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

A Phase 2, Open-Label, Multicenter, Clinical Trial to Evaluate the Pharmacokinetics, Safety and Efficacy of Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 in Children With Growth Failure Due to Noonan Syndrome

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.

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