Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency - Full Text View

Sponsor:	Ipsen
Information provided by:	Ipsen
ClinicalTrials.gov Identifier:	NCT00125190

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 II Phase III

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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:

Genetics Home Reference related topics: pseudoachondroplasia

MedlinePlus related topics: Diabetes Medicines Dwarfism Growth Disorders

Drug Information available for: Insulin Insulin beef Insulin-like growth factor I Mecasermin Mecasermin rinfabate

U.S. FDA Resources

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 ] [ 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.

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 ] [ 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 ]

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:

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

Ages Eligible for Study:	3 Years and older
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party:	Cathryn Clary MD, Ipsen
ClinicalTrials.gov Identifier:	NCT00125190 History of Changes
Other Study ID Numbers:	MS308
Study First Received:	July 27, 2005
Results First Received:	March 23, 2010
Last Updated:	March 17, 2011
Health Authority:	United States: Food and Drug Administration

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
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Hypoglycemic Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on February 09, 2012