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Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq (OPTIMA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00234533
Recruitment Status : Completed
First Posted : October 7, 2005
Results First Posted : August 9, 2018
Last Update Posted : January 8, 2019
Sponsor:
Information provided by (Responsible Party):
Ipsen

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Turner Syndrome
Renal Insufficiency, Chronic
Pituitary Diseases
Dwarfism
Intervention Drug: Somatropin (rDNA origin)
Enrollment 251
Recruitment Details 251 children presenting growth failure associated with Growth Hormone Deficiency (GHD), Turner Syndrome (TS) or Chronic Renal Insufficiency (CRI) were screened (with informed consent) in 46 study centers across Europe. First patient enrolled: 7 June 2004; last subject completed: 22 July 2008
Pre-assignment Details Of the 251 children screened 3 did not receive study medication, 1 due to withdrawal of consent, 1 did not meet screening criteria for height and 1 was not included due to the investigator's decision.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description

Patients received daily subcutaneous (s.c.) injections of NutropinAq 10 milligrams (mg)/ 2 milliliters (mL) for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/ kilogram (kg) bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Period Title: Overall Study
Started 251
Completed 240
Not Completed 11
Reason Not Completed
Withdrawal by Subject             6
Adverse Event             2
Enrolled but not treated             3
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Baseline Participants 244
Hide Baseline Analysis Population Description
The Intention-to-Treat (ITT) Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 244 participants
9.9  (3.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 244 participants
Female
138
  56.6%
Male
106
  43.4%
Birth height  
Mean (Standard Deviation)
Unit of measure:  Centimeters (cm)
Number Analyzed 244 participants
48.49  (3.36)
Birth weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 244 participants
2.94  (0.60)
Duration of gestation  
Mean (Standard Deviation)
Unit of measure:  Weeks of amenorrhea
Number Analyzed 244 participants
38.7  (2.6)
Genetic target height   [1] 
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 244 participants
166.26  (8.16)
[1]
Measure Description: The genetic potential height, or target height was estimated using the adult height of each parent (i.e. genetic mother and father).
Calculated genetic target height Standard Deviation Score (SDS)   [1] 
Mean (Standard Deviation)
Unit of measure:  Standard Deviations (SD)
Number Analyzed 244 participants
-0.33  (0.95)
[1]
Measure Description:

The genetic potential height, or target height was estimated as follows:

  • Boys Target Height = [(mother height cm + father height cm)/2] + 6.5 cm.
  • Girls Target Height = [(mother height cm + father height cm)/2] - 6.5 cm. The French growth charts were used for the calculation of standard deviation score (SDS) parameters: the charts provide for each age range and sex a mean parameter and standard deviation (SD) value, from which the SDS parameter can be derived assuming a normal distribution. For example: Height SDS = (height – reference mean height (age, sex)) / reference SD (age, sex).
Duration of previous Growth Hormone (GH) treatment   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 59 participants
1.68  (1.56)
[1]
Measure Analysis Population Description: Data is presented for non-naïve patients only.
Auxological Parameter - Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 244 participants
122.95  (20.24)
Auxological Parameter - Calculated height SDS  
Mean (Standard Deviation)
Unit of measure:  SD
Number Analyzed 244 participants
-2.68  (1.33)
Auxological Parameter - Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 244 participants
27.97  (11.91)
Auxological Parameter - Calculated weight SDS  
Mean (Standard Deviation)
Unit of measure:  SD
Number Analyzed 244 participants
-1.85  (1.88)
Auxological Parameter - Annualised Growth Velocity (GV)  
Mean (Standard Deviation)
Unit of measure:  Cm/year
Number Analyzed 244 participants
4.94  (2.56)
Auxological Parameter - Calculated annualised GV SDS  
Mean (Standard Deviation)
Unit of measure:  SD
Number Analyzed 244 participants
-0.56  (2.67)
1.Primary Outcome
Title Insulin-Like Growth Factor I (IGF-I) Levels Measured Using the Timed Capillary Blood Spot Samples
Hide Description

Fingertip capillary blood was collected using filter paper cards for the assay of capillary blood spot IGF-I in line with the monitoring recommendations of the Lawson Wilkins Paediatric Endocrine Society (LWPES) for treatment with recombinant GH therapy in children.

