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

Completed · Phase 2/Phase 3

Conditions studied: Growth Hormone Insensitivity Syndrome (GHIS), Laron Syndrome

In brief

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.

Key facts

Study ID

NCT00368173

Run by

Insmed Incorporated

Last updated by the study team

2012-09-13

Who can join

Age: 2 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Children in puberty,
• Diagnosed malignancy,
• A diagnosis of diabetes mellitus

Where it is running

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

Full record on ClinicalTrials.gov

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