Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature
- Registration Number
- NCT00396097
- Lead Sponsor
- Pfizer
- Brief Summary
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
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 316
- 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
- Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Standard Genotropin Standard daily HGH treatment Formula-based Genotropin Formula-based dose regimen
- Primary Outcome Measures
Name Time Method Absolute On-target Difference (AOTD) at 24 Months 2 years 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
Name Time Method Time Cost (Months Until >= -2 SDS) 2 years Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, \>= -2SDS).
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months 4 years The estimated cost of long-term height gain until FAH was calculated.
Variability of Height SDS at 24 Months 2 years The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.
Computed Cost of Height Gain at 48 Months 4 years 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.
Change From Baseline in Height SDS at 48 Months. 4 years Change in height SDS was measured at 48 months.
Trial Locations
- Locations (42)
Central Ohio Pediatric Endocrinology/Diabetes Services (COPEDS)
🇺🇸Columbus, Ohio, United States
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
Cooks Children Medical Center/Dept. of Pediatric Endocrinology
🇺🇸Fort Worth, Texas, United States
Baystate Medical Center Children's Hospital
🇺🇸Springfield, Massachusetts, United States
Mount Sinai School of Medicine
🇺🇸New York, New York, United States
Johns Hopkins Pediatric Endocrinology
🇺🇸Baltimore, Maryland, United States
Nancy Wright, MD
🇺🇸Tallahassee, Florida, United States
Winthrop University Hospital
🇺🇸Mineola, New York, United States
Children's Endocrine Care of St. Louis, LLC
🇺🇸St. Louis, Missouri, United States
Joe DiMaggio Children's Hospital
🇺🇸Hollywood, Florida, United States
Pediatric Endocrine Associates
🇺🇸Greenwood Village, Colorado, United States
The Research Institute at Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Medical Center
🇺🇸Dallas, Texas, United States
Memorial Pediatric Specialty Clinic
🇺🇸Colorado Springs, Colorado, United States
University of Maryland Baltimore
🇺🇸Baltimore, Maryland, United States
Maimonides Medical Center
🇺🇸Brooklyn, New York, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Morristown Memorial Hospital
🇺🇸Morristown, New Jersey, United States
Diabetes & Endocrine Center for Children & Young Adults at Phelps Memorial Hospital
🇺🇸Sleepy Hollow, New York, United States
Pediatric Alliance, PC
🇺🇸Pittsburgh, Pennsylvania, United States
The Endocrine Clinic P.C.
🇺🇸Memphis, Tennessee, United States
Ellen Sher, MD PA
🇺🇸Dallas, Texas, United States
Women & Children's Hospital of Buffalo
🇺🇸Buffalo, New York, United States
Children's Hospital at Montefiore
🇺🇸Bronx, New York, United States
Pediatric Endocrine Associates at The Longmont Clinic
🇺🇸Longmont, Colorado, United States
University of Iowa Children's Hospital
🇺🇸Iowa City, Iowa, United States
Saint Barnabas Ambulatory Care Center
🇺🇸Livingston, New Jersey, United States
The Steven and Alexandra Cohen Children's Medical Center of New York (CCMC)
🇺🇸Lake Success, New York, United States
Nemours Children's Clinic
🇺🇸Orlando, Florida, United States
Children's Hospital
🇺🇸Boston, Massachusetts, United States
Vanderbilt University
🇺🇸Nashville, Tennessee, United States
Rady Children's Hospital - San Diego
🇺🇸San Diego, California, United States
Pediatric Endocrinology of San Diego Medical Group Incorporated
🇺🇸San Diego, California, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Childrens Hospital Colorado
🇺🇸Aurora, Colorado, United States
Pediatric Endocrinology Associates
🇺🇸Miami, Florida, United States
Arnold Palmer Hospital for Children
🇺🇸Orlando, Florida, United States
Miller School Of Medicine, University of Miami/Jackson Memorial Medical Center
🇺🇸Miami, Florida, United States
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Thomas Jefferson University
🇺🇸Philadelphia, Pennsylvania, United States