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Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years

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ClinicalTrials.gov Identifier: NCT01066052
Recruitment Status : Completed
First Posted : February 10, 2010
Results First Posted : December 14, 2018
Last Update Posted : December 14, 2018
Sponsor:
Information provided by (Responsible Party):
Merck KGaA, Darmstadt, Germany

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Turner's Syndrome
Intervention Drug: r-hGH
Enrollment 115
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Recombinant Human Growth Hormone (r-hGH) Historical Control
Hide Arm/Group Description Participants (girls) received r-hGH (Saizen®) as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower received 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years. This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated.
Period Title: Overall Study
Started 64 51
Treated 61 0
Completed 61 51
Not Completed 3 0
Reason Not Completed
Enrolled But Not Treated             3             0
Arm/Group Title r-hGH Historical Control Total
Hide Arm/Group Description Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years. This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. Total of all reporting groups
Overall Number of Baseline Participants 64 51 115
Hide Baseline Analysis Population Description
All Participants.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 64 participants 51 participants 115 participants
<=18 years
64
 100.0%
51
 100.0%
115
 100.0%
Between 18 and 65 years
0
   0.0%
0
   0.0%
0
   0.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 64 participants 51 participants 115 participants
Female
64
 100.0%
51
 100.0%
115
 100.0%
Male
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Height SDS at Year 4
Hide Description Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.
Time Frame Year 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All treated participants from r-hGH arm and all participants from Historical Control arm.
Arm/Group Title r-hGH Historical Control
Hide Arm/Group Description:
Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated.
Overall Number of Participants Analyzed 61 51
Mean (Standard Deviation)
Unit of Measure: standard deviation score
-1.35  (0.86) -2.44  (0.73)
2.Secondary Outcome
Title Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels
Hide Description HbA1c develops when hemoglobin, a protein within red blood cells that carries oxygen throughout the body, joins with glucose in the blood, becoming glycated. The higher the level of glucose in the blood, the higher the level of HbA1c is detectable on red blood cells. The normal range for HbA1c is 4 percent (%) to 5.9%. Number of participants, who had abnormal HbA1c levels any time during the assessment, were reported.
Time Frame Baseline up to Year 2
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title r-hGH
Hide Arm/Group Description:
Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
1
3.Secondary Outcome
Title Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)
Hide Description BA was determined using left wrist and hand X-ray. CA was determined using the date of birth. Difference of BA and CA (BA-CA) was reported.
Time Frame Baseline, Year 1, Year 2
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All treated participants from r-hGH arm. This outcome measure was only planned in the r-hGH arm. Overall number of participants analyzed = participants with available data for this outcome; number analyzed = participants with available data for this outcome at specified timepoint.
Arm/Group Title r-hGH
Hide Arm/Group Description:
Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
Overall Number of Participants Analyzed 60
Mean (Standard Deviation)
Unit of Measure: months
Baseline Number Analyzed 58 participants
-8.9  (7.5)
Year 1 Number Analyzed 60 participants
-8.7  (6.5)
Year 2 Number Analyzed 57 participants
-8.1  (7.8)
4.Secondary Outcome
Title Number of Participants With Anti r-hGH Antibodies
Hide Description [Not Specified]
Time Frame Baseline up to Year 2
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Data for this outcome was not collected from any participant.
Arm/Group Title r-hGH Historical Control
Hide Arm/Group Description:
Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
This arm included matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated.
Overall Number of Participants Analyzed 0 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels
Hide Description The normal range for IGF1 levels is 45 to 117 nanogram per milliliter (ng/mL) for girls aged less than (<) 3 years and 80 to 236 ng/mL for girls aged 3 to 6 years. Values outside the normal range were considered abnormal. Number of participants, who had abnormal IGF1 levels any time during the assessment, were reported.
Time Frame Baseline up to Year 2
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All treated participants from r-hGH arm. This outcome measure was only planned in the r-hGH arm.
Arm/Group Title r-hGH
Hide Arm/Group Description:
Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
46
6.Secondary Outcome
Title Number of Participants Who Reached Normal Height at Year 4
Hide Description Participants with normal height were those who attained a height which was within +/- 2 height SDS of reference population standard. Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.
Time Frame Year 4
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All treated participants from r-hGH arm. This outcome measure was only planned in the r-hGH arm.
Arm/Group Title r-hGH
Hide Arm/Group Description:
Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
49
Time Frame [Not Specified]
Adverse Event Reporting Description Adverse events were collected only from participants treated with r-hGH. No adverse event data collection occurred in Historical Control arm.
 
Arm/Group Title r-hGH
Hide Arm/Group Description Participants (girls) received r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received depended on participants’ baseline height SDS relative to the general population standard: participants with a height SDS of -2 SD or lower received 0.05 mg/kg per day r-hGH and those with a height SDS between -1 and -2 SD received 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants received a fixed dose of 0.05 mg/kg per day for a further 2 years.
All-Cause Mortality
r-hGH
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
r-hGH
Affected / at Risk (%)
Total   8/61 (13.11%) 
Congenital, familial and genetic disorders   
Chronic persistent cytolysis * 1  1/61 (1.64%) 
Immune system disorders   
Immunoglobulin G4 deficiency * 1  1/61 (1.64%) 
Nervous system disorders   
Serious otitis * 1  1/61 (1.64%) 
Respiratory, thoracic and mediastinal disorders   
Arterial hypertension * 1  1/61 (1.64%) 
Skin and subcutaneous tissue disorders   
Measles * 1  1/61 (1.64%) 
Surgical and medical procedures   
Trauma leading to scalp surgery * 1  1/61 (1.64%) 
Surgery to remove large mass (meningocele/benign cyst) * 1  1/61 (1.64%) 
Transtympanic drainage tube placement * 1  1/61 (1.64%) 
Tonsillectomy/adenoidectomy * 1  1/61 (1.64%) 
Vascular disorders   
Urethral reflux * 1  1/61 (1.64%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, No Coding Applied
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
r-hGH
Affected / at Risk (%)
Total   47/61 (77.05%) 
Infections and infestations   
Infection * 1  47/61 (77.05%) 
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, No Coding Applied
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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: Merck KGaA Communication Center,
Organization: Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany
Phone: 496151725200
Responsible Party: Merck KGaA, Darmstadt, Germany
ClinicalTrials.gov Identifier: NCT01066052     History of Changes
Other Study ID Numbers: GF 5834
First Submitted: February 5, 2010
First Posted: February 10, 2010
Results First Submitted: September 7, 2017
Results First Posted: December 14, 2018
Last Update Posted: December 14, 2018