Trial Outcomes & Findings for Efficacy and Safety of a High Dosage Compared to the Label Dosage of Somatropin in Early Pubertal Stage Children With Growth Hormone Deficiency (NCT NCT00191165)
NCT ID: NCT00191165
Last Updated: 2009-06-10
Results Overview
Height velocity (difference between 2 height measurements, divided by years elapsed between measurements) SDS was derived by subtracting age and gender-matched population mean height velocity from patient's height velocity (based on measurements 12 months apart) then dividing this value by age and gender-matched population height velocity SD.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
27 participants
Primary outcome timeframe
12-Months
Results posted on
2009-06-10
Participant Flow
Participant milestones
| Measure |
High Dose Somatropin
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
13
|
|
Overall Study
COMPLETED
|
12
|
11
|
|
Overall Study
NOT COMPLETED
|
2
|
2
|
Reasons for withdrawal
| Measure |
High Dose Somatropin
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Overall Study
Adverse Event
|
2
|
0
|
|
Overall Study
Protocol Violation
|
0
|
1
|
|
Overall Study
Participant Moved Away
|
0
|
1
|
Baseline Characteristics
Efficacy and Safety of a High Dosage Compared to the Label Dosage of Somatropin in Early Pubertal Stage Children With Growth Hormone Deficiency
Baseline characteristics by cohort
| Measure |
High Dose Somatropin
n=14 Participants
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
n=13 Participants
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age Continuous
|
13.5 years
STANDARD_DEVIATION 1.2 • n=5 Participants
|
13.0 years
STANDARD_DEVIATION 1.5 • n=7 Participants
|
13.3 years
STANDARD_DEVIATION 1.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4.0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
23.0 Participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
14 participants
n=5 Participants
|
13 participants
n=7 Participants
|
27.0 participants
n=5 Participants
|
|
Race/Ethnicity
Caucasian
|
14 participants
n=5 Participants
|
12 participants
n=7 Participants
|
26.0 participants
n=5 Participants
|
|
Race/Ethnicity
Other
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1.0 participants
n=5 Participants
|
|
Baseline Predicted Height
|
168 centimeters
STANDARD_DEVIATION 6.8 • n=5 Participants
|
169 centimeters
STANDARD_DEVIATION 10.7 • n=7 Participants
|
168 centimeters
STANDARD_DEVIATION 8.7 • n=5 Participants
|
|
Baseline Predicted Height Standard Deviation Score (SDS)
|
-0.9 standard deviation score
STANDARD_DEVIATION 0.8 • n=5 Participants
|
-0.5 standard deviation score
STANDARD_DEVIATION 1.0 • n=7 Participants
|
-0.7 standard deviation score
STANDARD_DEVIATION 0.9 • n=5 Participants
|
|
Body Mass Index
|
18.9 kilograms per square meter (kg/m2)
STANDARD_DEVIATION 3.2 • n=5 Participants
|
19.4 kilograms per square meter (kg/m2)
STANDARD_DEVIATION 3.7 • n=7 Participants
|
19.2 kilograms per square meter (kg/m2)
STANDARD_DEVIATION 3.4 • n=5 Participants
|
|
Bone Age
|
12.4 years
STANDARD_DEVIATION 0.7 • n=5 Participants
|
12.1 years
STANDARD_DEVIATION 1.0 • n=7 Participants
|
12.2 years
STANDARD_DEVIATION 0.8 • n=5 Participants
|
|
Height
|
145 centimeters
STANDARD_DEVIATION 5.1 • n=5 Participants
|
146 centimeters
STANDARD_DEVIATION 7.8 • n=7 Participants
|
145 centimeters
STANDARD_DEVIATION 6.5 • n=5 Participants
|
|
Height Standard Deviation Score (SDS)
|
-1.7 standard deviation score
STANDARD_DEVIATION 1.0 • n=5 Participants
|
-1.1 standard deviation score
STANDARD_DEVIATION 1.0 • n=7 Participants
|
-1.4 standard deviation score
STANDARD_DEVIATION 1.0 • n=5 Participants
|
|
Target Height
|
168 centimeters
STANDARD_DEVIATION 5.0 • n=5 Participants
|
168 centimeters
STANDARD_DEVIATION 7.5 • n=7 Participants
|
168 centimeters
STANDARD_DEVIATION 6.2 • n=5 Participants
|
|
Target Height Standard Deviation Score (SDS)
|
-0.9 standard deviation score
STANDARD_DEVIATION 0.7 • n=5 Participants
|
-0.6 standard deviation score
STANDARD_DEVIATION 0.5 • n=7 Participants
|
-0.8 standard deviation score
STANDARD_DEVIATION 0.6 • n=5 Participants
|
PRIMARY outcome
Timeframe: 12-MonthsPopulation: All enrolled participants with baseline and 12-month height measurements.
