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

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

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

Study Overview

• Status: Completed
• Conditions: Idiopathic Short Stature
• Intervention / Treatment: Drug: Genotropin

• Study Type: Interventional
• Enrollment (Actual): 316
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Alaska
    • Little Rock, Alaska, United States, 72202
      • Arkansas Children's Hospital
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Los Angeles, California, United States, 90027
      • Children's Hospital of Los Angeles
    • San Diego, California, United States, 32123-4282
      • Rady Children's Hospital - San Diego
    • San Diego, California, United States, 92123
      • Pediatric Endocrinology of San Diego Medical Group Incorporated
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Childrens Hospital Colorado
    • Colorado Springs, Colorado, United States, 80909
      • Memorial Pediatric Specialty Clinic
    • Greenwood Village, Colorado, United States, 80111
      • Pediatric Endocrine Associates
    • Longmont, Colorado, United States, 80501
      • Pediatric Endocrine Associates at The Longmont Clinic
  • Florida
    • Hollywood, Florida, United States, 33021
      • Joe DiMaggio Children's Hospital
    • Miami, Florida, United States, 33136
      • Miller School Of Medicine, University of Miami/Jackson Memorial Medical Center
    • Miami, Florida, United States, 33155
      • Pediatric Endocrinology Associates
    • Orlando, Florida, United States, 32806
      • Arnold Palmer Hospital for Children
    • Orlando, Florida, United States, 32806
      • Nemours Children's Clinic
    • Tallahassee, Florida, United States, 32308
      • Nancy Wright, MD
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Children's Hospital
  • Maryland
    • Baltimore, Maryland, United States, 21201
      • University of Maryland Baltimore
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins Pediatric Endocrinology
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
    • Boston, Massachusetts, United States, 02115
      • Children's Hospital
    • Springfield, Massachusetts, United States, 01199
      • Baystate Medical Center Children's Hospital
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Children's Mercy Hospital
    • St. Louis, Missouri, United States, 63141
      • Children's Endocrine Care of St. Louis, LLC
  • New Jersey
    • Livingston, New Jersey, United States, 07039
      • Saint Barnabas Ambulatory Care Center
    • Morristown, New Jersey, United States, 07962
      • Morristown Memorial Hospital
  • New York
    • Bronx, New York, United States, 10467-2490
      • Children's Hospital at Montefiore
    • Brooklyn, New York, United States, 11219
      • Maimonides Medical Center
    • Buffalo, New York, United States, 14222
      • Women & Children's Hospital of Buffalo
    • Lake Success, New York, United States, 11042
      • The Steven and Alexandra Cohen Children's Medical Center of New York (CCMC)
    • Mineola, New York, United States, 11501
      • Winthrop University Hospital
    • New York, New York, United States, 10029-6574
      • Mount Sinai School of Medicine
    • Sleepy Hollow, New York, United States, 10591
      • Diabetes & Endocrine Center for Children & Young Adults at Phelps Memorial Hospital
  • Ohio
    • Columbus, Ohio, United States, 43205
      • The Research Institute at Nationwide Children's Hospital
    • Columbus, Ohio, United States, 43235
      • Central Ohio Pediatric Endocrinology/Diabetes Services (COPEDS)
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Thomas Jefferson University
    • Pittsburgh, Pennsylvania, United States, 15224
      • Children's Hospital of Pittsburgh of UPMC
    • Pittsburgh, Pennsylvania, United States, 15218
      • Pediatric Alliance, PC
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Tennessee
    • Memphis, Tennessee, United States, 38119
      • The Endocrine Clinic P.C.
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt University
  • Texas
    • Dallas, Texas, United States, 75235
      • Children's Medical Center
    • Dallas, Texas, United States, 75230
      • Ellen Sher, MD PA
    • Fort Worth, Texas, United States, 76104
      • Cooks Children Medical Center/Dept. of Pediatric Endocrinology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Absolute On-target Difference (AOTD) at 24 Months	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).	2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Variability of Height SDS at 24 Months	The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.	2 years
Time Cost (Months Until >= -2 SDS)	Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, >= -2SDS).	2 years
Computed Cost of Height Gain at 48 Months	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.	4 years
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months	The estimated cost of long-term height gain until FAH was calculated.	4 years
Change From Baseline in Height SDS at 48 Months.	Change in height SDS was measured at 48 months.	4 years

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

Helpful Links

• To obtain contact information for a study center near you, click here.

Study record dates

Study Major Dates

• Study Start: December 1, 2006
• Primary Completion (Actual): August 1, 2012
• Study Completion (Actual): August 1, 2012

Study Registration Dates

• First Submitted: November 2, 2006
• First Submitted That Met QC Criteria: November 2, 2006
• First Posted (Estimate): November 6, 2006

Study Record Updates

• Last Update Posted (Estimate): April 5, 2016
• Last Update Submitted That Met QC Criteria: March 9, 2016
• Last Verified: March 1, 2016

More Information

Terms related to this study

• Keywords: ISS; human growth hormone
• Additional Relevant MeSH Terms: Endocrine System Diseases; Genetic Diseases, Inborn; Musculoskeletal Diseases; Bone Diseases; Bone Diseases, Developmental; Dwarfism
• Other Study ID Numbers: A6281280