NCT00351221

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.

Trial Health

35
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_2

Geographic Reach
1 country

2 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 11, 2006

Completed
1 day until next milestone

First Posted

Study publicly available on registry

July 12, 2006

Completed
Last Updated

March 30, 2007

Status Verified

February 1, 2007

First QC Date

July 11, 2006

Last Update Submit

March 29, 2007

Conditions

Interventions

Eligibility Criteria

Age2 Years - 16 Years
Sexall
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • Clinically significant diseases
  • Chronic illnesses
  • Prior treatment with rhIGF-1

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Schneider Children's Hospital

New Hyde Park, New York, United States

Location

Columbus Children's Hospital

Columbus, Ohio, 43205, United States

Location

MeSH Terms

Conditions

Noonan Syndrome

Condition Hierarchy (Ancestors)

Craniofacial AbnormalitiesMusculoskeletal AbnormalitiesMusculoskeletal DiseasesHeart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesConnective Tissue DiseasesSkin and Connective Tissue Diseases

Study Officials

  • Kenneth Attie, MD

    Insmed, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

July 11, 2006

First Posted

July 12, 2006

Last Updated

March 30, 2007

Record last verified: 2007-02

Locations