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

This study has been completed.

Study NCT00125164 Information provided by Ipsen

First Received on July 27, 2005. Last Updated on March 17, 2011 History of Changes

Results First Received: July 8, 2009

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Conditions:	Growth Disorders Insulin-Like Growth Factor-1 Deficiency
Intervention:	Drug: rhIGF-1 (mecasermin, Tercica, Inc.)

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Study Initiation Date: 14 March 2004. Study completion Date: 02 July 2008. 44 investigators screened subjects; 14 did not enroll any subjects. 137 subjects were enrolled. Consent for one randomized subject was withdrawn prior to any study measurements. This subject was excluded from all analyses.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Subject screening consisted of three staged clinic visits over 6 weeks, including a medical history, complete physical examination, IGF-1 measurements, a Growth Hormone (GH) stimulation test, and an IGF-1 generation test.

Reporting Groups

	Description
Untreated	Observational Group
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.
80 μg/kg BID (Twice Daily Dosing)	Injection of rhIGF-1 80 μg/kg BID
120 μg/kg BID (Twice Daily Dosing)	Injection of rhIGF-1 120 μg/kg BID

Participant Flow: Overall Study

	Untreated	40 μg/kg BID (Twice Daily Dosing)	80 μg/kg BID (Twice Daily Dosing)	120 μg/kg BID (Twice Daily Dosing)
STARTED	25	16	44	51
COMPLETED	23	16	40	45
NOT COMPLETED	2	0	4	6
Adverse Event	0	0	2	4
Lost to Follow-up	0	0	1	1
Withdrawal by Subject	2	0	1	1

Baseline Characteristics

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Reporting Groups

	Description
Untreated	Observational Group
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.
80 μg/kg BID (Twice Daily Dosing)	Injection of rhIGF-1 80 μg/kg BID
120 μg/kg BID (Twice Daily Dosing)	Injection of rhIGF-1 120 μg/kg BID

Baseline Measures

	Untreated	40 μg/kg BID (Twice Daily Dosing)	80 μg/kg BID (Twice Daily Dosing)	120 μg/kg BID (Twice Daily Dosing)	Total
Number of Participants [units: participants]	25	16	44	51	136
Age [units: years] Mean ± Standard Deviation	7.0 ± 2.4	7.6 ± 2.4	7.7 ± 2.4	7.6 ± 2.7	7.5 ± 2.5
Gender [units: participants]
Female	10	4	14	8	36
Male	15	12	30	43	100
Race/Ethnicity, Customized [units: participants]
Asian	1	0	1	2	4
Black	0	0	0	2	2
Hispanic	2	0	4	4	10
Native Hawaiian or Pacific Islander	0	0	0	1	1
White	22	16	38	41	117
Other	0	0	1	1	2
Stratum ^[1] [units: participants]
Height Standard Deviation Score < -2.6	13	8	25	30	76
Height Standard Deviation Score >= -2.6	12	8	19	21	60
Height Standard Deviation (SD) Score ^[1] [units: SD] Mean ± Standard Deviation	-2.8 ± 0.7	-2.6 ± 0.6	-2.7 ± 0.5	-2.9 ± 0.8	-2.8 ± 0.7
IGF-1 Standard Deviation (SD) Score ^[2] [units: SD] Mean ± Standard Deviation	-2.6 ± 0.7	-2.8 ± 0.8	-2.6 ± 0.7	-3.0 ± 0.8	-2.8 ± 0.8
Maximum Stimulated Growth Hormone ^[3] [units: ng/mL] Mean ± Standard Deviation	17.0 ± 6.5	16.9 ± 6.2	17.8 ± 9.0	20.7 ± 16.8	18.6 ± 12.1
Weight [units: Kg] Mean ± Standard Deviation	17.8 ± 4.6	18.5 ± 4.7	19.4 ± 5.4	18.5 ± 5.7	18.6 ± 5.3

[1]	Please note that SD is a standard term used in growth studies and represents Standard Deviations calculated as the patient value minus the mean divided by the standard deviation. Standard Deviation Scores vary depending on the age and sex of the child.
[2]	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.
[3]	Growth Hormone Stimulation Test results expressed in ng/mL

Outcome Measures

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1. Primary:

Height Velocity During the First Year - Intent to Treat (ITT)Population [ Time Frame: Measured at baseline and at one year ]

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2. Secondary:

Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population [ Time Frame: Measured at baseline and at one year ]

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3. Secondary:

Changes in Bone Age From Baseline to One Year [ Time Frame: Measured at baseline and at one year ]

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4. Secondary:

Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year [ Time Frame: Measured at baseline and at one year ]

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5. Secondary:

Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year [ Time Frame: Measured at baseline and at one year ]

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6. Secondary:

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 ]

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7. Secondary:

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 ]

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8. Secondary:

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 ]

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9. Secondary:

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 ]

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10. Post-Hoc:

Height Velocity During the First Year for Subjects - Completers [ Time Frame: One Year ]

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11. Post-Hoc:

Change From Baseline in Height Standard Deviation (SD) Score at One Year - Completers [ Time Frame: One Year ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: Each investigator may publish or report data from their own subjects. The trial data in aggregate are the property of Tercica, Inc. and may not be published without permission of Tercica, Inc. Tercica, Inc. will be the final arbitrator of issues relating to the publication or presentation of the aggregate data.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.

Results Point of Contact:

Name/Title: Medical Director, Pediatric Endocrinology
Organization: Ipsen
phone: clinical.trials@ipsen.com
e-mail: clinical.trials@ipsen.com

No publications provided

Responsible Party:	Cathryn Clary, MD, Ipsen
ClinicalTrials.gov Identifier:	NCT00125164 History of Changes
Other Study ID Numbers:	MS301
Study First Received:	July 27, 2005
Results First Received:	July 8, 2009
Last Updated:	March 17, 2011
Health Authority:	United States: Food and Drug Administration