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 2

Terminated

• Conditions: Noonan Syndrome

• Registration Number: NCT00351221
• Lead Sponsor: Insmed Incorporated

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2006-07-11
• Study First Submitted QC: 2006-07-11
• Study First Posted: 2006-07-12
• Status Verified: 2007-02
• Last Update Submitted: 2007-03-29
• Last Update Posted: 2007-03-30

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 24

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Trial Locations

Locations (2)

Columbus Children's Hospital; 🇺🇸 Columbus, Ohio, United States

Schneider Children's Hospital; 🇺🇸 New Hyde Park, New York, United States