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

Insmed

Status and phase: Terminated; Phase 2

Conditions: Noonan Syndrome

Treatments: Drug: rhIGF-1/rhIGFBP-3

Study type: Interventional

Funder types: Industry

Identifiers

NCT00351221

INMS-110-801

Details and patient eligibility

About

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.

Sex: All

Ages: 2 to 16 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)