Capillary IGF-I assays were performed by the patient at home one day per week during Weeks 21, 22 and 23 only (same week day). The samples were scheduled in the evening prior to the injection of NutropinAq and between 7:00 and 9:00 the following morning. An extended window from 6:00 to 12:00 was allowed for defining protocol deviations.

The number of capillary blood spot IGF-I measurements and the optimal timing of samples to assess the IGF-I status of NutropinAq treated patients was assessed. IGF-I measurements for the morning and evening sampling are presented.

Time Frame At Weeks 21, 22 and 23
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data. Only evaluable subjects with an assessment at the specified timepoint were included in each individual analysis.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/ kilogram kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: nanograms/milliliter (ng/mL)
Week 21 Morning sampling Number Analyzed 226 participants
223.54  (161.86)
Week 21 Evening sampling Number Analyzed 230 participants
212.97  (153.61)
Week 22 Morning sampling Number Analyzed 223 participants
238.02  (177.89)
Week 22 Evening sampling Number Analyzed 229 participants
226.90  (164.38)
Week 23 Morning sampling Number Analyzed 226 participants
241.58  (167.75)
Week 23 Evening sampling Number Analyzed 225 participants
235.47  (168.48)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments An Intra-class Correlation Coefficient (ICC) for the evening series (defined as 12:00 to 24:00) was determined for the overall ITT population to confirm whether only one measurement would be sufficient to accurately represent the IGF-I measurement over Weeks 21, 22 and 23. The ICC expresses the relative magnitude of the 2 components of the total variability, i.e. the biological variability and random error, in a series of measurements on different patients.
Type of Statistical Test Other
Comments An one-way Analysis of Covariance (ANOVA) was used to calculate the between patient variation and within patient variation and then the ICC and its confidence limitations.
Method of Estimation Estimation Parameter ICC
Estimated Value 0.90
Confidence Interval (2-Sided) 95%
0.88 to 0.92
Estimation Comments An ICC ≥ 0.8 was considered satisfactory and indicative that a single sample would be representative of the overall IGF-I status.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments An ICC for the morning series (defined as 06:00 to 12:00) was determined for the overall ITT population to confirm whether only one measurement would be sufficient to accurately represent the IGF-I measurement over Weeks 21, 22 and 23. The ICC expresses the relative magnitude of the 2 components of the total variability, i.e. the biological variability and random error, in a series of measurements on different patients.
Type of Statistical Test Other
Comments An one-way ANOVA was used to calculate the between patient variation and within patient variation and then the ICC and its confidence limitations.
Method of Estimation Estimation Parameter ICC
Estimated Value 0.92
Confidence Interval (2-Sided) 95%
0.90 to 0.93
Estimation Comments An ICC ≥ 0.8 was considered satisfactory and indicative that a single sample would be representative of the overall IGF-I status.
2.Secondary Outcome
Title Assessment of IGF-I Levels: Categorised by Weekly Timing (Weeks 21-23) and Daily Timing (Morning and Evening)
Hide Description The influence of daily and weekly timing on the IGF-I value as measured using the capillary blood spot method was analysed. A 3-way analyses of variance (ANOVA) was performed with patient, day and daily timing as factors after appropriate transformation to obtain normally distributed parameters. The interaction day*time was tested and kept in the model only if p-value<0.1. Parameter estimates from the statistical model are presented as least squares means for the categories of daily timing (Morning and Evening) and weekly timing (Week 21, Week 22 and Week 23). The values reported for Week 21, 22, and 23 represent the average IGF-I levels from the morning and evening samples at each week. The values reported for Evening represent the Evening IGF-I levels averaged across Weeks 21, 22, and 23, and similarly for the Morning values.
Time Frame At Weeks 21, 22 and 23
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Least Squares Mean (95% Confidence Interval)
Unit of Measure: ng/mL
Week 21
218.3
(203.0 to 233.5)
Week 22
232.4
(217.1 to 247.8)
Week 23
238.5
(223.2 to 253.9)
Evening
225.1
(212.6 to 237.6)
Morning
234.4
(221.8 to 246.9)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the weekly timing effect (Week 21 versus Week 22 versus Week 23) on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Fisher's F statistic (F) value
Estimated Value 1.79
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the weekly timing on the IGF-I levels; F value = 1.79 and the significance probability value, PR>F = 0.1678.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the daily timing effect (morning versus evening) on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 1.06
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the daily timing on the IGF-I levels; F value = 1.06 and the significance probability value, PR>F = 0.3035
3.Secondary Outcome
Title Assessment of IGF-I Levels: Categorised by Sex and Prepubertal Status
Hide Description The influence of sex and prepubertal status on the IGF-I value as measured using the capillary blood spot method was analysed. Parameter estimates from the statistical model are presented as least squares means for the categories of sex (male and female) and prepubertal status (pubertal and prepubertal). The values reported represent average IGF-I levels as determined from the 6 measurements taken (i.e. morning and evening samples at Weeks 21, 22 and 23).
Time Frame At Weeks 21, 22 and 23
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Least Squares Mean (95% Confidence Interval)
Unit of Measure: ng/mL
Female Number Analyzed 138 participants
234.4
(224.7 to 244.4)
Male Number Analyzed 106 participants
172.1
(162.7 to 181.9)
Pubertal Number Analyzed 116 participants
238.9
(224.8 to 253.5)
Prepubertal Number Analyzed 128 participants
168.4
(160.7 to 176.3)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the effect of the patient's sex (male versus female) on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 83.97
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the patient's sex on the IGF-I levels; F value = 83.97 and the significance probability value, PR>F = <0.0001.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the effect of the patient's pubertal status (pubertal versus prepubertal) on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 68.35
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the patient's pubertal status on the IGF-I levels; F value = 68.35 and the significance probability value, PR>F = <0.0001.
4.Secondary Outcome
Title Multivariate Linear Regression Analyses to Assess Factors Affecting the Variability of IGF-I Levels: Categorised by Disease Condition and Location
Hide Description

