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

Phase 2

Completed

• Conditions: Growth Hormone Insensitivity Syndrome (GHIS); Laron Syndrome

• Registration Number: NCT00368173
• Lead Sponsor: Insmed Incorporated

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2006-08-23
• Study First Submitted QC: 2006-08-23
• Study First Posted: 2006-08-24
• Status Verified: 2012-09
• Last Update Submitted: 2012-09-11
• Last Update Posted: 2012-09-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. A diagnosis of GHIS such as Laron syndrome,
2. 2 - 18 years of age,
3. Height less than or equal to -3SD for age,
4. Pre-pubertal, defined as Tanner breast stage 1 or testis volume <4mL

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Exclusion Criteria

1. Children in puberty,
2. Diagnosed malignancy,
3. A diagnosis of diabetes mellitus

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Trial Locations

Locations (15)

Maria Carolina Salerno; 🇮🇹 Naples, Italy

Dr. Durval Damiani; 🇧🇷 Sao Paulo, Brazil

Dr. Bruce King; 🇦🇺 Newcastle, Australia

Dr. Alicia Belgorosky; 🇦🇷 Buenos Aires, Argentina

Dr. Kwok-leung NG; 🇨🇳 Hong Kong, China

Robert Rapaport, MD; 🇺🇸 New York, New York, United States

Professor Annette Greuters; 🇩🇪 Berlin, Germany

Dr. Christina Bazan; 🇦🇷 Tucuman, Argentina

Professor Nursen Yordam; 🇹🇷 Ankara, Turkey

Tiosano Dov; 🇮🇱 Haifa, Israel

Dr. Cecilia Camacho-Hubner; 🇬🇧 London, United Kingdom

Dr. Magdalena Paskova; 🇸🇰 Kosice, Slovakia

Dr. Hilde Bjorndalen; 🇳🇴 Oslo, Norway

Dr. Carlos del Aguila; 🇵🇪 Lima, Peru

Dr. Mohamed EL Kholy; 🇪🇬 Cairo, Egypt