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

Insmed Incorporated

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Conditions

Growth Hormone Insensitivity Syndrome (GHIS)Laron Syndrome

Interventions

rhIGF-I/rhIGFBP-3DRUG

Eligibility

Sex: ALLMin age: 2 YearsMax age: 18 Years

Medical Language ↔ Plain English

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

Locations (15)

Robert Rapaport, MD

New York, New York, United States

Dr. Alicia Belgorosky

Buenos Aires, Argentina

Dr. Christina Bazan

San Miguel de Tucumán, Argentina

Data from ClinicalTrials.gov

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