Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature - Full Text View

Sponsor:	Pfizer
Information provided by (Responsible Party):	Pfizer
ClinicalTrials.gov Identifier:	NCT00396097

Purpose

To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment. The endpoint at 4 years is to explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment

Condition	Intervention	Phase
Idiopathic Short Stature	Drug: Genotropin	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A Four-Year Open-Label Multi-Center Randomized Two-Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target-Driven Treatment Regimen To Standard Dosing Of Genotropin

Resource links provided by NLM:

Genetics Home Reference related topics: metatropic dysplasia pseudoachondroplasia

MedlinePlus related topics: Dwarfism

Drug Information available for: Somatropin

U.S. FDA Resources

Further study details as provided by Pfizer:

Primary Outcome Measures:

To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment [ Time Frame: 2 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

More uniformly achieve the desired height gain at 24 months with the goal of minimizing the variability between subjects. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
Demonstrate safety of Genotropin treatment with this treatment paradigm [ Time Frame: 4 years ] [ Designated as safety issue: No ]
Identify pharmacogenomic variants related to growth/stature [ Time Frame: 4 years ] [ Designated as safety issue: No ]
Explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment [ Time Frame: 4 years ] [ Designated as safety issue: No ]
Explore maintained treatment efficacy at four years of two formula-based dose regimens (sub-arms) compared to standard treatment [ Time Frame: 4 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	312
Study Start Date:	October 2006
Estimated Study Completion Date:	September 2012
Estimated Primary Completion Date:	September 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: Standard Standard daily HGH treatment	Drug: Genotropin Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.
Active Comparator: Formula-based Formula-based dose regimen	Drug: Genotropin Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.

Eligibility

Ages Eligible for Study:	3 Years to 10 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females
Naive to Growth Hormone treatment

Exclusion Criteria:

Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00396097

Show 42 Study Locations

Sponsors and Collaborators

Pfizer

Investigators

Study Director:

Pfizer CT.gov Call Center

Pfizer

More Information

Additional Information:

To obtain contact information for a study center near you, click here. This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	Pfizer
ClinicalTrials.gov Identifier:	NCT00396097 History of Changes
Other Study ID Numbers:	A6281280
Study First Received:	November 2, 2006
Last Updated:	May 2, 2012
Health Authority:	United States: Food and Drug Administration

Keywords provided by Pfizer:

ISS
human growth hormone

Additional relevant MeSH terms:

Dwarfism
Bone Diseases, Developmental
Bone Diseases

Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases

ClinicalTrials.gov processed this record on May 24, 2012