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

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ClinicalTrials.gov Identifier: NCT01187550
Recruitment Status : Completed
First Posted : August 24, 2010
Results First Posted : August 7, 2012
Last Update Posted : February 13, 2014
Sponsor:
Collaborator:
Merck Serono Co., Ltd., China
Information provided by (Responsible Party):
Merck KGaA, Darmstadt, Germany

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Dwarfism, Pituitary
Intervention Drug: Recombinant human growth hormone (r-hGH)
Enrollment 214
Recruitment Details  
Pre-assignment Details Out of 214 participants enrolled in the study, 1 participant could not be categorized as appropriate for gestational age (AGA) or small for gestational age (SGA) since weight and height at birth was not available.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks. Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Period Title: Overall Study
Started 183 30
Treated 175 30
Completed 169 29
Not Completed 14 1
Reason Not Completed
Protocol Violation             3             1
Other             3             0
Randomized but not treated             8             0
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA) Total
Hide Arm/Group Description Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks. Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks. Total of all reporting groups
Overall Number of Baseline Participants 175 30 205
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 175 participants 30 participants 205 participants
10.52  (3.844) 9.39  (4.349) 10.35  (3.931)
[1]
Measure Description: Out of a total of 213 participants, data for baseline measure (age) was available for only 205 participants who were treated.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 175 participants 30 participants 205 participants
Female
46
  26.3%
6
  20.0%
52
  25.4%
Male
129
  73.7%
24
  80.0%
153
  74.6%
[1]
Measure Description: Out of a total of 213 participants, data for baseline measure (gender) was available for only 205 participants who were treated.
1.Primary Outcome
Title Change From Baseline in Serum Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) Levels at Week 4
Hide Description 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.
Time Frame Baseline and Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
The intent-to-treat (ITT) population set included all the participants who received at least 1 dose of study medication.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description:
Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks.
Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Overall Number of Participants Analyzed 175 30
Mean (Standard Deviation)
Unit of Measure: nanogram/millilter (ng/mL)
Baseline -2.04  (2.341) -2.22  (2.390)
Change at Week 4 1.38  (1.358) 0.85  (1.257)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.194
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
2.Secondary Outcome
Title Change From Baseline in Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) Levels at Week 4
Hide Description [Not Specified]
Time Frame Baseline and Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population set included all the participants who received at least 1 dose of study medication.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description:
Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks.
Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Overall Number of Participants Analyzed 175 30
Mean (Standard Deviation)
Unit of Measure: microgram/mL (mcg/mL)
Baseline 3.16  (1.386) 2.57  (1.525)
Change at Week 4 0.58  (0.869) 0.51  (0.805)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.752
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in Fasting Glucose at Week 4
Hide Description [Not Specified]
Time Frame Baseline and Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population set included all the participants who received at least 1 dose of study medication. Here ‘N’ (Number of participants analyzed) signified those participants who were evaluable for this measure.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description:
Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks.
Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Overall Number of Participants Analyzed 173 30
Mean (Standard Deviation)
Unit of Measure: millimole/liter (mmol/L)
Baseline 4.83  (0.471) 4.46  (1.106)
Change at Week 4 0.17  (0.578) 0.22  (0.886)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.710
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in Fasting Insulin at Week 4
Hide Description [Not Specified]
Time Frame Baseline and Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population set included all the participants who received at least 1 dose of study medication. Here ‘N’ (Number of participants analyzed) signified those participants who were evaluable for this measure.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description:
Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks.
Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Overall Number of Participants Analyzed 172 26
Mean (Standard Deviation)
Unit of Measure: picomole/L (pmol/L)
Baseline 36.17  (74.575) 23.58  (14.892)
Change at Week 4 10.08  (81.674) 12.04  (32.389)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.265
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) Test at Week 4
Hide Description 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.
Time Frame Baseline and Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population set included all the participants who received at least 1 dose of study medication. Here ‘N’ (Number of participants analyzed) signified those participants who were evaluable for this measure.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description:
Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks.
Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Overall Number of Participants Analyzed 171 26
Mean (Standard Deviation)
Unit of Measure: pmol/L*mmol/L
Baseline 7.91  (15.662) 5.06  (3.290)
Change at Week 4 2.96  (18.458) 2.48  (6.527)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.308
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
6.Secondary Outcome
Title Change From Baseline in Lipid Profile at Week 4
Hide Description Total cholesterol, high-density lipoprotein (HDL)-cholesterol, low-density lipoprotein (LDL)-cholesterol and triglycerides levels were evaluated.
Time Frame Baseline and Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population set included all the participants who received at least 1 dose of study medication. Here ‘N’ (Number of participants analyzed) signified those participants who were evaluable for this measure.
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description:
Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks.
Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
Overall Number of Participants Analyzed 174 30
Mean (Standard Deviation)
Unit of Measure: mmol/L
Baseline (Total Cholesterol) 4.16  (0.752) 4.22  (0.833)
Baseline (HDL-Cholesterol) 1.56  (0.452) 1.59  (0.443)
Baseline (LDL-Cholesterol) 2.18  (0.672) 2.19  (0.709)
Baseline (Triglycerides) 0.99  (0.527) 1.02  (0.638)
Change at Week 4 (Total Cholesterol) -0.28  (0.601) -0.03  (0.612)
Change at Week 4 (HDL-Cholesterol) -0.10  (0.317) 0.02  (0.329)
Change at Week 4 (LDL-Cholesterol) -0.24  (0.546) 0.02  (0.502)
Change at Week 4 (Triglycerides) 0.12  (0.551) -0.09  (0.831)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments For total cholesterol: Wilcoxon rank sum test was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.078
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments For HDL-cholesterol: Wilcoxon rank sum test was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.033
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments For LDL-cholesterol: Wilcoxon rank sum test was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.041
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Appropriate for Gestational Age (AGA), Small for Gestational Age (SGA)
Comments For triglycerides: Wilcoxon rank sum test was used to calculate p-value.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.091
Comments [Not Specified]
Method Wilcoxon rank sum test
Comments [Not Specified]
Time Frame Adverse Events (AEs) are collected on an ongoing basis from day of written informed consent. All new AEs must be recorded until 4 weeks post drug administration. AEs are classified as pre-treatment, treatment-emergent and post-treatment.
Adverse Event Reporting Description Pre-Treatment:Medical conditions present at initial study visit that did not worsen in severity or frequency during study;Treatment-Emergent: If onset date of AE was on or after the first dose date of the study medication; Post-Treatment: If the onset date of AE was post 4 weeks after drug administration for participants who completed the study.
 
