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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With Primary IGF-1 Deficiency: A Multi-Center, Open Label, Concentration-Contr

Brief Summary: This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.

Detailed Summary:

Growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in growth hormone (GH) action. In this protocol, primary IGFD is defined as short stature (<-2 standard deviations [SDs] below the mean for age and gender), and abnormal serum IGF-1 (<-2 SDs below the mean for age and gender).

The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.

Current Primary Outcome:

• Height Velocity Over the Study Period 0 - 34 Weeks [Intent to Treat Population] [ Time Frame: 34 weeks ]
  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 Over the Study Period 34 - 86 Weeks [Intent to Treat Population] [ Time Frame: Weeks 34 to 86 ]
  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: Increase in height velocity over the study period

Current Secondary Outcome:

• Changes in Height Standard Deviation (SD) Score Over the Study Period 0 - 34 Weeks [Intent to Treat Population] [ Time Frame: Weeks 0 - 34 ]
  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. Please note that Standard Deviation (SD) Score is a term used in growth studies. The SD Score is calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child.
• Changes in Height Standard Deviation (SD) Score Over the Study Period 34 - 86 Weeks [ Time Frame: Weeks 34 - 86 ]
  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. Please note that Standard Deviation (SD) Score is a term used in growth studies. The SD Score is calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child.
• 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 ]
  Plain X-rays of the left hand and wrist exposed for bone age appraisal. The films are sent to a central facility for standardized evaluation.
• Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-1 (IGFBP-1) From Baseline to Week 86 [ Time Frame: 86 weeks ]
  Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-1 in the growth factor panel.
• Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-2 (IGFBP-2) From Baseline to Week 86 [ Time Frame: 86 weeks ]
  Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-2 in the growth factor panel.
• Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-3 (IGFBP-3) From Baseline to Week 86 [ Time Frame: 86 weeks ]
  Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-3 in the growth factor panel.
• Percent Changes in Serum Concentration of Acid Labile Subunit (ALS) From Baseline to Week 86 [ Time Frame: 86 weeks ]
  Blood sample was collected while subject is in a fasting state for measuring the level of Serum Concentration of Acid Labile Subunit (ALS).
• 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 ]

Original Secondary Outcome:

• Changes in serum concentration of insulin-like growth factor binding-1 (IGFBP-1, IGFBP-2, IGFBP-3) and acid labile subunit (ALS)
• Changes in height SD score over one year
• Changes in bone age over one year
• rhIGF-1 Doses Required to Achieve the Serum IGF-1 Targets With Measures Taken at Each Study Visit

Dates:
Date Received: July 27, 2005
Date Started: July 2005
Date Completion:
Last Updated: June 14, 2012
Last Verified: June 2012