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Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency

This study has been terminated.
(Unacceptable frequency of hypoglycemia observed at and above 200 ug/kg/day)
Sponsor:
Information provided by:
Ipsen
ClinicalTrials.gov Identifier:
NCT00330668
First received: May 26, 2006
Last updated: May 31, 2011
Last verified: May 2011
History of Changes
Results First Received: February 28, 2011  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Growth Disorders
Intervention: Drug: rh IGF-1 (mecasermin)

  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

Study Initiation Date: 21 OCT 2005. Study completion Date: 19 JAN 2010. 44 investigators enrolled 114 subjects.

Patients who completed a prior Tercica study, MS301 (NCT00125164).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients initially received either mecasermin 80 or 120 µg/kg twice daily. Patients who received 80 µg/kg then switched to receive 120 µg/kg. During the once daily period, all patients were first switched to receive mecasermin 160 µg/kg, followed by individual dose-escalation to a targeted maximum dose of 240 µg/kg.

Reporting Groups
  Description
rhIGF-1 Injection All patients entering study began rhIGF-1 twice daily treatment (range from 40 ug/kg to 120 ug/kg). Following protocol Amendment 2, all patients first received 160 ug/kg once daily followed by individual dose-escalation to 240 ug/kg once daily.

Participant Flow:   Overall Study
    rhIGF-1 Injection  
STARTED     114  
COMPLETED     0  
NOT COMPLETED     114  
Adverse Event                 1  
Lost to Follow-up                 9  
Non-Compliance                 5  
Withdrawal by Subject                 20  
Sponsor's decision                 76  
Lack of Efficacy                 1  
Use of exclusionary medication                 1  
Patient started Lupron                 1  



  Baseline Characteristics
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Reporting Groups
  Description
rhIGF-1 Injection All patients entering study began rhIGF-1 twice daily treatment (range from 40 ug/kg to 120 ug/kg). Following protocol Amendment 2, all patients first received 160 ug/kg once daily followed by individual dose-escalation to 240 ug/kg once daily.

Baseline Measures
    rhIGF-1 Injection  
Number of Participants  
[units: participants]
 		  114  
Age  
[units: years]
Mean ± Standard Deviation 		  8.5  ± 2.4  
Gender  
[units: participants]
 		 
Female     32  
Male     82  
Race/Ethnicity, Customized  
[units: Participants]
 		 
Hispanic     6  
White     100  
Black     1  
Asian     4  
Native Hawaiian     1  
Other     2  
Region of Enrollment  
[units: participants]
 		 
United States     114  
Height Standard Deviation (SD) Score [1]
[units: SD score]
Mean ± Standard Deviation 		  -2.4  ± 0.7  
IGF-1 Standard Deviation (SD) Score [1]
[units: SD score]
Mean ± Standard Deviation 		  -0.9  ± 1.4  
Maximum Stimulated Growth Hormone [2]
[units: ng/mL]
Mean ± Standard Deviation 		  19.2  ± 12.9  
Weight  
[units: Kg]
Mean ± Standard Deviation 		  21.8  ± 6.2  
[1] Please note that SD is a standard term used in growth studies and represents Standard Deviations calculated as the patient value minus the mean divided by the standard deviation. Standard Deviation Scores vary depending on the age and sex of the child.
[2] Growth Hormone Stimulation Test results expressed in ng/mL.



  Outcome Measures
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1.  Primary:   Height Velocity in Modified Intent-to-Treat Population (ITT Patients Randomized to 120 Mcg/kg Twice Daily)   [ Time Frame: after one year of treatment ]
  Show Outcome Measure 1

2.  Secondary:   Height Velocities During Subsequent Years of rh IGF-1 Treatment   [ Time Frame: after 2, 3 and 5 years of treatment ]
  Show Outcome Measure 2

3.  Secondary:   Height Velocity Standard Deviation (SD) Score   [ Time Frame: during the course of the study ]
  Show Outcome Measure 3

4.  Secondary:   Height SD Score   [ Time Frame: during the course of the study ]
  Show Outcome Measure 4


  Serious Adverse Events
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  Other Adverse Events
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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 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.


Limitations and Caveats
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
The study was terminated by the sponsor on identification of an unacceptable frequency of hypoglycemia which was reported at doses of ≥200 mcg/kg once daily. Only safety results are presented as efficacy data were not analyzed according to protocol.  


Results Point of Contact:  
Name/Title: Sr Vice President, Clinical Development and Medical Affairs
Organization: Ipsen, US
phone: clinical.trials@ipsen.com
e-mail: clinical.trials@ipsen.com


No publications provided


Responsible Party: Senior Vice President, Clinical Development and Medical Affairs, Ipsen
ClinicalTrials.gov Identifier: NCT00330668     History of Changes
Other Study ID Numbers: MS306
Study First Received: May 26, 2006
Results First Received: February 28, 2011
Last Updated: May 31, 2011
Health Authority: United States: Food and Drug Administration