0.067 mg/kg/day will be given for the duration of 12 months.
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.
Other Names:
  • Somatropin
  • r-hGH
Active Comparator: Control group
For the first 6 months during the study no treatment will be given and for next 6 months Saizen® 0.067 mg/kg/day will be given.
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.
Other Names:
  • Somatropin
  • r-hGH

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01746862

Contacts
Contact: Merck KGaA Communication Center +49-6151-72-5200 service@merckgroup.com

Locations
Korea, Republic of
Please contact Merck KGaA Communication Center for Recruting Sites Recruiting
Located in, Korea, Republic of
Sponsors and Collaborators
Merck KGaA
Investigators
Study Director: Medical Responsible Merck Ltd.
  More Information

No publications provided

Responsible Party: Merck KGaA
ClinicalTrials.gov Identifier: NCT01746862     History of Changes
Other Study ID Numbers: EMR200104-533
Study First Received: November 30, 2012
Last Updated: February 7, 2014
Health Authority: Korea: Food and Drug Administration
Korea: Institutional Review Board

Keywords provided by Merck KGaA:
Saizen®
Recombinant human growth hormone (r-hGH)
Idiopathic short stature
Height velocity
Bone age

Additional relevant MeSH terms:
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases

ClinicalTrials.gov processed this record on April 16, 2014