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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

July 27, 2005
June 14, 2012
July 2005
January 2009   (final data collection date for primary outcome measure)
  • Height Velocity Over the Study Period 0 - 34 Weeks [Intent to Treat Population] [ Time Frame: 34 weeks ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.
Increase in height velocity over the study period
Complete list of historical versions of study NCT00125190 on ClinicalTrials.gov Archive Site
  • Changes in Height Standard Deviation (SD) Score Over the Study Period 0 - 34 Weeks [Intent to Treat Population] [ Time Frame: Weeks 0 - 34 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
  • 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
Not Provided
Not Provided
 
Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency
Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With Primary IGF-1 Deficiency: A Multi-Center, Open Label, Concentration-Controlled Study

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.

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.

Interventional
Phase 2
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Insulin-Like Growth Factor-1 Deficiency
  • Growth Disorders
Drug: rhIGF-1 (mecasermin) for a period of 86 weeks
once a day rhIGF-1 injections
Not Provided
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
45
January 2009
January 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Chronological age ≥ 3
  • Chronological age or bone age ≤ 12 for boys and ≤ 11 for girls
  • Prepubertal at Visit 1
  • Height SD score of < -2
  • IGF-1 SD score of < -2

Exclusion Criteria:

  • Prior treatment with GH, IGF-1, or other growth-influencing medications
  • Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)
  • Chronic illness such as diabetes, cystic fibrosis, etc.
Both
3 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00125190
MS308
Yes
Ipsen
Ipsen
Not Provided
Study Director: Ipsen Study Director, M.D. Ipsen
Ipsen
June 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP