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Completed

NCT Number: NCT00571727

Long-Term Treatment With rhIGF-1 in GHIS

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).

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Key information

Conditions

Growth Hormone Insensitivity Syndrome Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dwarfism Endocrine System Diseases Genetic Diseases, Inborn Laron Syndrome Musculoskeletal Diseases

Age range

2 year–21 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Ipsen

Brisbane, California, 94005, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

mecasermin

Drug

injections BID of rhIGF-1, mecasermin

Other names: Increlex

Primary outcomes

  1. Annualized Height Velocity Up to 12 Years

    Time frame: Baseline (Pre-dose) and up to 12 years

    Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.

  2. Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment

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

    Height measurements were performed using wall-mounted stadiometers for analysis of growth data.

Secondary outcomes

  1. Height Velocity Standard Deviation Score Up to 12 Years

    Time frame: 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.

  2. Height Standard Deviation Score Up to 12 Years

    Time frame: 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.

  3. Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height

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

    Height measurements were performed using wall-mounted stadiometers for analysis of growth data.

Sponsors and collaborators

Lead sponsor

Ipsen

Industry

Collaborators

  • University of Oklahoma

Registry information

Official study 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)

Important dates

Study start
1991
Primary completion
2011
Study completion
2011
First posted
Dec 12, 2007
Registry last updated
Jul 6, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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