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Trial record 24 of 31 for:    IGFBP2

Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency

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ClinicalTrials.gov Identifier: NCT00125190
Recruitment Status : Completed
First Posted : July 29, 2005
Results First Posted : April 15, 2010
Last Update Posted : June 21, 2012
Sponsor:
Information provided by (Responsible Party):
Ipsen

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Insulin-Like Growth Factor-1 Deficiency
Growth Disorders
Intervention Drug: rhIGF-1 (mecasermin) for a period of 86 weeks
Enrollment 45
Recruitment Details First Subject Screened: 12 January 2005. Last Subject Completed: 14 January 2009. 12 investigators screened subjects, 1 did not enroll any subject. 89 subjects were screened, 41 were ineligible, 3 declined treatment and 45 were treated and analyzed.
Pre-assignment Details Screening consisted of two-staged clinic visits for up to 6 weeks, and included the following evaluations: medical history, complete physical examination, measurements of serum IGF-1 and IGF-1 binding proteins (IGFBP 1, IGFBP 2 and IGFBP-3), GH binding protein, Acid-labile subunit, GH stimulation test.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Period Title: Overall Study
Started 45
Completed 30
Not Completed 15
Reason Not Completed
Adverse Event             1
Lost to Follow-up             4
Patient/Parent Decision             7
other             1
Non-compliance             2
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Baseline Participants 45
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 45 participants
8.7  (2.8)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 45 participants
Female
7
  15.6%
Male
38
  84.4%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 45 participants
Black 1
Hispanic 12
White 31
Other 1
Body Mass Index SD Score  
Mean (Standard Deviation)
Unit of measure:  SDs
Number Analyzed 45 participants
-0.4  (0.7)
Bone Age  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 45 participants
7.2  (2.6)
Height SD Score  
Mean (Standard Deviation)
Unit of measure:  SDs
Number Analyzed 45 participants
-2.7  (0.6)
IGF Binding Protein-3 SD Score  
Mean (Standard Deviation)
Unit of measure:  SDs
Number Analyzed 45 participants
-0.7  (1.0)
IGF-1 SD Score  
Mean (Standard Deviation)
Unit of measure:  SDs
Number Analyzed 45 participants
-2.6  (0.5)
Maximum Stimulated GH  
Mean (Standard Deviation)
Unit of measure:  ng/mL
Number Analyzed 45 participants
20.5  (9.9)
Weight SD Score  
Mean (Standard Deviation)
Unit of measure:  SDs
Number Analyzed 45 participants
-2.3  (0.7)
1.Primary Outcome
Title Height Velocity Over the Study Period 0 - 34 Weeks [Intent to Treat Population]
Hide 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.
Time Frame 34 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT principle was used for the primary analysis, with imputation of missing height velocity and missing height SD score.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 45
Mean (Standard Deviation)
Unit of Measure: cm/yr
7.0  (1.5)
2.Primary Outcome
Title Height Velocity Over the Study Period 34 - 86 Weeks [Intent to Treat Population]
Hide 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.
Time Frame Weeks 34 to 86
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT principle was used for the primary analysis, with imputation of missing height velocity and missing height SD score. Only subjects continuing past Week 34 were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: cm/yr
6.7  (1.8)
3.Secondary Outcome
Title Changes in Height Standard Deviation (SD) Score Over the Study Period 0 - 34 Weeks [Intent to Treat Population]
Hide 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.
Time Frame Weeks 0 - 34
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT principle was used for the primary analysis, with imputation of missing height velocity and missing height SD score.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 45
Mean (Standard Deviation)
Unit of Measure: SDs
0.21  (0.20)
4.Secondary Outcome
Title Changes in Height Standard Deviation (SD) Score Over the Study Period 34 - 86 Weeks
Hide 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.
Time Frame Weeks 34 - 86
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT principle was used for the primary analysis, with imputation of missing height velocity and missing height SD score. Only subjects continuing past Week 34 were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 40
Mean (Standard Deviation)
Unit of Measure: SDs
0.45  (0.39)
5.Secondary Outcome
Title Bone Age - Change From Pretreatment Minus Change in Chronological Age Over the Study Period 0 - 86 Weeks [Intent to Treat Population]
Hide 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.
Time Frame Weeks 0 - 86
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who had both baseline and week 86 measurements were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 30
Mean (Standard Deviation)
Unit of Measure: years
0.2  (0.68)
6.Secondary Outcome
Title Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-1 (IGFBP-1) From Baseline to Week 86
Hide Description Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-1 in the growth factor panel.
