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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Brief Summary

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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.

Detailed Description

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Conditions

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Noonan Syndrome

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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rhIGF-1/rhIGFBP-3

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

2 Years

Maximum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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Kenneth Attie, MD

Role: STUDY_DIRECTOR

Insmed, Inc.

Locations

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Schneider Children's Hospital

New Hyde Park, New York, United States

Site Status

Columbus Children's Hospital

Columbus, Ohio, United States

Site Status

Countries

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United States

Other Identifiers

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INMS-110-801

Identifier Type: -

Identifier Source: org_study_id