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

Phase 3

Completed

• Conditions: Idiopathic Short Stature
• Interventions: Drug: Genotropin

• 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

Study Timeline

• Study First Submitted: 2006-11-02
• Study First Submitted QC: 2006-11-02
• Study First Posted: 2006-11-06
• Study Start: 2006-12
• Primary Completion: 2012-08
• Study Completion: 2012-08
• Results First Submitted: 2013-08-30
• Results First Submitted QC: 2013-08-30
• Results First Posted: 2013-11-13
• Status Verified: 2016-03
• Last Update Submitted: 2016-03-09
• Last Update Posted: 2016-04-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 316

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 & 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
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.
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.

Trial Locations

Locations (42)

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

Children's Hospital of Los Angeles; 🇺🇸 Los Angeles, California, 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

Childrens Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Memorial Pediatric Specialty Clinic; 🇺🇸 Colorado Springs, Colorado, United States

Pediatric Endocrine Associates; 🇺🇸 Greenwood Village, Colorado, United States

Pediatric Endocrine Associates at The Longmont Clinic; 🇺🇸 Longmont, Colorado, United States

Joe DiMaggio Children's Hospital; 🇺🇸 Hollywood, Florida, United States

Miller School Of Medicine, University of Miami/Jackson Memorial Medical Center; 🇺🇸 Miami, Florida, United States

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