A multivariate linear regression analysis of factors on within-subject coefficient of variation (WCV) using a stepwise forward-backward elimination was used to determine the effect of individual factors on IGF-I values as measured using the capillary blood spot method (p=0.15 for a variable to enter and remain in the model). The WCV was computed from the series of 6 measurements (2 samplings in each of Weeks 21, 22 and 23). The influence of disease condition and country clusters on the IGF-I value were assessed.

Country clusters: cluster 1: France; cluster 2: Spain, Greece, Romania and Italy; cluster 3: UK, Belgium, Czech Republic, Denmark, Germany, Slovakia, Austria and Finland ; cluster 4: Russia ; cluster 5: Ukraine.

Parameter estimates from the statistical model presented as least squares means for categories of disease condition (GHD and TS) and location (Clusters 1, 2, 3, 4 and 5) are presented.

Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data. Only evaluable subjects within each of the individual subgroups are presented for each category. As there was only 1 patient with CRI, no analysis was performed for this disease condition.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Least Squares Mean (Standard Error)
Unit of Measure: Regression coefficient
Disease condition: GHD Number Analyzed 158 participants
0.058  (0.031)
Disease condition: TS Number Analyzed 85 participants
0.000  (0)
Cluster 1 Number Analyzed 81 participants
0.014  (0.045)
Cluster 2 Number Analyzed 36 participants
-0.070  (0.048)
Cluster 3 Number Analyzed 45 participants
-0.013  (0.046)
Cluster 4 Number Analyzed 52 participants
-0.059  (0.040)
Cluster 5 Number Analyzed 30 participants
0.000  (0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the disease condition (GHD versus TS) on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 10.73
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the disease condition on the IGF-I levels; F value = 10.73 and the significance probability value, PR>F = 0.0012.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the country cluster on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 2.20
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the country cluster on the IGF-I levels; F value = 2.20 and the significance probability value, PR>F = 0.0701.
5.Secondary Outcome
Title Multivariate Linear Regression Analyses to Assess Factors Affecting the Variability of IGF-I Levels: Categorised by Time of Year, Calculated Age at Enrolment and Disease Condition
Hide Description

A multivariate linear regression analysis of factors on WCV using a stepwise forward-backward elimination was used to determine the effect of individual factors on IGF-I values as measured using the capillary blood spot method (p=0.15 for a variable to enter and remain in the model). The WCV was computed from the series of 6 measurements (2 samplings in each of Weeks 21, 22 and 23). The influence of the time of the year (1st, 2nd, 3rd and 4th quarters), calculated age at enrolment and disease condition on the IGF-I value were assessed.

