Back to resultsLong-Term Treatment With rhIGF-1 in GHIS
Primary Purpose
Growth Hormone Insensitivity Syndrome
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
mecasermin
Sponsored by
About this trial
This is an interventional treatment trial for Growth Hormone Insensitivity Syndrome focused on measuring growth, insulin like growth factor, Laron syndrome
Eligibility Criteria
Inclusion Criteria:
- Height <-2SD for age and gender
- IGF-1 <-2SD for age and gender
- Evidence of GH resistance
Exclusion Criteria:
- closed epiphyses
- prior active malignancy
- major organ disfunction
- treatment with medications that would diminish growth
- clinically significant cardiac abnormalities
Sites / Locations
- Ipsen
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
mecasermin, injections BID of rhIGF-1
Arm Description
Outcomes
Primary Outcome Measures
Annualized Height Velocity Up to 12 Years
Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Secondary Outcome Measures
Height Velocity Standard Deviation Score Up to 12 Years
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height velocity-standard deviation score was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference mean height velocity. Greater height velocity standard deviation score indicates better outcome.
Height Standard Deviation Score Up to 12 Years
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height standard deviation score was calculated as height minus reference mean height divided by standard deviation of the reference mean height. A higher height standard deviation score indicates a better outcome.
Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Full Information
NCT ID
NCT00571727
First Posted
December 10, 2007
Last Updated
July 4, 2023
Sponsor
Ipsen
Collaborators
University of Oklahoma
1. Study Identification
Unique Protocol Identification Number
NCT00571727
Brief Title
Long-Term Treatment With rhIGF-1 in GHIS
Official Title
A Study of Long-Term Human Recombinant Insulin-Like Growth Factor-1 (rhIGF-1) in Children With Short Stature Due to Growth Hormone Insensitivity Syndrome (GHIS)
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
May 20, 1991 (Actual)
Primary Completion Date
December 15, 2011 (Actual)
Study Completion Date
December 15, 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ipsen
Collaborators
University of Oklahoma
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
5. Study Description
Brief Summary
The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormone Insensitivity Syndrome
Keywords
growth, insulin like growth factor, Laron syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
92 (Actual)
8. Arms, Groups, and Interventions
Arm Title
mecasermin, injections BID of rhIGF-1
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
mecasermin
Other Intervention Name(s)
Increlex
Intervention Description
injections BID of rhIGF-1, mecasermin
Primary Outcome Measure Information:
Title
Annualized Height Velocity Up to 12 Years
Description
Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Time Frame
Baseline (Pre-dose) and up to 12 years
Title
Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment
Description
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Time Frame
Baseline (Pre-dose) and 1 year
Secondary Outcome Measure Information:
Title
Height Velocity Standard Deviation Score Up to 12 Years
Description
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height velocity-standard deviation score was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference mean height velocity. Greater height velocity standard deviation score indicates better outcome.
Time Frame
Baseline (Pre-dose) and up to 12 years
Title
Height Standard Deviation Score Up to 12 Years
Description
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height standard deviation score was calculated as height minus reference mean height divided by standard deviation of the reference mean height. A higher height standard deviation score indicates a better outcome.
Time Frame
Baseline (Pre-dose) and up to 12 years
Title
Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height
Description
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Time Frame
Baseline (Pre-dose) and up to 19 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Height <-2SD for age and gender
IGF-1 <-2SD for age and gender
Evidence of GH resistance
Exclusion Criteria:
closed epiphyses
prior active malignancy
major organ disfunction
treatment with medications that would diminish growth
clinically significant cardiac abnormalities
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ipsen Medical Director
Organizational Affiliation
Ipsen
Official's Role
Study Director
Facility Information:
Facility Name
Ipsen
City
Brisbane
State/Province
California
ZIP/Postal Code
94005
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
17192294
Citation
Chernausek SD, Backeljauw PF, Frane J, Kuntze J, Underwood LE; GH Insensitivity Syndrome Collaborative Group. Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab. 2007 Mar;92(3):902-10. doi: 10.1210/jc.2006-1610. Epub 2006 Dec 27.
Results Reference
result
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Long-Term Treatment With rhIGF-1 in GHIS
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