Long-Term Treatment With rhIGF-1 in GHIS

Study Overview
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Brief Summary
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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).

Official Title
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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)

Conditions
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Growth Hormone Insensitivity Syndrome

Intervention / Treatment
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* Drug: mecasermin


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: mecasermin, injections BID of rhIGF-1<br> | Drug: mecasermin<br>* injections BID of rhIGF-1, mecasermin<br>* Other names: Increlex;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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 | 
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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growth, insulin like growth factor, Laron syndrome