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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Merck KGaA
ClinicalTrials.gov Identifier:
NCT01746862
First received: November 30, 2012
Last updated: July 1, 2014
Last verified: July 2014

November 30, 2012
July 1, 2014
January 2013
June 2016   (final data collection date for primary outcome measure)
Change from baseline in growth velocity (centimeter/year [cm/yr]) at Month 6 [ Time Frame: Baseline, Month 6 ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT01746862 on ClinicalTrials.gov Archive Site
  • 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 ]
Same as current
Not Provided
Not Provided
 
A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
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

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).

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Idiopathic Short Stature
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
  • Experimental: Saizen® treatment group
    Saizen® 0.067 mg/kg/day will be given for the duration of 12 months.
    Intervention: Drug: Saizen®
  • 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.
    Intervention: Drug: Saizen®
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
90
June 2016
June 2016   (final data collection date for primary outcome measure)

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
Both
5 Years and older
No
Contact information is only displayed when the study is recruiting subjects
Korea, Republic of
 
NCT01746862
EMR200104-533
Not Provided
Merck KGaA
Merck KGaA
Not Provided
Study Director: Medical Responsible Merck Ltd.
Merck KGaA
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP