Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency

NCTNCT00125164Other idMS301Other id2019-001020-36Other id2019

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Overview

Status: Completed
Phase: Phase 3
Enrollment: 137 participants (actual)
Sponsor: Ipsen · Industry
Design: Randomized · Open label (no masking)
Countries: 1 country
United States
Start: Mar 2004
Primary completion: Jul 2008
Completion: Jul 2008
First posted: 29 Jul 2005 (estimated)
Last update posted: 18 Sept 2019
Conditions: Growth Disorders · Insulin-Like Growth Factor-1 Deficiency

Timeline

1 trial · 1 completed · 1 with posted results

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

200420052006200720082009

Ph 3
Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency
NCT00125164
Mar 2004 to Jul 2008

Registry summary

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

This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.

Interventions

rhIGF-1 (mecasermin, Tercica, Inc.)Drug

Arms4

No intervention
Untreated
Observational Group
Experimental
40 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 40 μg/kg BID. Per protocol amendment these subjects were reassigned to receive 120 μg/kg BID. Due to the dose change, the efficacy results for these subjects were analysed in a separate subanalysis. For all outcome measures, mean and standard deviations were not calculated for this arm.
Experimental
80 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 80 μg/kg BID
Experimental
120 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 120 μg/kg BID

Primary outcomes1

Outcomes the sponsor listed as primary.

Height Velocity During the First Year - Intent to Treat (ITT)Population
Time frame · Measured at baseline and at one year
Registry description
Height to be measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Reposition subject between each measurement.

Secondary outcomes(8)

Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population
Time frame · Measured at baseline and at one year
Registry description
Height to be measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Reposition subject between each measurement. Please note that Standard Deviation (SD) Score is a term used in growth studies. The SD Score is calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child.
Changes in Bone Age From Baseline to One Year
Time frame · Measured at baseline and at one year
Registry description
Plain X-rays of the left hand and wrist exposed for bone age appraisal. The films are sent to a central facility for standardized evaluation.
Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year
Time frame · Measured at baseline and at one year
Registry description
Blood sample was collected while subject is in a fasting state for measuring the level of IGF-1 in the growth factor panel.
Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year
Time frame · Measured at baseline and at one year
Registry description
Blood sample was collected for measuring the level of IGF-2 in the growth factor panel.
Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year
Time frame · Measured at baseline and at one year
Registry description
Blood sample was collected for measuring the level of insulin-like growth factor binding protein-2 (IGFBP-2) in the growth factor panel.
Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year
Time frame · Measured at baseline and at one year
Registry description
Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-3 in the growth factor panel.
IGF Generation Test: Change of Serum IGF-1 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
Time frame · Study Day 1 and Day 7
Registry description
Blood drawn at Study Day 1, followed by 7 days of rhGH daily dosing at 0.05 mg/kg of body weight. Additional blood draw at Study Day 7.
IGF Generation Test: Change of Serum IGFBP-3 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
Time frame · Study Day 1 and Day 7
Registry description
Blood drawn at Study Day 1, followed by 7 days of rhGH daily dosing at 0.05 mg/kg of body weight. Additional blood draw at Study Day 7.

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· Measured at baseline and at one year