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

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ClinicalTrials.gov Identifier: NCT00330668
Recruitment Status : Terminated (Unacceptable frequency of hypoglycemia observed at and above 200 ug/kg/day)
First Posted : May 29, 2006
Results First Posted : June 27, 2011
Last Update Posted : June 27, 2011
Sponsor:
Information provided by:
Ipsen

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.

Condition or disease Intervention/treatment Phase
Growth Disorders Drug: rh IGF-1 (mecasermin) Phase 3

Detailed Description:

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.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 114 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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, Extension Study
Study Start Date : November 2005
Actual Primary Completion Date : February 2010
Actual Study Completion Date : March 2010



Intervention Details:
  • Drug: rh IGF-1 (mecasermin)
    Patients from untreated arm for prior study MS301 (NCT00125164) were randomized to a dose of either 80 or 120 mcg/kg twice daily. For patients receiving active treatment in previous study MS 301 (NCT00125164), they started on a dose of 80 or 120 mcg/kg twice daily based on the dose reached at end of the previous study. Following a protocol amendment in May 2009, all patients were switched to once daily doses of 160 µg/kg, escalated to a targeted maximum dose of 240 µg/kg.
    Other Name: Increlex


Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

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


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Ages Eligible for Study:   4 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Parents or legally authorized representatives must give signed informed consent before any trial related activities are conducted
  • Where required, assent of the subject will be appropriately documented prior to any study related activities
  • Completion of assessments at Visit 9 (Month 120 of Study MS301 [NCT00125164])

Exclusion Criteria:

  • Incomplete participation in MS301 (NCT00125164)
  • Known or suspected allergy to the trial product (mecasermin, recombinant human IGF-1 injection) or its formulation
  • Development or presence of a chronic condition except as approved by the Medical Monitor
  • Pregnancy
  • Any social or medical condition that, in the opinion of the investigator, would be detrimental to either the subject or the study

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00330668


Locations
France
Ipsen
Paris, France
Sponsors and Collaborators
Ipsen
Investigators
Study Director: Sr Vice President, Clinical Development and Medical Affairs Ipsen (formerly Tercica, Inc.)

Responsible Party: Senior Vice President, Clinical Development and Medical Affairs, Ipsen
ClinicalTrials.gov Identifier: NCT00330668     History of Changes
Other Study ID Numbers: MS306
First Posted: May 29, 2006    Key Record Dates
Results First Posted: June 27, 2011
Last Update Posted: June 27, 2011
Last Verified: May 2011

Keywords provided by Ipsen:
Insulin-like Growth Factor Deficiency
IGF-1
Short Stature

Additional relevant MeSH terms:
Growth Disorders
Failure to Thrive
Pathologic Processes
Signs and Symptoms
Mecasermin
Growth Substances
Physiological Effects of Drugs