Height velocity (difference between 2 height measurements, divided by years elapsed between measurements) SDS was derived by subtracting age and gender-matched population mean height velocity from patient's height velocity (based on measurements 12 months apart) then dividing this value by age and gender-matched population height velocity SD.
Outcome measures
| Measure |
High Dose Somatropin
n=14 Participants
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
n=12 Participants
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Height Velocity Standard Deviation Score (SDS) at 12-Month Endpoint
|
1.83 standard deviation score
Standard Deviation 0.91
|
1.38 standard deviation score
Standard Deviation 1.41
|
SECONDARY outcome
Timeframe: Baseline, 12-Months, 24-MonthsPopulation: All enrolled participants with at least one post-baseline height measurement, using last observation available prior to 12-month and 24-month visits respectively, if 12-month or 24-month height was not available.
This was derived by subtracting the age-and-gender-matched population 50th percentile height from the patient's height and then dividing this value by the age-and-gender-matched population height SD.
Outcome measures
| Measure |
High Dose Somatropin
n=14 Participants
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
n=12 Participants
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Change From Baseline to 12-Month and 24-Month Endpoints in Height Standard Deviation Score (SDS)
12-Month Height SDS Change (N=14, N=12)
|
0.49 standard deviation score
Standard Error 0.12
|
0.34 standard deviation score
Standard Error 0.13
|
|
Change From Baseline to 12-Month and 24-Month Endpoints in Height Standard Deviation Score (SDS)
24-Month Height SDS Change (N=13, N=11)
|
0.88 standard deviation score
Standard Error 0.17
|
0.40 standard deviation score
Standard Error 0.18
|
SECONDARY outcome
Timeframe: 24 MonthsPopulation: All enrolled participants with at least one post-baseline height velocity measurement, using the last observation available prior to the 24-month visit if the 24-month height velocity was not available.
Height velocity (difference between 2 height measurements, divided by years elapsed between measurements) SDS was derived by subtracting age and gender-matched population mean height velocity from patient's height velocity (based on measurements 12 months apart) then dividing this value by age and gender-matched population height velocity SD.
Outcome measures
| Measure |
High Dose Somatropin
n=13 Participants
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
n=11 Participants
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Height Velocity Standard Deviation Score (SDS) at 24 Month Endpoint
|
2.58 standard deviation score
Standard Deviation 1.86
|
1.45 standard deviation score
Standard Deviation 1.06
|
Adverse Events
High Dose Somatropin
Serious events: 1 serious events
Other events: 13 other events
Deaths: 0 deaths
Label Dose Somatropin
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
High Dose Somatropin
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Injury, poisoning and procedural complications
Vertebral injury
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
Other adverse events
| Measure |
High Dose Somatropin
Somatropin: 0.05 to 0.07 milligram/kilogram/day subcutaneous injection
|
Label Dose Somatropin
Somatropin: 0.025 to 0.035 milligram/kilogram/day subcutaneous injection
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Eye disorders
Retinoschisis
|
0.00%
0/14
|
7.7%
1/13 • Number of events 1
|
|
General disorders
Chest pain
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
General disorders
Pyrexia
|
14.3%
2/14 • Number of events 2
|
15.4%
2/13 • Number of events 2
|
|
Infections and infestations
Ear infection
|
7.1%
1/14 • Number of events 2
|
0.00%
0/13
|
|
Infections and infestations
Genital infection
|
0.00%
0/14
|
7.7%
1/13 • Number of events 1
|
|
Infections and infestations
Impetigo
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Infections and infestations
Influenza
|
14.3%
2/14 • Number of events 3
|
0.00%
0/13
|
|
Infections and infestations
Pharyngitis
|
7.1%
1/14 • Number of events 6
|
7.7%
1/13 • Number of events 1
|
|
Infections and infestations
Rhinitis
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Infections and infestations
Varicella
|
7.1%
1/14 • Number of events 1
|
7.7%
1/13 • Number of events 1
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Metabolism and nutrition disorders
Impaired fasting glucose
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Metabolism and nutrition disorders
Insulin resistance
|
14.3%
2/14 • Number of events 2
|
0.00%
0/13
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Musculoskeletal and connective tissue disorders
Polyarthritis
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
14.3%
2/14 • Number of events 2
|
0.00%
0/13
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
14.3%
2/14 • Number of events 2
|
7.7%
1/13 • Number of events 1
|
|
Nervous system disorders
Disturbance in attention
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Nervous system disorders
Headache
|
21.4%
3/14 • Number of events 10
|
0.00%
0/13
|
|
Nervous system disorders
Syncope
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Reproductive system and breast disorders
Epididymal cyst
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Reproductive system and breast disorders
Gynaecomastia
|
21.4%
3/14 • Number of events 3
|
7.7%
1/13 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/14
|
7.7%
1/13 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Periorbital oedema
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
7.1%
1/14 • Number of events 2
|
0.00%
0/13
|
Additional Information
Chief Medical Officer
Eli Lilly and Company
Phone: 800-545-5979
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60