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

This study is ongoing, but not recruiting participants.

First Received: August 23, 2006 Last Updated: March 29, 2007 History of Changes

Sponsor:	Insmed
Information provided by:	Insmed
ClinicalTrials.gov Identifier:	NCT00368173

Purpose

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.

Condition	Intervention	Phase
Growth Hormone Insensitivity Syndrome (GHIS) Laron Syndrome	Drug: rhIGF-I/rhIGFBP-3	Phase II Phase III

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment

Resource links provided by NLM:

Genetics Home Reference related topics: pseudoachondroplasia

U.S. FDA Resources

Eligibility

Ages Eligible for Study:	2 Years to 18 Years
Genders Eligible for Study:	Both

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00368173

Locations

United States, New York
Robert Rapaport, MD
New York, New York, United States
Argentina
Dr. Christina Bazan
Tucuman, Argentina
Dr. Alicia Belgorosky
Buenos Aires, Argentina
Australia
Dr. Bruce King
Newcastle, Australia
Brazil
Dr. Durval Damiani
Sao Paulo, Brazil
China
Dr. Kwok-leung NG
Hong Kong, China
Egypt
Dr. Mohamed EL Kholy
Cairo, Egypt
Germany
Professor Annette Greuters
Berlin, Germany
Israel
Tiosano Dov
Haifa, Israel
Italy
Maria Carolina Salerno
Naples, Italy
Norway
Dr. Hilde Bjorndalen
Oslo, Norway
Peru
Dr. Carlos del Aguila
Lima, Peru
Slovakia
Dr. Magdalena Paskova
Kosice, Slovakia
Turkey
Professor Nursen Yordam
Ankara, Turkey
United Kingdom
Dr. Cecilia Camacho-Hubner
London, United Kingdom

Sponsors and Collaborators

Insmed

More Information

No publications provided

Study ID Numbers:	INSM-110-303
Study First Received:	August 23, 2006
Last Updated:	March 29, 2007
ClinicalTrials.gov Identifier:	NCT00368173 History of Changes
Health Authority:	United States: Food and Drug Administration; Germany: Federal Institute for Drugs and Medical Devices; United Kingdom: Medicines and Healthcare Products Regulatory Agency; Turkey: Ministry of Health; Israel: Ministry of Health

Additional relevant MeSH terms:

Dwarfism
Pathologic Processes
Disease
Genetic Diseases, Inborn
Musculoskeletal Diseases

Syndrome
Bone Diseases, Developmental
Laron Syndrome
Endocrine System Diseases
Bone Diseases

ClinicalTrials.gov processed this record on November 30, 2009