Transgender Estradiol Affirming Therapy

NCT ID: NCT05010707

Last Updated: 2024-11-26

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-08-02

Study Completion Date

2024-01-01

Brief Summary

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The purpose of this open label, pilot, randomized clinical trial is to evaluate the effectiveness, safety and tolerability of estrogen use in transgender female and the degree of testosterone suppression achieved in this population when placed on gender affirming pharmacological therapy.

Detailed Description

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Transgender patients suffer from poor mental and medical health outcomes compared to their cisgender peers. Given the widespread acknowledgment of the health care needs of transgender people, priority should be given to those actions that will ensure safe and appropriate care in health centers.

The current hormone therapy is not uniform and depends on the health care system, cost considerations, and differences in the regional availability of estrogens and antiandrogens. A typical regimen includes estrogen to provide feminizing effects in conjunction with therapy to block testosterone (antiandrogens or gonadotropin-releasing hormone \[GnRH\] analogs). Estrogen also inhibits testosterone secretion.

Ethinyl estradiol was previously the mainstay of most estrogen-directed therapies; this is no longer the case due to its increased risk of cardiovascular death and increased incidence of deep venous thrombosis. 17-beta estradiol, which can be provided in tablet, patch, and injection, is currently the preferred formulation.

This open label, pilot, randomized clinical trial will evaluate the effectiveness and safety of gender affirming hormone therapy with estrogen and the degree of testosterone suppression achieved in transgender female patients when placed on daily sublingual 17-beta estradiol, twice daily sublingual 17-beta estradiol, or transdermal 17-beta estradiol. All patients will also receive spironolactone as antiandrogen.

One of the major complications from estradiol GAHT is thromboembolism. The investigators will also determine the effects of the different estradiol regimens on thrombosis markers. These studies will be the first to determine if the dosing regimen of estradiol affects risk markers in transgender women.

Conditions

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Transgenderism

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Participants who agreed to be in the study will undergo block randomization into three groups:

1. daily sublingual 17 beta estradiol
2. twice daily sublingual 17 beta estradiol
3. transdermal 17 beta estradiol

All patients will also receive spironolactone as antiandrogen.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Transdermal estradiol plus spironolactone

Starting dose will be 100 mcg/24hrs Plan to increase by 100 mcg/24hrs every month to a max dose of 400 mcg/24hrs Goal is to achieve a serum estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Group Type ACTIVE_COMPARATOR

Transdermal patch

Intervention Type DRUG

Participants will be prescribed the medication and dosed based on their hormonal profile. Goal is achieve estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

Pro-thrombotic markers

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, pro-thombotic markers will be checked at baseline and every 6 months. Pro-thrombotic markers will include: Factor II, Factor IX, Factor XI, Von Willebrand factor, Protein C, Protein S, activated Protein C resistance.

Metabolic markers

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, metabolic markers will be checked at baseline and every 6 months. Markers will include: Insulin level, fasting glucose, body mass index, waist circumference.

Hormone Profile

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, hormonal levels will be checked every 4 weeks until estradiol and testosterone levels are within goal as established by standard of care.

Spironolactone

Intervention Type DRUG

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Daily sublingual estradiol plus spironolactone

Starting dose will be 2 mg daily Plan to increase every month by 2 mg daily Goal is to achieve serum estradiol level between 100-200 pg/mL mL and to suppress testosterone to cisgender female levels.

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Spironolactone will be started at 50 mg daily and will increase to standard dose.

Group Type ACTIVE_COMPARATOR

Pro-thrombotic markers

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, pro-thombotic markers will be checked at baseline and every 6 months. Pro-thrombotic markers will include: Factor II, Factor IX, Factor XI, Von Willebrand factor, Protein C, Protein S, activated Protein C resistance.

Metabolic markers

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, metabolic markers will be checked at baseline and every 6 months. Markers will include: Insulin level, fasting glucose, body mass index, waist circumference.

Hormone Profile

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, hormonal levels will be checked every 4 weeks until estradiol and testosterone levels are within goal as established by standard of care.

