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Trial · Interventional

CompletedPhase 4Results postedRegistry data

Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism

NCTNCT00837616 (opens ClinicalTrials.gov in a new tab)Other id908-M01
Open at ClinicalTrials.govAsk about this trial

This trial does not test a peptide that PepBank tracks.

  • Names IGF-1 (mecasermin), does not test it. It appears in the conditions.

Overview

Status
Completed
Phase
Phase 4
Enrollment
41 participants (actual)
Sponsor
Nemours Children's Clinic · Other
Design
Randomized · Open label (no masking)
Countries
2 countries

United States, Chile

Start
Jan 2009
Primary completion
Jun 2012
Completion
Dec 2012
First posted
5 Feb 2009 (estimated)
Last update posted
16 May 2023
Conditions
Turner Syndrome · Hypogonadism · Premature Ovarian Failure

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20092010201120122013
  1. Ph 4

    Estrogen Dosing in Turner Syndrome: Pharmacology and Metabolism

    NCT00837616

    Jan 2009 to Dec 2012

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

Estrogen is necessary for feminization during puberty and to decrease bone resorption, the latter critical for the achievement of peak bone mass and normal bone health in the female. The practicing pediatric endocrinologist often faces the dilemma of how to best feminize girls with hypogonadism (lack of estrogen), manifested as delayed or arrested puberty, due to disorders of the brain or the ovaries. We propose a series of studies to address which type, dose, and route of delivery of estrogen are suitable choices in feminizing and sustaining estrogen concentrations in adolescent girls with Turner syndrome. To accomplish this we will study girls/young woman between the ages of 13 to 20 with Turner Syndrome in 2 protocols. In Protocol # 1 we will study 24 girls with TS, they will receive 3 different estrogen preparations, either by mouth or via a patch for a total of 6 weeks. They will come to the clinical research center for blood draws after 2 wks of taking the estrogen. With this study, we hope to learn how the body responds to estrogen differently, depending on the form estrogen is given and how high, estrogen levels gets in the blood in these girls with Turner Syndrome. We will be comparing these patients estrogen levels to girls that menstruate normally and do not have Turner Syndrome. In Protocol #2, 40 patients with TS will be recruited; these patients will take estrogen for 1 year, either by mouth or via a patch. Patients will come to the lab for blood drawn in 7 occasions and we will measure estrogen levels as well as other hormones and lipid levels. We will also perform a Dual-energy X-ray absorptiometry (DXA) study (like an X ray) to assess body composition and bone mineralization. We will adjust doses based on the estrogen levels we find. With this study we hope to learn how estrogen affects body composition, i.e., the amount of fat vs. muscle, and how different forms of estrogen affect blood cholesterol and other hormones. This study will allow us to understand better how to best replace young woman with Turner Syndrome with estrogen.

Interventions

  • 17 B estradiol orallyDrug
  • 17 B estradiolDrug

Arms2

  • Active comparator

    Group A

    Group A will receive the oral estradiol for 12 months

  • Active comparator

    Group B

    Group B will receive the transdermal estradiol for 12 months

Primary outcomes4

Outcomes the sponsor listed as primary.

  1. Change in Weight From Baseline at 12 Months

    Time frame · 12 months

  2. Change in Body Mass Index From Baseline at 12 Months

    Time frame · 12 months

  3. Change in Percent Fat Mass From Baseline in 12 Months

    Time frame · 12 months

  4. Change in Fat Free Mass From Baseline at 12 Months

    Time frame · 12 months

Secondary outcomes(6)

  1. Changes in Insulin Growth Factor-I From Baseline at 12 Months

    Time frame · 12 months

  2. Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

    Time frame · 12 months

  3. Rates of Lipid Oxidation After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

    Time frame · 12 months

  4. Serum 17B Estradiol Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

    Time frame · 12 months

  5. Serum Estrone Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

    Time frame · 12 months

  6. Serum Estrone Sulfate Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

    Time frame · 12 months

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· 12 months

Change in Weight From Baseline at 12 Months

GroupValue (kilograms)Spread
Oral Estradiol1.10.6
Transdermal Estradiol1.90.6
Primary outcomeMean· 12 months

Change in Body Mass Index From Baseline at 12 Months

GroupValue (kg/m2)Spread
Oral Estradiol0.0750.38
Transdermal Estradiol0.650.38
Primary outcomeMean· 12 months

Change in Percent Fat Mass From Baseline in 12 Months

GroupValue (percent fat mass)Spread
Oral Estradiol-0.140.56
Transdermal Estradiol-0.640.56
Primary outcomeMean· 12 months

Change in Fat Free Mass From Baseline at 12 Months

GroupValue (kg)Spread
Oral Estradiol1.030.37
Transdermal Estradiol1.670.4
Secondary and other outcome results(4)
Secondary outcomeMean· 12 months

Changes in Insulin Growth Factor-I From Baseline at 12 Months

GroupValue (ng/ml)Spread
Oral Estradiol-1612
Transdermal Estradiol2812
Secondary outcomeMean· 12 months

Lipids Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

GroupValue (mg/dl)Spread
Oral Estradiol1687
Transdermal Estradiol1536
Secondary outcomeMean· 12 months

Rates of Lipid Oxidation After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

GroupValue (Kcal/Fat Free Mass/day)Spread
Oral Estradiol102.8
Transdermal Estradiol7.31.6
Secondary outcomeMean· 12 months

Serum 17B Estradiol Concentrations After Using Oral Versus Transdermal 17B Estradiol Replacement for 12 Months

GroupValue (pg/ml)Spread
Oral Estradiol12419
Transdermal Estradiol7417

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
Group AGroup A received the oral estradiol for 12 months–0 / 200 / 20
Group BGroup B received the transdermal estradiol for 12 months–0 / 210 / 21

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • ResultTorres-Santiago L, Mericq V, Taboada M, Unanue N, Klein KO, Singh R, Hossain J, Santen RJ, Ross JL, Mauras N. Metabolic effects of oral versus transdermal 17beta-estradiol (E(2)): a randomized clinical trial in girls with Turner syndrome. J Clin Endocrinol Metab. 2013 Jul;98(7):2716-24. doi: 10.1210/jc.2012-4243. Epub 2013 May 15. PMID 23678038
  • ResultTaboada M, Santen R, Lima J, Hossain J, Singh R, Klein KO, Mauras N. Pharmacokinetics and pharmacodynamics of oral and transdermal 17beta estradiol in girls with Turner syndrome. J Clin Endocrinol Metab. 2011 Nov;96(11):3502-10. doi: 10.1210/jc.2011-1449. Epub 2011 Aug 31. PMID 21880799

Eligibility

Who could join, as posted.

Sex
Female
Age
from 13 Years up to 20 Years
Healthy volunteers
Accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: * Girls with Turner Syndrome (45X, or related karyotypes) diagnosed clinically and cytogenetically * Female subjects with Y material will be allowed providing gonadectomies have been performed previously * Age: 13-20 years * Subjects have completed or nearly completed their linear growth * Previous growth hormone (GH) therapy discontinued at least 6 months prior to study participation * Stable thyroid replacement therapy will be allowed * Celiac disease on stable diets will be allowed * Any previous hormone replacement therapy (HRT) will be allowed Exclusion Criteria: * Diabetes Mellitus on insulin therapy, insulin sensitizers or oral hypoglycemics * Inflammatory Bowel Disease (ulcerative colitis or Crohn's disease), celiac disease * Cigarette smoking * Any other chronic conditions, that, in the opinion of investigators could impair the metabolism of nutrients * Severe obesity, i.e., Body Mass Index (BMI) \>95th centile

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