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

This study has been terminated.
(Terminated due to a patent legal settlement)
Information provided by:
Insmed Incorporated Identifier:
First received: July 11, 2006
Last updated: March 29, 2007
Last verified: February 2007

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.

Condition Intervention Phase
Noonan Syndrome
Drug: rhIGF-1/rhIGFBP-3
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Insmed Incorporated:

Estimated Enrollment: 24

Ages Eligible for Study:   2 Years to 16 Years
Genders Eligible for Study:   Both

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00351221

United States, New York
Schneider Children's Hospital
New Hyde Park, New York, United States
United States, Ohio
Columbus Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Insmed Incorporated
Study Director: Kenneth Attie, MD Insmed, Inc.
  More Information

No publications provided Identifier: NCT00351221     History of Changes
Other Study ID Numbers: INMS-110-801
Study First Received: July 11, 2006
Last Updated: March 29, 2007
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Noonan Syndrome
Cardiovascular Abnormalities
Cardiovascular Diseases
Congenital Abnormalities
Connective Tissue Diseases
Craniofacial Abnormalities
Heart Defects, Congenital
Heart Diseases
Musculoskeletal Abnormalities
Musculoskeletal Diseases
Pathologic Processes
Insulin, Globin Zinc
Hypoglycemic Agents
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs processed this record on October 09, 2015