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)

Sponsor:

Information provided by:

Insmed Incorporated

ClinicalTrials.gov Identifier:

NCT00351221

First received: July 11, 2006

Last updated: March 29, 2007

Last verified: February 2007

History of Changes

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 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:

Genetics Home Reference related topics: branchio-oculo-facial syndrome Crouzon syndrome Noonan syndrome

MedlinePlus related topics: Diabetes Medicines

Drug Information available for: Mecasermin

Genetic and Rare Diseases Information Center resources: Children's Interstitial Lung Disease Noonan Syndrome Noonan Syndrome 1 Noonan Syndrome 6

U.S. FDA Resources

Further study details as provided by Insmed Incorporated:


Estimated Enrollment:	24

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

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 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 Incorporated

Investigators


Study Director:	Kenneth Attie, MD	Insmed, Inc.

More Information

No publications provided


ClinicalTrials.gov 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 Syndrome Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Connective Tissue Diseases Craniofacial Abnormalities Disease Heart Defects, Congenital	Heart Diseases Musculoskeletal Abnormalities Musculoskeletal Diseases Pathologic Processes Mitogens Mitosis Modulators Molecular Mechanisms of Pharmacological Action Pharmacologic Actions

ClinicalTrials.gov processed this record on February 27, 2015

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