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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:
ClinicalTrials.gov Identifier:
NCT00125190
First Posted: July 29, 2005
Last Update Posted: June 21, 2012
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Ipsen
  Purpose
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.

Condition Intervention Phase
Insulin-Like Growth Factor-1 Deficiency Growth Disorders Drug: rhIGF-1 (mecasermin) for a period of 86 weeks Phase 2 Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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-Controlled Study

Resource links provided by NLM:


Further study details as provided by Ipsen:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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 ]

Enrollment: 45
Study Start Date: July 2005
Study Completion Date: January 2009
Primary Completion Date: January 2009 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: rhIGF-1 (mecasermin) for a period of 86 weeks
    once a day rhIGF-1 injections
Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   3 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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): NCT00125190


Locations
United States, California
Ipsen
Brisbane, California, United States, 94005
Sponsors and Collaborators
Ipsen
Investigators
Study Director: Ipsen Study Director, M.D. Ipsen
  More Information

Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT00125190     History of Changes
Other Study ID Numbers: MS308
First Submitted: July 27, 2005
First Posted: July 29, 2005
Results First Submitted: March 23, 2010
Results First Posted: April 15, 2010
Last Update Posted: June 21, 2012
Last Verified: June 2012

Keywords provided by Ipsen:
Primary IGF-1 Deficiency
IGF-1

Additional relevant MeSH terms:
Dwarfism
Growth Disorders
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases
Pathologic Processes
Mitogens
Insulin, Globin Zinc
Insulin
Mecasermin
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Hypoglycemic Agents
Physiological Effects of Drugs
Growth Substances