Trial Outcomes & Findings for Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism (NCT NCT00837616)

NCT ID: NCT00837616

Last Updated: 2023-05-16

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 41 participants
• Primary outcome timeframe: 12 months
• Results posted on: 2023-05-16

Participant Flow

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.

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.

Participant milestones

Measure	Oral Estradiol Group A received the oral estradiol for 12 months	Transdermal Estradiol Group B received the transdermal estradiol for 12 months
Overall Study STARTED	20	21
Overall Study COMPLETED	20	20
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Oral Estradiol Group A received the oral estradiol for 12 months	Transdermal Estradiol Group B received the transdermal estradiol for 12 months
Overall Study Withdrawal by Subject	0	1

Baseline Characteristics

Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism

Baseline characteristics by cohort

Measure	Group A n=20 Participants Group A received the oral estradiol for 12 months	Group B n=21 Participants Group B received the transdermal estradiol for 12 months	Total n=41 Participants Total of all reporting groups
Age, Categorical <=18 years	16 Participants n=5 Participants	15 Participants n=7 Participants	31 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants	6 Participants n=7 Participants	10 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	16.7 years STANDARD_DEVIATION 1.7 • n=5 Participants	16.7 years STANDARD_DEVIATION 1.8 • n=7 Participants	16.7 years STANDARD_DEVIATION 1.7 • n=5 Participants
Sex: Female, Male Female	20 Participants n=5 Participants	21 Participants n=7 Participants	41 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants	14 participants n=7 Participants	25 participants n=5 Participants
Region of Enrollment Chile	9 participants n=5 Participants	7 participants n=7 Participants	16 participants n=5 Participants

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Change in Weight From Baseline at 12 Months	1.1 kilograms Standard Error 0.6	1.9 kilograms Standard Error 0.6

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Change in Body Mass Index From Baseline at 12 Months	0.075 kg/m2 Standard Error 0.38	0.65 kg/m2 Standard Error 0.38

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Change in Percent Fat Mass From Baseline in 12 Months	-0.14 percent fat mass Standard Error 0.56	-0.64 percent fat mass Standard Error 0.56

PRIMARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Change in Fat Free Mass From Baseline at 12 Months	1.03 kg Standard Error 0.37	1.67 kg Standard Error 0.4

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Changes in Insulin Growth Factor-I From Baseline at 12 Months	-16 ng/ml Standard Error 12	28 ng/ml Standard Error 12

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months Total Cholesterol	168 mg/dl Standard Error 7	153 mg/dl Standard Error 6
Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months Low Density Lipoprotein	93 mg/dl Standard Error 4	88 mg/dl Standard Error 5
Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months High Density Lipoprotein	56 mg/dl Standard Error 3	50 mg/dl Standard Error 2
Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months Triglycerides	95 mg/dl Standard Error 19	70 mg/dl Standard Error 9

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Rates of Lipid Oxidation After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months	10 Kcal/Fat Free Mass/day Standard Error 2.8	7.3 Kcal/Fat Free Mass/day Standard Error 1.6

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Serum 17B Estradiol Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months	124 pg/ml Standard Error 19	74 pg/ml Standard Error 17

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Serum Estrone Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months	504 pg/mL Standard Error 80	43 pg/mL Standard Error 7

SECONDARY outcome

Timeframe: 12 months

Outcome measures

Measure	Oral Estradiol n=20 Participants Group A received the oral estradiol for 12 months	Transdermal Estradiol n=20 Participants Group B received the transdermal estradiol for 12 months
Serum Estrone Sulfate Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months	63638 pg/mL Standard Error 21088	1875 pg/mL Standard Error 414

Adverse Events

Group A

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Group B

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Nelly Mauras

Nemours Children's Clinic

Phone: (904) 697-3674

Email: nmauras@nemours.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place