Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome

Phase 2TerminatedNCT00351221

Insmed Incorporated24 enrolled

Overview

The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested as a possible cause of short stature seen in Noonan syndrome children. Administration of our investigational product is intended to bypass the abnormalities in the GH-IGF axis, and hopefully improve body growth.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Noonan Syndrome

Interventions

rhIGF-1/rhIGFBP-3DRUG

Eligibility

Sex: ALLMin age: 2 YearsMax age: 16 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. A diagnosis of Noonan syndrome 2. Height less than the 3rd percentile for age and sex (height SDS \< -1.88) 3. Basal IGF-I less than the mean for age and sex (IGF-I SDS \< 0) 4. Chronological age greater than 2 years 5. Bone age ≤ 11 years for boys, and ≤ 10 years for girls 6. Pre-pubertal 7. Documented pre-treatment height velocity less than the mean for age and sex Exclusion Criteria: 1. Clinically significant diseases 2. Chronic illnesses 3. Prior treatment with rhIGF-1

Locations (2)

Schneider Children's Hospital

New Hyde Park, New York, United States

Columbus Children's Hospital

Columbus, Ohio, United States

Data from ClinicalTrials.gov

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