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

Overview

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.

Full Title of Study: “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”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)

Interventions

  • Drug: rhIGF-1/rhIGFBP-3

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 2 Years

Maximum Age: 16 Years

Investigator Details

  • Lead Sponsor
    • Insmed Incorporated
  • Overall Official(s)
    • Kenneth Attie, MD, Study Director, Insmed, Inc.

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