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

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00351221

Enrollment

Registered

2006-07-12

Start date

Unknown

Completion date

Unknown

Last updated

2007-03-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Noonan Syndrome

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.

Interventions

DRUGrhIGF-1/rhIGFBP-3

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to 16 Years

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026