Trial · Interventional

CompletedPhase 3Results postedRegistry data

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

NCTNCT00396097Other idA6281280

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Overview

Status: Completed
Phase: Phase 3
Enrollment: 316 participants (actual)
Sponsor: Pfizer · Industry
Design: Randomized · Open label (no masking)
Countries: 1 country
United States
Start: Dec 2006
Primary completion: Aug 2012
Completion: Aug 2012
First posted: 6 Nov 2006 (estimated)
Last update posted: 5 Apr 2016 (estimated)
Conditions: Idiopathic Short Stature

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

2006200820102012

Ph 3
Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature
NCT00396097
Dec 2006 to Aug 2012

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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

Interventions

GenotropinDrug
GenotropinDrug

Arms2

Active comparator
Standard
Standard daily HGH treatment
Active comparator
Formula-based
Formula-based dose regimen

Primary outcomes1

Outcomes the sponsor listed as primary.

Absolute On-target Difference (AOTD) at 24 Months
Time frame · 2 years
Registry 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).

Secondary outcomes(5)

Variability of Height SDS at 24 Months
Time frame · 2 years
Registry description
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
Registry description
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
Registry 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.
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months
Time frame · 4 years
Registry description
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
Registry description
Change in height SDS was measured at 48 months.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· 2 years

Absolute On-target Difference (AOTD) at 24 Months

Group	Value (Standard Deviation Score (SDS))	Spread
Standard Dose Arm	0.603	0.2948
Individualized Dose Arm	0.625	0.3003

Secondary and other outcome results(5)

Secondary outcomeMean· 2 years

Variability of Height SDS at 24 Months

Group	Value (Standard Deviation Score (SDS))	Spread
Standard Dose Arm	1.12	0.408
Individualized Dose Arm	1.11	0.518

Secondary outcomeMedian· 2 years

Time Cost (Months Until >= -2 SDS)

Group	Value (Months)
Standard Dose Arm	12.00
Individualized Dose Arm	12.00

Secondary outcomeMean· 4 years

Computed Cost of Height Gain at 48 Months

Group	Value (mg/cm)	Spread
Standard Dose Arm	72.77	17.914
Individualized Dose Arm Overall	67.30	24.382
Individualized Dose Arm 0.18 mg/kg/Week	63.07	21.656
Individualized Dose Arm 0.24 mg/kg/Week	69.62	21.903

Secondary outcomeMean· 4 years

Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months

Group	Value (mg/cm)	Spread
Standard Dose Arm	127.99	29.708
Individualized Dose Arm Overall	91.34	31.854
Individualized Dose Arm 0.18 mg/kg/Week	80.06	21.000
Individualized Dose Arm 0.24/mg/kg/Week	92.24	25.213

Secondary outcomeMean· 4 years

Change From Baseline in Height SDS at 48 Months.

Group	Value (Standard Deviation Score (SDS))	Spread
Standard Dose Arm	1.33	0.717
Individualized Dose Arm Overall	1.24	0.668
Individualized Dose Arm 0.18 mg/kg/Week	1.33	0.637
Individualized Dose Arm 0.24 mg/kg/Week	1.34	0.633

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
Standard Dose ArmThe participants received subcutaneous genotropin daily, at a maintained standard dose of 0.37 mg/kg/week, throughout the four years.	–	13 / 118	103 / 118
Individualized Dose ArmThe participants received subcutaneous genotropin daily, at formula-calculated dose (up to maximum dose of 0.7 mg/kg/week) for the initial 2 years and then lowered to one of two approximately physiological doses (0.18 mg/kg/week or 0.24 mg/kg/week) for the remaining 2 years.	–	7 / 198	165 / 198

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: from 3 Years up to 10 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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.

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