Parameter estimates from the statistical model are presented as least squares means for the categories of time of the year (1st, 2nd, 3rd and 4th quarters), calculated age at enrolment and disease condition (GHD and TS).

Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The Per Protocol Population consisted of all patients in the ITT Population for whom no major protocol violations/deviations occurred. Only evaluable subjects within each of the individual subgroups are presented for each category.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 175
Least Squares Mean (Standard Error)
Unit of Measure: Regression coefficient
1st quarter Number Analyzed 175 participants
0.118  (0.034)
2nd quarter Number Analyzed 175 participants
0.028  (0.033)
3rd quarter Number Analyzed 175 participants
0.029  (0.038)
4th quarter Number Analyzed 175 participants
0.000  (0)
Calculated age at enrolment Number Analyzed 175 participants
-0.007  (0.003)
Disease condition: GHD Number Analyzed 106 participants
0.051  (0.026)
Disease condition: TS Number Analyzed 69 participants
0.000  (0)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the time of the year on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 3.65
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the time of the year on the IGF-I levels; F value = 3.65 and the significance probability value, PR>F = 0.0139.
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the effect of the calculated age at enrolment on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 7.38
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the calculated age at enrolment on the IGF-I levels; F value = 7.38 and the significance probability value, PR>F = 0.0073.
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments Analysis of the effect of the disease condition on the IGF-I value as measured by capillary blood spot method.
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter F value
Estimated Value 3.86
Estimation Comments [Not Specified]
Other Statistical Analysis For the effect of the disease condition on the IGF-I levels; F value = 3.86 and the significance probability value, PR>F = 0.0511
6.Secondary Outcome
Title Change From Baseline at Week 24 in the IGF-I Levels as Measured by Capillary Blood Spot Method and Serum IGF-I Assay
Hide Description

3 simultaneous IGF-I measurements were taken at Weeks 0 (baseline), 12 and 24 by serum and capillary assay to determine the precision profile of the capillary blood spot method versus the serum IGF-I assay.

Change from baseline at Week 24 in the IGF-I measurements by capillary blood spot method and serum assay are presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data. Evaluable subjects with data available at Week 24 are presented.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: ng/mL
IGF-I Capillary blood spot method Number Analyzed 219 participants
123.49  (127.40)
Serum IGF-I assay Number Analyzed 151 participants
215.41  (188.03)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NutropinAq 10 mg/2 ml (30 IU)
Comments The Bland and Altman method was used to compare the results of each of the three simultaneous random capillary and serum measurements were compared. The difference between the capillary blood spot method and the serum IGF-I measurements is presented
Type of Statistical Test Other
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean difference
Estimated Value -164.79
Confidence Interval (2-Sided) 95%
-483.35 to -153.77
Parameter Dispersion
Type: Standard Error of the Mean
Value: 159.28
Estimation Comments [Not Specified]
7.Secondary Outcome
Title Change From Baseline at Week 12 and Week 24 in Insulin-Like Growth Factor Binding Protein 3 (IGFBP3) Measurements
Hide Description

The LWPES recommends that treatment for any indication with recombinant GH therapy in children be accompanied by regular monitoring of IGF-I and IGFBP3 concentrations. IGFBP3 binds circulating IGF-I and serum samples were taken at Visit 1 (Week 0), Visit 2 (Week 12) and Visit 3 (Week 24) in order to measure IGFBP3.

Change from baseline (Visit 1) at Visits 2 and 3 in IGFBP3 is presented.

Time Frame Baseline to Week 12 and Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data. Evaluable subjects with data available at each timepoint are presented.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: ng/mL
Change from baseline to Visit 2 Number Analyzed 235 participants
1112.4  (1321.4)
Change from baseline to Visit 3 Number Analyzed 230 participants
1285.2  (1350.9)
8.Secondary Outcome
Title Change From Baseline at Week 24 in the Auxological Parameter Height
Hide Description

The auxological parameter, height, was measured at Visit 1 (Baseline measurement), Visit 2 (Week 12) and Visit 3 (Week 24).