Daily Sublingual Tablet

Intervention Type DRUG

Participants will be prescribed the medication and dosed based on their hormonal profile. Participants will take dose as once daily medication. Goal is achieve estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

Spironolactone

Intervention Type DRUG

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Twice daily sublingual estradiol plus spironolactone

Starting dose will be 1 mg twice daily Plan to increase every month by 2 mg daily divided BID Goal is to achieve serum estradiol level between 100-200 pg/mL mL and to suppress testosterone to cisgender female levels

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Spironolactone will be started at 50 mg daily and will increase to standard dose.

Group Type ACTIVE_COMPARATOR

Pro-thrombotic markers

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, pro-thombotic markers will be checked at baseline and every 6 months. Pro-thrombotic markers will include: Factor II, Factor IX, Factor XI, Von Willebrand factor, Protein C, Protein S, activated Protein C resistance.

Metabolic markers

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, metabolic markers will be checked at baseline and every 6 months. Markers will include: Insulin level, fasting glucose, body mass index, waist circumference.

Hormone Profile

Intervention Type DIAGNOSTIC_TEST

Once participants are started on gender affirming hormone therapy, hormonal levels will be checked every 4 weeks until estradiol and testosterone levels are within goal as established by standard of care.

BID Sublingual Tablet

Intervention Type DRUG

Participants will be prescribed the medication and dosed based on their hormonal profile. Participants will take dose as twice daily medication. Goal is achieve estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

Spironolactone

Intervention Type DRUG

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Interventions

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Transdermal patch

Participants will be prescribed the medication and dosed based on their hormonal profile. Goal is achieve estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

Intervention Type DRUG

Pro-thrombotic markers

Once participants are started on gender affirming hormone therapy, pro-thombotic markers will be checked at baseline and every 6 months. Pro-thrombotic markers will include: Factor II, Factor IX, Factor XI, Von Willebrand factor, Protein C, Protein S, activated Protein C resistance.

Intervention Type DIAGNOSTIC_TEST

Metabolic markers

Once participants are started on gender affirming hormone therapy, metabolic markers will be checked at baseline and every 6 months. Markers will include: Insulin level, fasting glucose, body mass index, waist circumference.

Intervention Type DIAGNOSTIC_TEST

Hormone Profile

Once participants are started on gender affirming hormone therapy, hormonal levels will be checked every 4 weeks until estradiol and testosterone levels are within goal as established by standard of care.

Intervention Type DIAGNOSTIC_TEST

Daily Sublingual Tablet

Participants will be prescribed the medication and dosed based on their hormonal profile. Participants will take dose as once daily medication. Goal is achieve estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

Intervention Type DRUG

BID Sublingual Tablet

Participants will be prescribed the medication and dosed based on their hormonal profile. Participants will take dose as twice daily medication. Goal is achieve estradiol level between 100-200 pg/mL and to suppress testosterone to cisgender female levels

Intervention Type DRUG

Spironolactone

All patients will also receive spironolactone. Spironolactone will be started at 50 mg daily and will increase to standard dose.

Intervention Type DRUG

Other Intervention Names

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Daily Once daily dosing Twice daily dosing Daily

Eligibility Criteria

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Inclusion Criteria

* Female transgender patients between the ages of 18 to 30 years of age who are seen at the Washington University Transgender Center. Patients must have met the eligibility and readiness criteria for gender-affirming hormone therapy.

Exclusion Criteria

* GnRH agonist for the last 12 months
* History of liver disease
* Dyslipidemia requiring treatment
* Cigarette smoking
* Body mass index \>30 kg/m2
Minimum Eligible Age

18 Years

Maximum Eligible Age

30 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

Yes

Sponsors

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Washington University School of Medicine

OTHER

Sponsor Role lead

Responsible Party

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Ginger E Nicol

Associate Professor of Child & Adolescent Psychiatry

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Ginger Nicol, MD

Role: PRINCIPAL_INVESTIGATOR

Washington University School of Medicine

Locations

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Washington University Transgender Center

St Louis, Missouri, United States

Site Status

Countries

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United States

References

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Unger CA. Hormone therapy for transgender patients. Transl Androl Urol. 2016 Dec;5(6):877-884. doi: 10.21037/tau.2016.09.04.

