IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome

NCT ID: NCT00368173

Last Updated: 2012-09-13

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Study Classification

INTERVENTIONAL

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

STUDY OBJECTIVE

To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with GHIS.

STUDY DESIGN

This study is an open-label, multi-center clinical trial to evaluate the safety and effectiveness of rhIGF-I/rhIGFBP-3 to increase rate of growth when administered once daily for 12 months in children and adolescents with growth hormone insensitivity syndrome (GHIS) such as Laron Syndrome. At the end of the initial twelve-month treatment period, additional safety and long-term efficacy data will be assessed in a second 12 month treatment period.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Growth Hormone Insensitivity Syndrome (GHIS) Laron Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

rhIGF-I/rhIGFBP-3

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. A diagnosis of GHIS such as Laron syndrome,
2. 2 - 18 years of age,
3. Height less than or equal to -3SD for age,
4. Pre-pubertal, defined as Tanner breast stage 1 or testis volume \<4mL

Exclusion Criteria

1. Children in puberty,
2. Diagnosed malignancy,
3. A diagnosis of diabetes mellitus
Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Insmed Incorporated

INDUSTRY

Sponsor Role lead

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Robert Rapaport, MD

New York, New York, United States

Site Status

Dr. Alicia Belgorosky

Buenos Aires, , Argentina

Site Status

Dr. Christina Bazan

San Miguel de Tucumán, , Argentina

Site Status

Dr. Bruce King

Newcastle, , Australia

Site Status

Dr. Durval Damiani

São Paulo, , Brazil

Site Status

Dr. Kwok-leung NG

Hong Kong, , China

Site Status

Dr. Mohamed EL Kholy

Cairo, , Egypt

Site Status

Professor Annette Greuters

Berlin, , Germany

Site Status

Tiosano Dov

Haifa, , Israel

Site Status

Maria Carolina Salerno

Naples, , Italy

Site Status

Dr. Hilde Bjorndalen

Oslo, , Norway

Site Status

Dr. Carlos del Aguila

Lima, , Peru

Site Status

Dr. Magdalena Paskova

Košice, , Slovakia

Site Status

Professor Nursen Yordam

Ankara, , Turkey (Türkiye)

Site Status

Dr. Cecilia Camacho-Hubner

London, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Argentina Australia Brazil China Egypt Germany Israel Italy Norway Peru Slovakia Turkey (Türkiye) United Kingdom

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

INSM-110-303

Identifier Type: -

Identifier Source: org_study_id