Change from baseline in measured height at Visit 3 (Week 24) for the overall ITT population is presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: cm
4.73  (1.69)
9.Secondary Outcome
Title Change From Baseline at Week 24 in the Auxological Parameter Calculated Height SDS
Hide Description

The auxological parameter, height, was measured at Visit 1 (Baseline measurement), Visit 2 (Week 12) and Visit 3 (Week 24). The French growth charts were used for the calculation of SDS parameters: the charts provide for each age range and sex a mean parameter and SD value, from which the SDS parameter can be derived assuming a normal distribution. For example: Height SDS = (height – reference mean height (age, sex)) / reference SD (age, sex). The SDS indicates the number of standard deviations away from the mean. A SDS of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. A positive change in SDS indicates an improvement in growth, therefore, a favorable outcome.

Change from baseline in the calculated height SDS at Visit 3 (Week 24) for the overall ITT population is presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
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Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: SD Score
0.45  (0.37)
10.Secondary Outcome
Title Change From Baseline at Week 24 in the Auxological Parameter Weight
Hide Description

The auxological parameter, weight, was measured at Visit 1 (Baseline measurement), Visit 2 (Week 12) and Visit 3 (Week 24).

Change from baseline in measured weight at Visit 3 (Week 24) for the overall ITT population is presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: kg
2.01  (1.87)
11.Secondary Outcome
Title Change From Baseline at Week 24 in the Auxological Parameter Calculated Weight SDS
Hide Description

The auxological parameter, weight, was measured at Visit 1 (Baseline measurement), Visit 2 (Week 12) and Visit 3 (Week 24). The French growth charts were used for the calculation of SDS parameters: the charts provide for each age range and sex a mean parameter and SD value, from which the SDS parameter can be derived assuming a normal distribution. For example: Weight SDS = (weight – reference mean weight (age, sex)) / reference SD (age, sex). The SDS indicates the number of standard deviations away from the mean. A SDS of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. A positive change in SDS indicates an improvement in weight, therefore, a favorable outcome.

Change from baseline in the calculated weight SDS at Visit 3 (Week 24) for the overall ITT population is presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: SD Score
0.20  (0.56)
12.Secondary Outcome
Title Change From Baseline at Week 24 in the Auxological Parameter Annualised Growth Velocity
Hide Description

The auxological parameter, annualised growth velocity, was measured at Visit 1 (Baseline measurement), Visit 2 (Week 12) and Visit 3 (Week 24).

Change from baseline in the measured annualised growth velocity at Visit 3 (Week 24) for the overall ITT population is presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: cm/year
5.20  (4.72)
13.Secondary Outcome
Title Change From Baseline at Week 24 in the Auxological Parameter Annualised Growth Velocity SDS
Hide Description

The auxological parameter, annualised growth velocity, was measured at Visit 1 (Baseline measurement), Visit 2 (Week 12) and Visit 3 (Week 24). The French growth charts were used for the calculation of SDS parameters: the charts provide for each age range and sex a mean parameter and SD value, from which the SDS parameter can be derived assuming a normal distribution. For example: Annualised GV SDS = (annualised GV – reference mean annualised GV (age, sex)) / reference SD (age, sex). The SDS indicates the number of standard deviations away from the mean. A SDS of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. A positive change in SDS indicates an improvement in growth velocity, therefore, a favorable outcome.

Change from baseline in the annualised growth velocity SDS at Visit 3 (Week 24) for the overall ITT population is presented.

Time Frame Baseline to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Mean (Standard Deviation)
Unit of Measure: SD Score
5.10  (4.80)
14.Secondary Outcome
Title Percentage of Patients Rating the Overall Handling of the Administration Device, NutropinAq Pen, to Assess the Acceptability and Tolerance of NutropinAq and Its Pen
Hide Description

The acceptability was evaluated by a questionnaire at Month 5. The users (parents and/or child) of NutropinAq pen and compliance aid booklet were asked to describe and rate the pen, cartridge, compliance aid booklet and their ease of use.

The percentage of patients responding to each category for the assessment of the overall handling of the NutropinAq pen are presented. The categories are: Very easy, Easy, Moderately difficult, Difficult, Very difficult and Missing.

