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

• Status: Terminated
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2

Details

• Lead Sponsor: Insmed Incorporated
• Treatments: Insulin; Insulin, Globin Zinc; Mecasermin; Mitogens

Criteria

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