Reference Type BACKGROUND
PMID: 28078219 (View on PubMed)

Kaltiala-Heino R, Bergman H, Tyolajarvi M, Frisen L. Gender dysphoria in adolescence: current perspectives. Adolesc Health Med Ther. 2018 Mar 2;9:31-41. doi: 10.2147/AHMT.S135432. eCollection 2018.

Reference Type BACKGROUND
PMID: 29535563 (View on PubMed)

Connolly MD, Zervos MJ, Barone CJ 2nd, Johnson CC, Joseph CL. The Mental Health of Transgender Youth: Advances in Understanding. J Adolesc Health. 2016 Nov;59(5):489-495. doi: 10.1016/j.jadohealth.2016.06.012. Epub 2016 Aug 17.

Reference Type BACKGROUND
PMID: 27544457 (View on PubMed)

Hamidi O, Davidge-Pitts CJ. Transfeminine Hormone Therapy. Endocrinol Metab Clin North Am. 2019 Jun;48(2):341-355. doi: 10.1016/j.ecl.2019.02.001. Epub 2019 Mar 23.

Reference Type BACKGROUND
PMID: 31027544 (View on PubMed)

Asscheman H, Giltay EJ, Megens JA, de Ronde WP, van Trotsenburg MA, Gooren LJ. A long-term follow-up study of mortality in transsexuals receiving treatment with cross-sex hormones. Eur J Endocrinol. 2011 Apr;164(4):635-42. doi: 10.1530/EJE-10-1038. Epub 2011 Jan 25.

Reference Type BACKGROUND
PMID: 21266549 (View on PubMed)

Angus LM, Nolan BJ, Zajac JD, Cheung AS. A systematic review of antiandrogens and feminization in transgender women. Clin Endocrinol (Oxf). 2021 May;94(5):743-752. doi: 10.1111/cen.14329. Epub 2020 Oct 5.

Reference Type BACKGROUND
PMID: 32926454 (View on PubMed)

Spanos C, Bretherton I, Zajac JD, Cheung AS. Effects of gender-affirming hormone therapy on insulin resistance and body composition in transgender individuals: A systematic review. World J Diabetes. 2020 Mar 15;11(3):66-77. doi: 10.4239/wjd.v11.i3.66.

Reference Type BACKGROUND
PMID: 32180895 (View on PubMed)

Bagot CN, Marsh MS, Whitehead M, Sherwood R, Roberts L, Patel RK, Arya R. The effect of estrone on thrombin generation may explain the different thrombotic risk between oral and transdermal hormone replacement therapy. J Thromb Haemost. 2010 Aug;8(8):1736-44. doi: 10.1111/j.1538-7836.2010.03953.x. Epub 2010 Jun 14.

Reference Type BACKGROUND
PMID: 20553380 (View on PubMed)

Hembree WC, Cohen-Kettenis PT, Gooren L, Hannema SE, Meyer WJ, Murad MH, Rosenthal SM, Safer JD, Tangpricha V, T'Sjoen GG. Endocrine Treatment of Gender-Dysphoric/Gender-Incongruent Persons: An Endocrine Society Clinical Practice Guideline. J Clin Endocrinol Metab. 2017 Nov 1;102(11):3869-3903. doi: 10.1210/jc.2017-01658.

Reference Type BACKGROUND
PMID: 28945902 (View on PubMed)

Bao AM, Liu RY, van Someren EJ, Hofman MA, Cao YX, Zhou JN. Diurnal rhythm of free estradiol during the menstrual cycle. Eur J Endocrinol. 2003 Feb;148(2):227-32. doi: 10.1530/eje.0.1480227.

Reference Type BACKGROUND
PMID: 12590642 (View on PubMed)

Cortez S, Moog D, Lewis C, Williams K, Herrick C, Fields M, Gray T, Guo Z, Nicol G, Baranski T. Effectiveness and Safety of Different Estradiol Regimens in Transgender Women (TREAT Study): Protocol for a Randomized Controlled Trial. JMIR Res Protoc. 2023 Dec 22;12:e53092. doi: 10.2196/53092.

Reference Type DERIVED
PMID: 38133914 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

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202104092

Identifier Type: -

Identifier Source: org_study_id

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