Time Frame At Month 5
Hide Outcome Measure Data
Hide Analysis Population Description
The ITT Population consisted of all treated patients (enrolled patients who received at least one injection of treatment) and who provided any follow-up data.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 244
Measure Type: Number
Unit of Measure: Percentage of patients
Very easy 39.8
Easy 52.5
Moderately difficult 3.3
Difficult 0
Very difficult 0.4
Missing 4.1
15.Secondary Outcome
Title Posology of NutropinAq at Baseline (Visit 1) Summarised as Mean Dose
Hide Description It was intended that the posology (mg/kg/day) of NutropinAq would remain constant throughout the study. The mean posology adopted at Visit 1 is presented.
Time Frame Visit 1 (Baseline)
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all patients who received at least one injection of treatment.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 248
Mean (Standard Deviation)
Unit of Measure: mg/kg/day
0.0382  (0.0092)
16.Secondary Outcome
Title Extent of Exposure to NutropinAq Throughout the Study
Hide Description The extent of treatment exposure throughout the study is presented as the mean number of daily injections performed.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all patients who received at least one injection of treatment.
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description:

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

Overall Number of Participants Analyzed 248
Mean (Standard Deviation)
Unit of Measure: days
159.7  (35.8)
Time Frame From baseline (Week 0) up to Week 24.
Adverse Event Reporting Description Treatment emergent adverse events are presented for the Safety Population which consisted of all patients who received at least 1 injection of treatment.
 
Arm/Group Title NutropinAq 10 mg/2 ml (30 IU)
Hide Arm/Group Description

Patients received daily s.c. injections of NutropinAq 10 mg/ 2 mL for 6 months. The therapeutic daily doses administered were as follows:

  • GHD patients: 0.025 – 0.035 mg/kg bodyweight
  • TS patients: up to 0.05 mg/kg bodyweight
  • CRI patients: up to 0.05 mg/kg bodyweight

Patients visited the study clinic for a baseline visit and for 2 other visits every 3 months (Weeks 12 and 24). Additional home assessments were made at Weeks 21, 22 and 23.

The investigator determined the dose administered to each patient, and it was recommended to perform the injection in the evening.

