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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Recombinant Human Insulin-Like Growth Factor-1 (IGF-1) Treatment of Children With Growth Failure Associated With Primary IGF-1 Deficiency: An Open-Label, Multi-Center, Ext

Brief Summary: This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and adolescents treated for primary IGF-1 deficiency (IGFD). The secondary objective is to use the data collected to learn more about the relationship of IGF-1 exposure to the promotion of normal growth and pubertal development.

Detailed Summary:

Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, subjects that have completed one year of mecasermin treatment on Tercica protocol MS301 (NCT00125164) will be allowed to enroll in this extension study. All subjects were planned to receive treatment.

This is a Phase IIIb open-label, multi-center, parallel dose, extension study conducted in approximately 40 centers across the United States.


Sponsor: Ipsen

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

Height to be measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Reposition subject between each measurement.


Original Primary Outcome: Height velocity during the second year of rhIGF-1 treatment

Current Secondary Outcome:

  • Height Velocities During Subsequent Years of rh IGF-1 Treatment [ Time Frame: after 2, 3 and 5 years of treatment ]
    Height to be measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Reposition subject between each measurement.
  • Height Velocity Standard Deviation (SD) Score [ Time Frame: during the course of the study ]
  • Height SD Score [ Time Frame: during the course of the study ]


Original Secondary Outcome:

  • Height velocities during subsequent years of rhIGF-1 treatment
  • Height Velocity Standard Deviation (SD) Score
  • Height SD Score


Information By: Ipsen

Dates:
Date Received: May 26, 2006
Date Started: November 2005
Date Completion:
Last Updated: May 31, 2011
Last Verified: May 2011