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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Pfizer
ClinicalTrials.gov Identifier:
NCT00396097
First received: November 2, 2006
Last updated: December 20, 2013
Last verified: December 2013
  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:


Further study details as provided by Pfizer:

Primary Outcome Measures:
  • Absolute On-target Difference (AOTD) at 24 Months [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).


Secondary Outcome Measures:
  • Variability of Height SDS at 24 Months [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.

  • Time Cost (Months Until >= -2 SDS) [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, >= -2SDS).

  • Computed Cost of Height Gain at 48 Months [ Time Frame: 4 years ] [ Designated as safety issue: No ]
    The computed cost of height gain was defined as the amount of drug used relative to the observed height-gain, in terms of mg/cm, this was calculated at Month 48.

  • Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months [ Time Frame: 4 years ] [ Designated as safety issue: No ]
    The estimated cost of long-term height gain until FAH was calculated.

  • Change From Baseline in Height SDS at 48 Months. [ Time Frame: 4 years ] [ Designated as safety issue: No ]
    Change in height SDS was measured at 48 months.


Enrollment: 316
Study Start Date: December 2006
Study Completion Date: August 2012
Primary Completion Date: August 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
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: NCT00396097

  Show 42 Study Locations
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer
  More Information

Additional Information:
No publications provided

Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT00396097     History of Changes
Other Study ID Numbers: A6281280
Study First Received: November 2, 2006
Results First Received: August 30, 2013
Last Updated: December 20, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Pfizer:
ISS
human growth hormone

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

ClinicalTrials.gov processed this record on October 28, 2014