Long-Term Treatment With rhIGF-1 in GHIS

July 4, 2023 updated by: Ipsen

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)

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

92

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Brisbane, California, United States, 94005
        • Ipsen

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: mecasermin, injections BID of rhIGF-1
Drug: mecasermin
injections BID of rhIGF-1, mecasermin
Other Names:
  • Increlex

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
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
Time Frame: Baseline (Pre-dose) and 1 year
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
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.
Baseline (Pre-dose) and up to 12 years
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.
Baseline (Pre-dose) and up to 12 years
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.
Baseline (Pre-dose) and up to 19 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ipsen

Collaborators

University of Oklahoma

Investigators

  • Study Director: Ipsen Medical Director, Ipsen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 20, 1991

Primary Completion (Actual)

December 15, 2011

Study Completion (Actual)

December 15, 2011

Study Registration Dates

First Submitted

December 10, 2007

First Submitted That Met QC Criteria

December 10, 2007

First Posted (Estimated)

December 12, 2007

Study Record Updates

Last Update Posted (Actual)

July 6, 2023

Last Update Submitted That Met QC Criteria

July 4, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Study 1419

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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