Long-Term Treatment With rhIGF-1 in GHIS

Growth Hormone Insensitivity Syndrome Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00571727
• Other study ID #: Study 1419
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: May 20, 1991
• Est. completion date: December 15, 2011

Study information

• Verified date: July 2023
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 92
• Est. completion date: December 15, 2011
• Est. primary completion date: December 15, 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• Growth Hormone Insensitivity Syndrome
• Laron Syndrome
• Syndrome

Intervention

Drug:
• mecasermin
injections BID of rhIGF-1, mecasermin

Locations

Country	Name	City	State
United States	Ipsen	Brisbane	California

Sponsors (2)

Lead Sponsor	Collaborator
Ipsen	University of Oklahoma

Country where clinical trial is conducted

United States, 

References & Publications (1)

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 insensitiv — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized Height Velocity Up to 12 Years	Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.	Baseline (Pre-dose) and up to 12 years
Primary	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.	Baseline (Pre-dose) and 1 year
Secondary	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.	Baseline (Pre-dose) and up to 12 years
Secondary	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.	Baseline (Pre-dose) and up to 12 years
Secondary	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.	Baseline (Pre-dose) and up to 19 years