Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism

This study has been completed.
Sponsor:
Collaborator:
Genentech
Information provided by (Responsible Party):
Nelly Mauras, Nemours Children's Clinic
ClinicalTrials.gov Identifier:
NCT00837616
First received: February 3, 2009
Last updated: February 26, 2014
Last verified: February 2014
Results First Received: May 28, 2013  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Turner Syndrome
Hypogonadism
Premature Ovarian Failure
Interventions: Drug: 17 B estradiol orally
Drug: 17 B estradiol

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Forty-one girls with Turner Syndrome (45X and related karyotypes), between 13 and 20 years were recruited and followed among the 3 participating centers at the Nemours Children’s Clinic, Jacksonville (coordinating center), Nemours Jefferson, and Clínica las Condes/University of Chile, Santiago, Chile.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Any previous growth hormone (GH) therapy was discontinued at least 6 months prior to study participation. Estrogen replacement therapy was discontinued for at least 6 weeks prior to baseline studies. Subjects with significant obesity (BMI > 36 kg/m2) or history of systemic illness were excluded.

Reporting Groups
  Description
Oral Estradiol Group A received the oral estradiol for 12 months
Transdermal Estradiol Group B received the transdermal estradiol for 12 months

Participant Flow:   Overall Study
    Oral Estradiol     Transdermal Estradiol  
STARTED     20     21  
COMPLETED     20     20  
NOT COMPLETED     0     1  
Withdrawal by Subject                 0                 1  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Group A Group A received the oral estradiol for 12 months
Group B Group B received the transdermal estradiol for 12 months
Total Total of all reporting groups

Baseline Measures
    Group A     Group B     Total  
Number of Participants  
[units: participants]
  20     21     41  
Age  
[units: participants]
     
<=18 years     16     15     31  
Between 18 and 65 years     4     6     10  
>=65 years     0     0     0  
Age  
[units: years]
Mean ± Standard Deviation
  16.7  ± 1.7     16.7  ± 1.8     16.7  ± 1.7  
Gender  
[units: participants]
     
Female     20     21     41  
Male     0     0     0  
Region of Enrollment  
[units: participants]
     
United States     11     14     25  
Chile     9     7     16  



  Outcome Measures
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1.  Primary:   Change in Weight From Baseline at 12 Months   [ Time Frame: 12 months ]

2.  Primary:   Change in Body Mass Index From Baseline at 12 Months   [ Time Frame: 12 months ]

3.  Primary:   Change in Percent Fat Mass From Baseline in 12 Months   [ Time Frame: 12 months ]

4.  Primary:   Change in Fat Free Mass From Baseline at 12 Months   [ Time Frame: 12 months ]

5.  Secondary:   Changes in Insulin Growth Factor-I From Baseline at 12 Months   [ Time Frame: 12 months ]

6.  Secondary:   Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months   [ Time Frame: 12 months ]

7.  Secondary:   Rates of Lipid Oxidation After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months   [ Time Frame: 12 months ]

8.  Secondary:   Serum 17B Estradiol Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months   [ Time Frame: 12 months ]

9.  Secondary:   Serum Estrone Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months   [ Time Frame: 12 months ]

10.  Secondary:   Serum Estrone Sulfate Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months   [ Time Frame: 12 months ]

11.  Primary:   Characterize PK/PD and Relative Biological Potency of Different Oral vs TD Estrogen Preparations   [ Time Frame: 6 weeks ]
Results not yet posted.   Anticipated Posting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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: Dr. Nelly Mauras
Organization: Nemours Children's Clinic
phone: (904) 697-3674
e-mail: nmauras@nemours.org


Publications of Results:

Responsible Party: Nelly Mauras, Nemours Children's Clinic
ClinicalTrials.gov Identifier: NCT00837616     History of Changes
Other Study ID Numbers: 908-M01
Study First Received: February 3, 2009
Results First Received: May 28, 2013
Last Updated: February 26, 2014
Health Authority: United States: Institutional Review Board