Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Ipsen
ClinicalTrials.gov Identifier:
NCT00125190
First received: July 27, 2005
Last updated: June 14, 2012
Last verified: June 2012
Results First Received: March 23, 2010  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Insulin-Like Growth Factor-1 Deficiency
Growth Disorders
Intervention: Drug: rhIGF-1 (mecasermin) for a period of 86 weeks

  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
First Subject Screened: 12 January 2005. Last Subject Completed: 14 January 2009. 12 investigators screened subjects, 1 did not enroll any subject. 89 subjects were screened, 41 were ineligible, 3 declined treatment and 45 were treated and analyzed.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Screening consisted of two-staged clinic visits for up to 6 weeks, and included the following evaluations: medical history, complete physical examination, measurements of serum IGF-1 and IGF-1 binding proteins (IGFBP 1, IGFBP 2 and IGFBP-3), GH binding protein, Acid-labile subunit, GH stimulation test.

Reporting Groups
  Description
rhIGF-1 QD During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.

Participant Flow:   Overall Study
    rhIGF-1 QD  
STARTED     45  
COMPLETED     30  
NOT COMPLETED     15  
Adverse Event                 1  
Lost to Follow-up                 4  
Patient/Parent Decision                 7  
other                 1  
Non-compliance                 2  



  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.
No text entered.

Reporting Groups
  Description
rhIGF-1 QD During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.

Baseline Measures
    rhIGF-1 QD  
Number of Participants  
[units: participants]
  45  
Age  
[units: years]
Mean ± Standard Deviation
  8.7  ± 2.8  
Gender  
[units: participants]
 
Female     7  
Male     38  
Race/Ethnicity, Customized  
[units: participants]
 
Black     1  
Hispanic     12  
White     31  
Other     1  
Body Mass Index SD Score  
[units: SDs]
Mean ± Standard Deviation
  -0.4  ± 0.7  
Bone Age  
[units: years]
Mean ± Standard Deviation
  7.2  ± 2.6  
Height SD Score  
[units: SDs]
Mean ± Standard Deviation
  -2.7  ± 0.6  
IGF Binding Protein-3 SD Score  
[units: SDs]
Mean ± Standard Deviation
  -0.7  ± 1.0  
IGF-1 SD Score  
[units: SDs]
Mean ± Standard Deviation
  -2.6  ± 0.5  
Maximum Stimulated GH  
[units: ng/mL]
Mean ± Standard Deviation
  20.5  ± 9.9  
Weight SD Score  
[units: SDs]
Mean ± Standard Deviation
  -2.3  ± 0.7  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Height Velocity Over the Study Period 0 - 34 Weeks [Intent to Treat Population]   [ Time Frame: 34 weeks ]

2.  Primary:   Height Velocity Over the Study Period 34 - 86 Weeks [Intent to Treat Population]   [ Time Frame: Weeks 34 to 86 ]

3.  Secondary:   Changes in Height Standard Deviation (SD) Score Over the Study Period 0 - 34 Weeks [Intent to Treat Population]   [ Time Frame: Weeks 0 - 34 ]

4.  Secondary:   Changes in Height Standard Deviation (SD) Score Over the Study Period 34 - 86 Weeks   [ Time Frame: Weeks 34 - 86 ]

5.  Secondary:   Bone Age - Change From Pretreatment Minus Change in Chronological Age Over the Study Period 0 - 86 Weeks [Intent to Treat Population]   [ Time Frame: Weeks 0 - 86 ]

6.  Secondary:   Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-1 (IGFBP-1) From Baseline to Week 86   [ Time Frame: 86 weeks ]

7.  Secondary:   Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-2 (IGFBP-2) From Baseline to Week 86   [ Time Frame: 86 weeks ]

8.  Secondary:   Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-3 (IGFBP-3) From Baseline to Week 86   [ Time Frame: 86 weeks ]

9.  Secondary:   Percent Changes in Serum Concentration of Acid Labile Subunit (ALS) From Baseline to Week 86   [ Time Frame: 86 weeks ]

10.  Post-Hoc:   Increase in Height Velocity Over the Study Period 0 - 34 Weeks [Completers]   [ Time Frame: Weeks 0 -34 ]

11.  Post-Hoc:   Increase in Height Velocity Over the Study Period 34 - 86 Weeks [Completers]   [ Time Frame: Weeks 34 - 86 ]

12.  Secondary:   rhIGF-1 Doses Required to Achieve the Serum IGF-1 Targets With Measures Taken at Each Study Visit   [ Time Frame: 34, 52 and 86 weeks ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  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
No text entered.


  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Cathryn Clary, M.D.
Organization: Ipsen
phone: +1 650-624-4919
e-mail: cathryn.clary@ipsen.com


No publications provided


Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT00125190     History of Changes
Other Study ID Numbers: MS308
Study First Received: July 27, 2005
Results First Received: March 23, 2010
Last Updated: June 14, 2012
Health Authority: United States: Food and Drug Administration