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IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome

Primary Purpose

Growth Hormone Insensitivity Syndrome (GHIS), Laron Syndrome

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
rhIGF-I/rhIGFBP-3
Sponsored by
Insmed Incorporated
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Growth Hormone Insensitivity Syndrome (GHIS)

Eligibility Criteria

2 Years - 18 Years (Child, Adult)All Sexes

Inclusion Criteria: A diagnosis of GHIS such as Laron syndrome, 2 - 18 years of age, Height less than or equal to -3SD for age, Pre-pubertal, defined as Tanner breast stage 1 or testis volume <4mL Exclusion Criteria: Children in puberty, Diagnosed malignancy, A diagnosis of diabetes mellitus

Sites / Locations

  • Robert Rapaport, MD
  • Dr. Alicia Belgorosky
  • Dr. Christina Bazan
  • Dr. Bruce King
  • Dr. Durval Damiani
  • Dr. Kwok-leung NG
  • Dr. Mohamed EL Kholy
  • Professor Annette Greuters
  • Tiosano Dov
  • Maria Carolina Salerno
  • Dr. Hilde Bjorndalen
  • Dr. Carlos del Aguila
  • Dr. Magdalena Paskova
  • Professor Nursen Yordam
  • Dr. Cecilia Camacho-Hubner

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
August 23, 2006
Last Updated
September 11, 2012
Sponsor
Insmed Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT00368173
Brief Title
IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
September 2012
Overall Recruitment Status
Completed
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor
Insmed Incorporated

4. Oversight

5. Study Description

Brief Summary
STUDY OBJECTIVE To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with GHIS. STUDY DESIGN This study is an open-label, multi-center clinical trial to evaluate the safety and effectiveness of rhIGF-I/rhIGFBP-3 to increase rate of growth when administered once daily for 12 months in children and adolescents with growth hormone insensitivity syndrome (GHIS) such as Laron Syndrome. At the end of the initial twelve-month treatment period, additional safety and long-term efficacy data will be assessed in a second 12 month treatment period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormone Insensitivity Syndrome (GHIS), Laron Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
rhIGF-I/rhIGFBP-3

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
18 Years
Eligibility Criteria
Inclusion Criteria: A diagnosis of GHIS such as Laron syndrome, 2 - 18 years of age, Height less than or equal to -3SD for age, Pre-pubertal, defined as Tanner breast stage 1 or testis volume <4mL Exclusion Criteria: Children in puberty, Diagnosed malignancy, A diagnosis of diabetes mellitus
Facility Information:
Facility Name
Robert Rapaport, MD
City
New York
State/Province
New York
Country
United States
Facility Name
Dr. Alicia Belgorosky
City
Buenos Aires
Country
Argentina
Facility Name
Dr. Christina Bazan
City
Tucuman
Country
Argentina
Facility Name
Dr. Bruce King
City
Newcastle
Country
Australia
Facility Name
Dr. Durval Damiani
City
Sao Paulo
Country
Brazil
Facility Name
Dr. Kwok-leung NG
City
Hong Kong
Country
China
Facility Name
Dr. Mohamed EL Kholy
City
Cairo
Country
Egypt
Facility Name
Professor Annette Greuters
City
Berlin
Country
Germany
Facility Name
Tiosano Dov
City
Haifa
Country
Israel
Facility Name
Maria Carolina Salerno
City
Naples
Country
Italy
Facility Name
Dr. Hilde Bjorndalen
City
Oslo
Country
Norway
Facility Name
Dr. Carlos del Aguila
City
Lima
Country
Peru
Facility Name
Dr. Magdalena Paskova
City
Kosice
Country
Slovakia
Facility Name
Professor Nursen Yordam
City
Ankara
Country
Turkey
Facility Name
Dr. Cecilia Camacho-Hubner
City
London
Country
United Kingdom

12. IPD Sharing Statement

Learn more about this trial

IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome

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