Long-Term Treatment With rhIGF-1 in GHIS

Ipsen92 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Growth Hormone Insensitivity Syndrome

Interventions

mecaserminDRUG

injections BID of rhIGF-1, mecasermin

Eligibility

Sex: ALLMin age: 2 YearsMax age: 21 Years

Medical Language ↔ Plain English

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

Locations (1)

Ipsen

Brisbane, California, United States

Outcomes

Primary Outcomes

Annualized Height Velocity Up to 12 Years

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

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.

Time frame: Baseline (Pre-dose) and 1 year

Secondary Outcomes

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.

Time frame: 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.

Time frame: 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.

Time frame: Baseline (Pre-dose) and up to 19 years

Data from ClinicalTrials.gov

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