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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Merck KGaA
ClinicalTrials.gov Identifier:
NCT01746862
First received: November 30, 2012
Last updated: August 24, 2016
Last verified: August 2016
Results First Received: August 24, 2016  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Idiopathic Short Stature
Intervention: Drug: Saizen®

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Saizen Test Group Subjects in the Saizen test group received 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.
Saizen Control Group Subjects in the Saizen control group received no treatment for the first 6 months and thereafter received Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months.

Participant Flow:   Overall Study
    Saizen Test Group   Saizen Control Group
STARTED   60   30 
Treated   59   30 
COMPLETED   52   27 
NOT COMPLETED   8   3 
Withdrawal by Subject                1                1 
Protocol Violation                1                0 
Consent withdrawal by parent’s guardian                5                2 
Entered puberty during the study                1                0 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Safety analysis set (SAS) included all randomized subjects who received at least 1 dose of planned trial treatment and had follow-up safety data. SAS also included the safety data for all subjects during non-treatment (the first 6 months after enrollment) period who belonged to control group.

Reporting Groups
  Description
Saizen Test Group Subjects in the Saizen test group received 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.
Saizen Control Group Subjects in the Saizen control group received no treatment for the first 6 months and thereafter received Saizen (r-hGH) subcutaneously 6 days per week at a weight based dose of 0.067 mg/kg/day for the next 6 months.
Total Total of all reporting groups

Baseline Measures
   Saizen Test Group   Saizen Control Group   Total 
Overall Participants Analyzed 
[Units: Participants]
 59   30   89 
Age 
[Units: Years]
Mean (Standard Deviation)
 6.79  (1.54)   6.83  (1.61)   6.80  (1.55) 
Gender 
[Units: Participants]
     
Female   29   13   42 
Male   30   17   47 


  Outcome Measures
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1.  Primary:   Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method   [ Time Frame: Baseline, Month 6 ]

2.  Secondary:   Change From Baseline in Height Velocity at Month 12   [ Time Frame: Baseline, Month 12 ]

3.  Secondary:   Change From Baseline in Height at Month 6 and 12   [ Time Frame: Baseline, Month 6, Month 12 ]

4.  Secondary:   Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12   [ Time Frame: Baseline, Month 6, Month 12 ]

5.  Secondary:   Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12   [ Time Frame: Baseline, Month 6, Month 12 ]

6.  Secondary:   Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12   [ Time Frame: Baseline, Month 6, Month 12 ]

7.  Secondary:   Percentage of Adherence to Study Treatment   [ Time Frame: 6 months post-dose (Saizen Test Group and Saizen Control Group); 12 months post-dose (Saizen Test Group) ]

8.  Secondary:   Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs   [ Time Frame: Baseline up to Month 13 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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:  
Name/Title: Merck KGaA Communication Center
Organization: Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany
phone: +49-6151-72-5200
e-mail: service@merckgroup.com



Responsible Party: Merck KGaA
ClinicalTrials.gov Identifier: NCT01746862     History of Changes
Other Study ID Numbers: EMR200104-533
Study First Received: November 30, 2012
Results First Received: August 24, 2016
Last Updated: August 24, 2016
Health Authority: Korea: Food and Drug Administration
Korea: Institutional Review Board