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

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.

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

Exclusion Criteria:

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