Long-Term Treatment With rhIGF-1 in GHIS

Phase 2

Completed

• Conditions: Growth Hormone Insensitivity Syndrome
• Interventions: Drug: mecasermin

• Registration Number: NCT00571727
• Lead Sponsor: Ipsen

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).

Detailed Description

Not available

Study Timeline

• Study Start: 1991-05-20
• Study First Submitted: 2007-12-10
• Study First Submitted QC: 2007-12-10
• Study First Posted: 2007-12-12
• Primary Completion: 2011-12-15
• Study Completion: 2011-12-15
• Results First Submitted: 2023-04-25
• Status Verified: 2023-07
• Results First Submitted QC: 2023-07-04
• Last Update Submitted: 2023-07-04
• Results First Posted: 2023-07-06
• Last Update Posted: 2023-07-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 92

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
mecasermin, injections BID of rhIGF-1	mecasermin	-

Primary Outcome Measures

Name	Time	Method
Annualized Height Velocity Up to 12 Years	Baseline (Pre-dose) and 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	Baseline (Pre-dose) and 1 year	Height measurements were performed using wall-mounted stadiometers for analysis of growth data.

Secondary Outcome Measures

Name	Time	Method
Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height	Baseline (Pre-dose) and up to 19 years	Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Height Velocity Standard Deviation Score Up to 12 Years	Baseline (Pre-dose) and 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	Baseline (Pre-dose) and 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.

Trial Locations

Locations (1)

Ipsen; 🇺🇸 Brisbane, California, United States