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.

Sponsored by:	Insmed
Information provided by:	Insmed
ClinicalTrials.gov Identifier:	NCT00351221

Purpose

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 II

Genetics Home Reference related topics: Baller-Gerold syndrome Crouzon syndrome Noonan syndrome

Drug Information available for: Insulin Insulin-like growth factor I Mecasermin rinfabate Mecasermin

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Active Control, Single Group Assignment
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

Further study details as provided by Insmed:

Estimated Enrollment:

Eligibility

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

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00351221

Locations

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

Investigators

Study Director:

Kenneth Attie, MD

Insmed, Inc.

More Information

Study ID Numbers:	INMS-110-801
Study First Received:	July 11, 2006
Last Updated:	March 29, 2007
ClinicalTrials.gov Identifier:	NCT00351221
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Turner-like syndrome
Musculoskeletal Diseases
Craniofacial Abnormalities
Connective Tissue Diseases
Noonan Syndrome

Pseudo-Turner syndrome
Noonan syndrome
Congenital Abnormalities
Insulin
Musculoskeletal Abnormalities

Additional relevant MeSH terms:

Hypoglycemic Agents
Pathologic Processes
Disease

Syndrome
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009