Time Frame 86 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who had both baseline and week 34 measurements were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: percent
-89.6
(-98 to 69)
7.Secondary Outcome
Title Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-2 (IGFBP-2) From Baseline to Week 86
Hide Description Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-2 in the growth factor panel.
Time Frame 86 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who had both baseline and week 86 measurements were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: percent
37.9
(-41 to 518)
8.Secondary Outcome
Title Percent Changes in Serum Concentration of Insulin-like Growth Factor Binding-3 (IGFBP-3) From Baseline to Week 86
Hide Description Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-3 in the growth factor panel.
Time Frame 86 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who had both baseline and week 86 measurements were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: percent
0
(-42 to 64)
9.Secondary Outcome
Title Percent Changes in Serum Concentration of Acid Labile Subunit (ALS) From Baseline to Week 86
Hide Description Blood sample was collected while subject is in a fasting state for measuring the level of Serum Concentration of Acid Labile Subunit (ALS).
Time Frame 86 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who had both baseline and week 86 measurements were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 29
Median (Full Range)
Unit of Measure: percent
-7.7
(-56 to 90)
10.Secondary Outcome
Title rhIGF-1 Doses Required to Achieve the Serum IGF-1 Targets With Measures Taken at Each Study Visit
Hide Description [Not Specified]
Time Frame 34, 52 and 86 weeks
Outcome Measure Data Not Reported
11.Post-Hoc Outcome
Title Increase in Height Velocity Over the Study Period 0 - 34 Weeks [Completers]
Hide 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.
Time Frame Weeks 0 -34
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT principle was used for the primary analysis, with imputation of missing height velocity and missing height SD score. Only subjects completing Week 34 were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 43
Mean (Standard Deviation)
Unit of Measure: cm/yr
1.3  (3.56)
12.Post-Hoc Outcome
Title Increase in Height Velocity Over the Study Period 34 - 86 Weeks [Completers]
Hide 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.
Time Frame Weeks 34 - 86
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT principle was used for the primary analysis, with imputation of missing height velocity and missing height SD score. Only subjects completing Week 86 were included in the analysis.
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description:
During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
Overall Number of Participants Analyzed 30
Mean (Standard Deviation)
Unit of Measure: cm/yr
1.3  (3.58)
Time Frame 21 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title rhIGF-1 QD
Hide Arm/Group Description During the treatment phase, subjects received subcutaneous (SC) injections of rhIGF-1 at an initial dose of 60 µg/kg QD, with subsequent dose adjustments made in order to achieve the target serum IGF-1 concentration for the subject’s age and sex.
All-Cause Mortality
rhIGF-1 QD
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
rhIGF-1 QD
Affected / at Risk (%) # Events
Total   2/45 (4.44%)    
Congenital, familial and genetic disorders   
Arnold-Chiari Malformation * 1  1/45 (2.22%)  1
Infections and infestations   
Gastroenteritis * 1  1/45 (2.22%)  1
Nervous system disorders   
Syringomyelia * 1  1/45 (2.22%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
rhIGF-1 QD
Affected / at Risk (%) # Events
Total   41/45 (91.11%)    
Gastrointestinal disorders   
Abdominal Pain * 1  3/45 (6.67%)  3
Abdominal Pain Upper * 1  3/45 (6.67%)  4
Diarrhoea * 1  5/45 (11.11%)  6
Stomach Discomfort * 1  3/45 (6.67%)  3
Vomiting * 1  18/45 (40.00%)  21
General disorders   
Injection Site Bruising * 1  3/45 (6.67%)  5
Pyrexia * 1  14/45 (31.11%)  18
Infections and infestations   
Gastroenteritis * 1  6/45 (13.33%)  8
Gastroenteritis Viral * 1  3/45 (6.67%)  4
Influenza * 1  5/45 (11.11%)  6
Nasopharyngitis * 1  3/45 (6.67%)  5
Otitis Media * 1  5/45 (11.11%)  7
Sinusitis * 1  4/45 (8.89%)  4
Upper Respiratory Tract Infection * 1  13/45 (28.89%)  24
Viral Infection * 1  7/45 (15.56%)  13
Metabolism and nutrition disorders   
Hypoglycaemia  1  5/45 (11.11%)  14
Musculoskeletal and connective tissue disorders   
Arthralgia * 1  6/45 (13.33%)  8
Nervous system disorders   
Headache * 1  18/45 (40.00%)  32
Respiratory, thoracic and mediastinal disorders   
Cough * 1  11/45 (24.44%)  13
Nasal Congestion * 1  6/45 (13.33%)  9
Pharyngolaryngeal Pain * 1  4/45 (8.89%)  5
Rhinorrhoea * 1  3/45 (6.67%)  3
Skin and subcutaneous tissue disorders   
Dermatitis Contact * 1  3/45 (6.67%)  3
Rash * 1  5/45 (11.11%)  7
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, MedDRA (11.0)
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.
 
Results Point of Contact
Name/Title: Cathryn Clary, M.D.
Organization: Ipsen
Phone: +1 650-624-4919
Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT00125190     History of Changes
Other Study ID Numbers: MS308
First Submitted: July 27, 2005
First Posted: July 29, 2005
Results First Submitted: March 23, 2010
Results First Posted: April 15, 2010
Last Update Posted: June 21, 2012