All-Cause Mortality
NutropinAq 10 mg/2 ml (30 IU)
Affected / at Risk (%)
Total   0/248 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
NutropinAq 10 mg/2 ml (30 IU)
Affected / at Risk (%) # Events
Total   6/248 (2.42%)    
Congenital, familial and genetic disorders   
Retinitis Pigmentosa  1  1/248 (0.40%)  1
Infections and infestations   
Bronchopneumonia  1  1/248 (0.40%)  1
Gastroenteritis  1  1/248 (0.40%)  1
Pyelonephritis acute  1  1/248 (0.40%)  1
Injury, poisoning and procedural complications   
Accidental overdose  1  1/248 (0.40%)  1
Femur fracture  1  1/248 (0.40%)  1
Musculoskeletal and connective tissue disorders   
Pain in extremity  1  1/248 (0.40%)  1
1
Term from vocabulary, MedDRA 9.1
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
NutropinAq 10 mg/2 ml (30 IU)
Affected / at Risk (%) # Events
Total   81/248 (32.66%)    
Blood and lymphatic system disorders   
Lymphadenopathy  1  1/248 (0.40%)  1
Cardiac disorders   
Tachycardia  1  1/248 (0.40%)  1
Ear and labyrinth disorders   
Auricular swelling  1  1/248 (0.40%)  1
Endocrine disorders   
Hypothyroidism  1  2/248 (0.81%)  2
Thyroiditis acute  1  1/248 (0.40%)  1
Eye disorders   
Conjunctivitis allergic  1  1/248 (0.40%)  1
Gastrointestinal disorders   
Abdominal pain  1  4/248 (1.61%)  4
Abdominal pain upper  1  4/248 (1.61%)  5
Aptyalism  1  1/248 (0.40%)  1
Diarrhoea  1  3/248 (1.21%)  4
Gastritis  1  1/248 (0.40%)  1
Gastrooesophageal reflux disease  1  1/248 (0.40%)  1
Nausea  1  3/248 (1.21%)  3
Stomatitis  1  1/248 (0.40%)  1
Vomiting  1  5/248 (2.02%)  8
General disorders   
Asthenia  1  2/248 (0.81%)  3
Face oedema  1  1/248 (0.40%)  1
Feeling cold  1  1/248 (0.40%)  1
Hyperthermia  1  1/248 (0.40%)  1
Injection site bruising  1  2/248 (0.81%)  2
Injection site oedema  1  1/248 (0.40%)  1
Injection site pain  1  1/248 (0.40%)  1
Malaise  1  1/248 (0.40%)  1
Oedema peripheral  1  1/248 (0.40%)  1
Pyrexia  1  9/248 (3.63%)  10
Infections and infestations   
Acute sinusitis  1  1/248 (0.40%)  1
Bronchitis  1  1/248 (0.40%)  1
Bronchitis acute  1  3/248 (1.21%)  3
Bronchopneumonia  1  1/248 (0.40%)  1
Cellulitis  1  1/248 (0.40%)  1
Ear infection  1  1/248 (0.40%)  2
Enterovirus infection  1  1/248 (0.40%)  1
Gastroenteritis  1  1/248 (0.40%)  1
Gastroenteritis viral  1  1/248 (0.40%)  1
Influenza  1  3/248 (1.21%)  4
Lower respiratory tract infection  1  2/248 (0.81%)  3
Nasopharyngitis  1  3/248 (1.21%)  3
Otitis media  1  5/248 (2.02%)  6
Rhinitis  1  6/248 (2.42%)  6
Scarlet fever  1  1/248 (0.40%)  1
Tonsillitis  1  2/248 (0.81%)  2
Tonsillitis bacterial  1  1/248 (0.40%)  2
Upper respiratory tract infection  1  7/248 (2.82%)  10
Viral infection  1  1/248 (0.40%)  1
Viral upper respiratory tract infection  1  1/248 (0.40%)  1
Injury, poisoning and procedural complications   
Arthropod bite  1  1/248 (0.40%)  1
Arthropod sting  1  1/248 (0.40%)  1
Foot fracture  1  1/248 (0.40%)  1
Hand fracture  1  2/248 (0.81%)  2
Wound complication  1  1/248 (0.40%)  1
Investigations   
Insulin-like Growth Factor increased  1  13/248 (5.24%)  13
Metabolism and nutrition disorders   
Decreased appetite  1  1/248 (0.40%)  2
Hyperglycaemia  1  1/248 (0.40%)  1
Polydipsia  1  1/248 (0.40%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  3/248 (1.21%)  3
Back pain  1  3/248 (1.21%)  3
Pain in extremity  1  2/248 (0.81%)  3
Pubic pain  1  1/248 (0.40%)  1
Scoliosis  1  1/248 (0.40%)  1
Nervous system disorders   
Headache  1  14/248 (5.65%)  27
Migraine  1  1/248 (0.40%)  1
Renal and urinary disorders   
Polyuria  1  1/248 (0.40%)  1
Respiratory, thoracic and mediastinal disorders   
Allergic bronchitis  1  1/248 (0.40%)  1
Cough  1  1/248 (0.40%)  1
Epistaxis  1  1/248 (0.40%)  1
Pharyngolaryngeal pain  1  1/248 (0.40%)  1
Skin and subcutaneous tissue disorders   
Alopecia  1  1/248 (0.40%)  1
Dermatitis allergic  1  1/248 (0.40%)  1
Dry skin  1  1/248 (0.40%)  1
Eczema  1  1/248 (0.40%)  1
Rash  1  2/248 (0.81%)  2
Swelling face  1  1/248 (0.40%)  1
Vascular disorders   
Wound haemorrhage  1  1/248 (0.40%)  1
1
Term from vocabulary, MedDRA 9.1
Indicates events were collected by systematic assessment
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: Medical Director, Pediatric Endocrinology
Organization: Ipsen
Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT00234533     History of Changes
Other Study ID Numbers: 2-79-58035-700
First Submitted: October 5, 2005
First Posted: October 7, 2005
Results First Submitted: May 9, 2017
Results First Posted: August 9, 2018
Last Update Posted: January 8, 2019