- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00125164
Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency
Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Prepubertal Children With Growth Failure Associated With Primary IGF-1 Deficiency: A Phase 3, Randomized, Open Label, Observation-Controlled, Multicenter, Parallel-Dose Comparison Trial
Study Overview
Status
Intervention / Treatment
Detailed Description
Prepubertal growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, primary IGFD is defined as short stature (height standard deviation score[SDS]<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS <-2 below the mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after a GH stimulation test. Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.
This trial is one year, randomized, open label, observation-controlled, parallel-dose comparison efficacy and safety study conducted in approximately 40 centers across the United States.
Study Type
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
-
-
California
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Brisbane, California, United States, 94005
- Ipsen (formerly Tercica)
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;
- Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys
- Prepubertal
- Height SD score of < -2
- IGF-1 SD score of < -2
Exclusion Criteria:
- Prior treatment with rhGH, rhIGF-1, or other growth-influencing medications
- Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)
- Chronic illness such as diabetes, cystic fibrosis, etc.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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.
|
Twice Daily Injection
|
Experimental: 80 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 80 μg/kg BID
|
Twice Daily Injection
|
Experimental: 120 μg/kg BID (twice daily dosing)
Injection of rhIGF-1 120 μg/kg BID
|
Twice Daily Injection
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Height Velocity During the First Year - Intent to Treat (ITT)Population
Time Frame: Measured at baseline and at one year
|
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.
|
Measured at baseline and at one year
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population
Time Frame: Measured at baseline and at one year
|
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.
|
Measured at baseline and at one year
|
Changes in Bone Age From Baseline to One Year
Time Frame: Measured at baseline and at one year
|
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.
|
Measured at baseline and at one year
|
Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year
Time Frame: Measured at baseline and at one year
|
Blood sample was collected while subject is in a fasting state for measuring the level of IGF-1 in the growth factor panel.
|
Measured at baseline and at one year
|
Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year
Time Frame: Measured at baseline and at one year
|
Blood sample was collected for measuring the level of IGF-2 in the growth factor panel.
|
Measured at baseline and at one year
|
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
|
Blood sample was collected for measuring the level of insulin-like growth factor binding protein-2 (IGFBP-2) in the growth factor panel.
|
Measured at baseline and at one year
|
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
|
Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-3 in the growth factor panel.
|
Measured at baseline and at one year
|
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
|
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.
|
Study Day 1 and 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
|
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.
|
Study Day 1 and Day 7
|
Collaborators and Investigators
Sponsor
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- MS301
- 2019-001020-36 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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