Back to resultsResearch Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
Primary Purpose
Noonan Syndrome
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
rhIGF-1/rhIGFBP-3
Sponsored by
About this trial
This is an interventional treatment trial for Noonan Syndrome
Eligibility Criteria
Inclusion Criteria: A diagnosis of Noonan syndrome Height less than the 3rd percentile for age and sex (height SDS < -1.88) Basal IGF-I less than the mean for age and sex (IGF-I SDS < 0) Chronological age greater than 2 years Bone age ≤ 11 years for boys, and ≤ 10 years for girls Pre-pubertal Documented pre-treatment height velocity less than the mean for age and sex Exclusion Criteria: Clinically significant diseases Chronic illnesses Prior treatment with rhIGF-1
Sites / Locations
- Schneider Children's Hospital
- Columbus Children's Hospital
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00351221
Brief Title
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
Official Title
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
Interventional
2. Study Status
Record Verification Date
February 2007
Overall Recruitment Status
Terminated
Why Stopped
Terminated due to a patent legal settlement
Study Start Date
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Primary Completion Date
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Study Completion Date
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3. Sponsor/Collaborators
Name of the Sponsor
Insmed Incorporated
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Noonan Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
24 (false)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
rhIGF-1/rhIGFBP-3
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
16 Years
Eligibility Criteria
Inclusion Criteria:
A diagnosis of Noonan syndrome
Height less than the 3rd percentile for age and sex (height SDS < -1.88)
Basal IGF-I less than the mean for age and sex (IGF-I SDS < 0)
Chronological age greater than 2 years
Bone age ≤ 11 years for boys, and ≤ 10 years for girls
Pre-pubertal
Documented pre-treatment height velocity less than the mean for age and sex
Exclusion Criteria:
Clinically significant diseases
Chronic illnesses
Prior treatment with rhIGF-1
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kenneth Attie, MD
Organizational Affiliation
Insmed, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Schneider Children's Hospital
City
New Hyde Park
State/Province
New York
Country
United States
Facility Name
Columbus Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
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