Clinical Trial: Predictive Markers in Chinese Growth Hormone Deficiency (GHD) Children Treated With Saizen®

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase IV Open-label Study of Predictive Markers in Growth Hormone Deficient Pre-pubertal Children Treated With Saizen®

Brief Summary: This is an open-label, prospective, multicentric, non-comparative, non-randomized Phase IV interventional study in which subjects pre-diagnosed with Growth Hormone Deficiency (GHD) were treated for 4 weeks with Saizen to compare the response between GHD children born appropriate for gestational age (AGA) and those born small for gestation age (SGA) after 4 weeks of Saizen therapy.

Detailed Summary:

The response to growth hormone (GH) treatment, short-term as well as long-term, displays considerable inter individual variability. This is particularly evident for the endpoint of paediatric GH administration, that is (i.e.) the growth response, which is pronounced in children who are affected by GHD. This is an open-label, multicentric study in which subjects pre-diagnosed with GHD were treated for 4 weeks with Saizen. Two hundred fourteen GHD evaluable pre-pubertal subjects were planned to be recruited in approximately 9 sites in China. Demographic data, medical history, tanner stage, physical examination, body weight, height, bone age measurement, body mass index, review of baseline medications and procedures and blood sampling were performed at baseline visit, end of treatment visit (week 4) and at 4 week follow-up visit.

OBJECTIVES

Primary objective:

  • To compare the response between GHD children born AGA and those born SGA after 4 weeks of Saizen therapy

Secondary Objectives:

  • To explore the contribution of selected genes to the phenotype of GHD children
  • To explore the impact of gene polymorphisms on the levels of specific serum biomarkers in GHD children after 4 weeks of Saizen therapy
  • To explore the relationships between changes in gene expression and changes in serum biomarkers after 4 weeks of Saizen therapy and the spectrum of gene polymorphisms in GHD children

Sponsor: Merck KGaA

Current Primary Outcome: Change From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4 [ Time Frame: Baseline and Week 4 ]

Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) was calculated as logarithm (log) 10 actual value of IGF-1 - log 10 (mean reference value of IGF-1) divided by log10 reference standard deviation of IGF-1.


Original Primary Outcome: Changes in serum Insulin like growth factor-1 standard deviation score (IGF-1SDS) [ Time Frame: At 4 weeks ]

Current Secondary Outcome:

  • Change From Baseline in Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels at Week 4 [ Time Frame: Baseline and Week 4 ]
  • Change From Baseline in Fasting Glucose at Week 4 [ Time Frame: Baseline and Week 4 ]
  • Change From Baseline in Fasting Insulin at Week 4 [ Time Frame: Baseline and Week 4 ]
  • Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) Test at Week 4 [ Time Frame: Baseline and Week 4 ]
    HOMA-IR is used to assess insulin resistance and calculated by an empirical mathematical formula based on fasting plasma glucose and fasting plasma insulin levels. HOMA-IR = fasting plasma insulin (picomole/liter [pmol/L]) * fasting plasma glucose (millimole/liter [mmol/L]) divided by 22.5.
  • Change From Baseline in Lipid Profile at Week 4 [ Time Frame: Baseline and Week 4 ]
    Total cholesterol, high-density lipoprotein (HDL)-cholesterol, low-density lipoprotein (LDL)-cholesterol and triglycerides levels were evaluated.


Original Secondary Outcome:

  • Insulin like Growth Factor binding protein-3 (IGFBP-3) levels [ Time Frame: At 4 weeks ]
  • Fasting glucose, fasting insulin, insulin resistance [ Time Frame: At 4 weeks ]
  • Lipid profile [ Time Frame: At 4 weeks ]


Information By: Merck KGaA

Dates:
Date Received: August 22, 2010
Date Started: March 2007
Date Completion: April 2009
Last Updated: January 20, 2014
Last Verified: January 2014