NCT00571727

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
92

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started May 1991

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 20, 1991

Completed
16.6 years until next milestone

First Submitted

Initial submission to the registry

December 10, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 12, 2007

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 15, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 15, 2011

Completed
11.6 years until next milestone

Results Posted

Study results publicly available

July 6, 2023

Completed
Last Updated

July 6, 2023

Status Verified

July 1, 2023

Enrollment Period

20.6 years

First QC Date

December 10, 2007

Results QC Date

April 25, 2023

Last Update Submit

July 4, 2023

Conditions

Growth Hormone Insensitivity Syndrome

Keywords

growthinsulin like growth factorLaron syndrome

Outcome Measures

Primary Outcomes (2)

  • 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 Outcomes (3)

  • Height Velocity Standard Deviation Score Up to 12 Years

    Baseline (Pre-dose) and up to 12 years

  • Height Standard Deviation Score Up to 12 Years

    Baseline (Pre-dose) and up to 12 years

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

    Baseline (Pre-dose) and up to 19 years

Study Arms (1)

mecasermin, injections BID of rhIGF-1

EXPERIMENTAL
Drug: mecasermin

Interventions

mecaserminDRUG

injections BID of rhIGF-1, mecasermin

Also known as: Increlex
mecasermin, injections BID of rhIGF-1

Eligibility Criteria

Age2 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Height \<-2SD for age and gender
  • IGF-1 \<-2SD for age and gender
  • Evidence of GH resistance

You may not qualify if:

  • closed epiphyses
  • prior active malignancy
  • major organ disfunction
  • treatment with medications that would diminish growth
  • clinically significant cardiac abnormalities

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ipsen

Brisbane, California, 94005, United States

Location

Related Publications (1)

  • Chernausek SD, Backeljauw PF, Frane J, Kuntze J, Underwood LE; GH Insensitivity Syndrome Collaborative Group. Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab. 2007 Mar;92(3):902-10. doi: 10.1210/jc.2006-1610. Epub 2006 Dec 27.

    PMID: 17192294RESULT

MeSH Terms

Conditions

Laron Syndrome

Interventions

mecasermin

Condition Hierarchy (Ancestors)

DwarfismBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEndocrine System Diseases

Results Point of Contact

Title
Medical Director
Organization
Ipsen
clinical.trials@ipsen.com
see email

Study Officials

  • Ipsen Medical Director

    Ipsen

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 10, 2007

First Posted

December 12, 2007

Study Start

May 20, 1991

Primary Completion

December 15, 2011

Study Completion

December 15, 2011

Last Updated

July 6, 2023

Results First Posted

July 6, 2023

Record last verified: 2023-07

Locations

US(1)