Arm/Group Title Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Hide Arm/Group Description Participants in AGA group received Saizen (recombinant human growth hormone, r-hGH) subcutaneously (sc) at the daily dose of 0.033 milligram/kilogram (mg/kg) body weight for 4 weeks. Participants in SGA group received Saizen (r-hGH) sc at the daily dose of 0.033 mg/kg body weight for 4 weeks.
All-Cause Mortality
Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Affected / at Risk (%) Affected / at Risk (%)
Total   1/175 (0.57%)   0/30 (0.00%) 
Infections and infestations     
Appendicitis * 1  1/175 (0.57%)  0/30 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Appropriate for Gestational Age (AGA) Small for Gestational Age (SGA)
Affected / at Risk (%) Affected / at Risk (%)
Total   27/175 (15.43%)   3/30 (10.00%) 
Eye disorders     
Conjunctivitis * 1  1/175 (0.57%)  0/30 (0.00%) 
Eye Oedema * 1  1/175 (0.57%)  0/30 (0.00%) 
Ocular Hypertension * 1  2/175 (1.14%)  0/30 (0.00%) 
Gastrointestinal disorders     
Abdominal Pain Upper * 1  1/175 (0.57%)  0/30 (0.00%) 
Nausea * 1  1/175 (0.57%)  0/30 (0.00%) 
Vomiting * 1  2/175 (1.14%)  0/30 (0.00%) 
General disorders     
Hyperhidrosis * 1  0/175 (0.00%)  1/30 (3.33%) 
Injection Site Dermatitis * 1  1/175 (0.57%)  0/30 (0.00%) 
Injection Site Reaction * 1  1/175 (0.57%)  0/30 (0.00%) 
Pyrexia * 1  5/175 (2.86%)  1/30 (3.33%) 
Investigations     
Liver Function Test Abnormal * 1  1/175 (0.57%)  0/30 (0.00%) 
Metabolism and nutrition disorders     
Anorexia * 1  0/175 (0.00%)  1/30 (3.33%) 
Musculoskeletal and connective tissue disorders     
Pain In Extremity * 1  1/175 (0.57%)  1/30 (3.33%) 
Nervous system disorders     
Headache * 1  3/175 (1.71%)  0/30 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Bronchitis * 1  1/175 (0.57%)  0/30 (0.00%) 
Cough * 1  1/175 (0.57%)  0/30 (0.00%) 
Nasopharyngitis * 1  2/175 (1.14%)  0/30 (0.00%) 
Rhinorrhoea * 1  2/175 (1.14%)  0/30 (0.00%) 
Tonsillitis * 1  1/175 (0.57%)  0/30 (0.00%) 
Upper Respiratory Tract Infection * 1  4/175 (2.29%)  0/30 (0.00%) 
Skin and subcutaneous tissue disorders     
Skin Infection * 1  1/175 (0.57%)  0/30 (0.00%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.1)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Merck KGaA Communication Center
Organization: Merck Serono, a division of Merck KGaA
Phone: +49-6151-72-5200
EMail: service@merckgroup.com
Layout table for additonal information
Responsible Party: Merck KGaA, Darmstadt, Germany
ClinicalTrials.gov Identifier: NCT01187550     History of Changes
Other Study ID Numbers: 27709
First Submitted: August 22, 2010
First Posted: August 24, 2010
Results First Submitted: May 9, 2012
Results First Posted: August 7, 2012
Last Update Posted: February 13, 2014