search
Back to results

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

Primary Purpose

Idiopathic Short Stature

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Genotropin
Genotropin
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Idiopathic Short Stature focused on measuring ISS, human growth hormone

Eligibility Criteria

3 Years - 10 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Arkansas Children's Hospital
  • Arkansas Children's Hospital
  • Children's Hospital of Los Angeles
  • Rady Children's Hospital - San Diego
  • Pediatric Endocrinology of San Diego Medical Group Incorporated
  • Childrens Hospital Colorado
  • Memorial Pediatric Specialty Clinic
  • Pediatric Endocrine Associates
  • Pediatric Endocrine Associates at The Longmont Clinic
  • Joe DiMaggio Children's Hospital
  • Miller School Of Medicine, University of Miami/Jackson Memorial Medical Center
  • Pediatric Endocrinology Associates
  • Arnold Palmer Hospital for Children
  • Nemours Children's Clinic
  • Nancy Wright, MD
  • University of Iowa Children's Hospital
  • University of Maryland Baltimore
  • Johns Hopkins Pediatric Endocrinology
  • Massachusetts General Hospital
  • Children's Hospital
  • Baystate Medical Center Children's Hospital
  • Children's Mercy Hospital
  • Children's Endocrine Care of St. Louis, LLC
  • Saint Barnabas Ambulatory Care Center
  • Morristown Memorial Hospital
  • Children's Hospital at Montefiore
  • Maimonides Medical Center
  • Women & Children's Hospital of Buffalo
  • The Steven and Alexandra Cohen Children's Medical Center of New York (CCMC)
  • Winthrop University Hospital
  • Mount Sinai School of Medicine
  • Diabetes & Endocrine Center for Children & Young Adults at Phelps Memorial Hospital
  • The Research Institute at Nationwide Children's Hospital
  • Central Ohio Pediatric Endocrinology/Diabetes Services (COPEDS)
  • Thomas Jefferson University
  • Pediatric Alliance, PC
  • Children's Hospital of Pittsburgh of UPMC
  • Medical University of South Carolina
  • The Endocrine Clinic P.C.
  • Vanderbilt University
  • Ellen Sher, MD PA
  • Children's Medical Center
  • Cooks Children Medical Center/Dept. of Pediatric Endocrinology

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

Standard

Formula-based

Arm Description

Standard daily HGH treatment

Formula-based dose regimen

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

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.
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).
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.
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.
Change From Baseline in Height SDS at 48 Months.
Change in height SDS was measured at 48 months.

Full Information

First Posted
November 2, 2006
Last Updated
March 9, 2016
Sponsor
Pfizer
search

1. Study Identification

Unique Protocol Identification Number
NCT00396097
Brief Title
Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2016
Overall Recruitment Status
Completed
Study Start Date
December 2006 (undefined)
Primary Completion Date
August 2012 (Actual)
Study Completion Date
August 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Idiopathic Short Stature
Keywords
ISS, human growth hormone

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
316 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Standard
Arm Type
Active Comparator
Arm Description
Standard daily HGH treatment
Arm Title
Formula-based
Arm Type
Active Comparator
Arm Description
Formula-based dose regimen
Intervention Type
Drug
Intervention Name(s)
Genotropin
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Genotropin
Intervention Description
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.
Primary Outcome Measure Information:
Title
Absolute On-target Difference (AOTD) at 24 Months
Description
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).
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Variability of Height SDS at 24 Months
Description
The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.
Time Frame
2 years
Title
Time Cost (Months Until >= -2 SDS)
Description
Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, >= -2SDS).
Time Frame
2 years
Title
Computed Cost of Height Gain at 48 Months
Description
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.
Time Frame
4 years
Title
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months
Description
The estimated cost of long-term height gain until FAH was calculated.
Time Frame
4 years
Title
Change From Baseline in Height SDS at 48 Months.
Description
Change in height SDS was measured at 48 months.
Time Frame
4 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
10 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Alaska
ZIP/Postal Code
72202
Country
United States
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
Children's Hospital of Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Rady Children's Hospital - San Diego
City
San Diego
State/Province
California
ZIP/Postal Code
32123-4282
Country
United States
Facility Name
Pediatric Endocrinology of San Diego Medical Group Incorporated
City
San Diego
State/Province
California
ZIP/Postal Code
92123
Country
United States
Facility Name
Childrens Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Memorial Pediatric Specialty Clinic
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80909
Country
United States
Facility Name
Pediatric Endocrine Associates
City
Greenwood Village
State/Province
Colorado
ZIP/Postal Code
80111
Country
United States
Facility Name
Pediatric Endocrine Associates at The Longmont Clinic
City
Longmont
State/Province
Colorado
ZIP/Postal Code
80501
Country
United States
Facility Name
Joe DiMaggio Children's Hospital
City
Hollywood
State/Province
Florida
ZIP/Postal Code
33021
Country
United States
Facility Name
Miller School Of Medicine, University of Miami/Jackson Memorial Medical Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Pediatric Endocrinology Associates
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Arnold Palmer Hospital for Children
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Nemours Children's Clinic
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Facility Name
Nancy Wright, MD
City
Tallahassee
State/Province
Florida
ZIP/Postal Code
32308
Country
United States
Facility Name
University of Iowa Children's Hospital
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Maryland Baltimore
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Johns Hopkins Pediatric Endocrinology
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Baystate Medical Center Children's Hospital
City
Springfield
State/Province
Massachusetts
ZIP/Postal Code
01199
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Children's Endocrine Care of St. Louis, LLC
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63141
Country
United States
Facility Name
Saint Barnabas Ambulatory Care Center
City
Livingston
State/Province
New Jersey
ZIP/Postal Code
07039
Country
United States
Facility Name
Morristown Memorial Hospital
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07962
Country
United States
Facility Name
Children's Hospital at Montefiore
City
Bronx
State/Province
New York
ZIP/Postal Code
10467-2490
Country
United States
Facility Name
Maimonides Medical Center
City
Brooklyn
State/Province
New York
ZIP/Postal Code
11219
Country
United States
Facility Name
Women & Children's Hospital of Buffalo
City
Buffalo
State/Province
New York
ZIP/Postal Code
14222
Country
United States
Facility Name
The Steven and Alexandra Cohen Children's Medical Center of New York (CCMC)
City
Lake Success
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
Winthrop University Hospital
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
Mount Sinai School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10029-6574
Country
United States
Facility Name
Diabetes & Endocrine Center for Children & Young Adults at Phelps Memorial Hospital
City
Sleepy Hollow
State/Province
New York
ZIP/Postal Code
10591
Country
United States
Facility Name
The Research Institute at Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Central Ohio Pediatric Endocrinology/Diabetes Services (COPEDS)
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43235
Country
United States
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States
Facility Name
Pediatric Alliance, PC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15218
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
The Endocrine Clinic P.C.
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38119
Country
United States
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Ellen Sher, MD PA
City
Dallas
State/Province
Texas
ZIP/Postal Code
75230
Country
United States
Facility Name
Children's Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Cooks Children Medical Center/Dept. of Pediatric Endocrinology
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States

12. IPD Sharing Statement

Links:
URL
https://trialinfoemail.pfizer.com/pages/landing.aspx?StudyID=A6281280&StudyName=Individualized%2C%20Target-Driven%20Treatment%20Of%20Children%20With%20Idiopathic%20Short%20Stature
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

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

We'll reach out to this number within 24 hrs