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

This study has been completed.
Sponsor:
Information provided by:
Insmed
ClinicalTrials.gov Identifier:
NCT00368173
First received: August 23, 2006
Last updated: September 11, 2012
Last verified: September 2012
  Purpose

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.


Condition Intervention Phase
Growth Hormone Insensitivity Syndrome (GHIS)
Laron Syndrome
Drug: rhIGF-I/rhIGFBP-3
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Insmed:

  Eligibility

Ages Eligible for Study:   2 Years to 18 Years
Genders Eligible for Study:   Both
Criteria

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
  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: NCT00368173

Locations
United States, New York
Robert Rapaport, MD
New York, New York, United States
Argentina
Dr. Alicia Belgorosky
Buenos Aires, Argentina
Dr. Christina Bazan
Tucuman, Argentina
Australia
Dr. Bruce King
Newcastle, Australia
Brazil
Dr. Durval Damiani
Sao Paulo, Brazil
China
Dr. Kwok-leung NG
Hong Kong, China
Egypt
Dr. Mohamed EL Kholy
Cairo, Egypt
Germany
Professor Annette Greuters
Berlin, Germany
Israel
Tiosano Dov
Haifa, Israel
Italy
Maria Carolina Salerno
Naples, Italy
Norway
Dr. Hilde Bjorndalen
Oslo, Norway
Peru
Dr. Carlos del Aguila
Lima, Peru
Slovakia
Dr. Magdalena Paskova
Kosice, Slovakia
Turkey
Professor Nursen Yordam
Ankara, Turkey
United Kingdom
Dr. Cecilia Camacho-Hubner
London, United Kingdom
Sponsors and Collaborators
Insmed
  More Information

No publications provided

ClinicalTrials.gov Identifier: NCT00368173     History of Changes
Other Study ID Numbers: INSM-110-303
Study First Received: August 23, 2006
Last Updated: September 11, 2012
Health Authority: United States: Food and Drug Administration
Germany: Federal Institute for Drugs and Medical Devices
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Turkey: Ministry of Health
Israel: Ministry of Health

Additional relevant MeSH terms:
Laron Syndrome
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases

ClinicalTrials.gov processed this record on August 28, 2014