What side effects are associated with this type of intervention?

Common treatments for Polycystic Ovary Syndrome (PCOS) include combination oral contraceptives (COCs) and antiandrogens like flutamide and spironolactone. COCs can increase the risk of venous thromboembolism (VTE), particularly in women over 40 and those with obesity. Antiandrogens, such as flutamide and spironolactone, can cause side effects like liver toxicity, gastrointestinal disturbances, and potential teratogenic effects, necessitating concurrent use of contraception. Both treatments may also have modest effects on metabolic abnormalities associated with PCOS.

What prior FDA approvals exist?

The FDA has approved several treatments for managing symptoms of Polycystic Ovary Syndrome (PCOS), including clomiphene citrate for ovulation induction and metformin for improving insulin sensitivity. While flutamide, a testosterone blocker, is being studied for its potential benefits in PCOS, it is not specifically FDA-approved for this condition. However, spironolactone, another antiandrogen, is commonly used off-label to treat hirsutism associated with PCOS. Combined oral contraceptives are also frequently prescribed to manage menstrual irregularities and hyperandrogenic symptoms in PCOS patients.

What other types of research exist?

Promising novel alternatives to Flutamide for PCOS treatment include Letrozole, which is suggested as a first-line therapy for ovulation induction in PCOS patients, and Metformin, which has been shown to improve ovulation and pregnancy rates. Additionally, antiandrogens like Spironolactone, often used in combination with oral contraceptives, are effective in reducing hirsutism and other androgenic symptoms in PCOS.

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Antiandrogen

Hormone Therapy for Polycystic Ovary Syndrome (CRM010 Trial)

Phase < 1

Recruiting

Led By Christopher M McCartney, MD

Research Sponsored by University of Virginia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Post-pubertal (> 4 years post-menarche) adult woman aged 18-30 years

Willing to strictly avoid pregnancy with use of reliable non-hormonal methods during the study period

Must not have

History and/or physical exam findings suggestive of Cushing's syndrome, adrenal insufficiency, or acromegaly

Previous diagnosis of diabetes, fasting glucose > or = 126 mg/dl, or a hemoglobin A1c > or = 6.5%

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after 4 weeks of placebo administration

Summary

This trial is testing if flutamide, which blocks testosterone, can help women with PCOS improve their hormone regulation. The study aims to see if this medication can enhance the body's response to progesterone. Flutamide has been used to treat high levels of male hormones in women with PCOS, but its use is limited by potential liver damage and cost.

Who is the study for?

This trial is for post-pubertal women aged 18-30 with PCOS, showing signs of excess male hormones and irregular periods. Participants must be in good health, not pregnant or lactating, avoid pregnancy during the study, and have no liver issues or significant medical history like diabetes or cancer. They should weigh over 110 pounds without severe blood disorders.

What is being tested?

The study tests if flutamide can improve pituitary function in women with PCOS when pre-treated with estradiol and given progesterone. It's a randomized, placebo-controlled double-blinded crossover study to see if flutamide enhances FSH release.

What are the potential side effects?

Flutamide may cause liver problems; participants will be monitored for this. Other side effects could include allergic reactions to any of the drugs used: micronized progesterone, transdermal estradiol, or flutamide itself.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am a woman aged 18-30 and have been menstruating for over 4 years.

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I am committed to using non-hormonal birth control methods during the study.

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I am able and willing to agree to the study's procedures.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I might have Cushing's syndrome, adrenal insufficiency, or acromegaly based on my symptoms or doctor's exam.

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I have diabetes or my blood sugar/A1c levels are high.

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My kidney function is reduced with a GFR under 60.

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My liver function tests are normal.

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I have signs of increased male traits like more body hair, deeper voice, or enlarged clitoris.

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I am not allergic to micronized progesterone, flutamide, or transdermal estradiol.

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I am either younger than 18 or older than 30.

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My testosterone level is above 150 ng/dl, indicating a possible tumor.

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My BMI is either below 18 or above 40.

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I have had breast, ovarian, or endometrial cancer before.

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I am female.

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I am unable to understand or sign the consent form.

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I have a serious heart or lung condition.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after 4 weeks of placebo administration

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after 4 weeks of placebo administration

This trial's timeline: 3 weeks for screening, Varies for treatment, and after 4 weeks of placebo administration for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in mean FSH concentration with progesterone administration

Secondary study objectives

Change in mean LH concentration with progesterone administration

Side effects data

From 2011 Phase 4 trial • 115 Patients • NCT00611923

38%

headache

31%

stomach pain

23%

dry skin

15%

nausea

15%

hot flushes

15%

insomnia

15%

Diarrhea

fatigue

leg pain

parasthesia

dry mouth

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Flutamide

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: FlutamideExperimental Treatment3 Interventions

Prior to the first or second admission (randomly determined), subjects will be pretreated for 4 weeks with Flutamide (250 mg twice daily)

Group II: PlaceboPlacebo Group3 Interventions

Prior to the first or the second admission (randomly determined), participants will be pretreated for 2 weeks with placebo (twice daily).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Flutamide

2013

Completed Phase 4

~2110

Micronized progesterone

2021

Completed Phase 4

~1730

Estradiol patch

2019

Completed Phase 1

~530

0 / 16Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Polycystic Ovary Syndrome (PCOS) include antiandrogens like flutamide, combined oral contraceptives (COCs), and metformin. Flutamide works by blocking androgen receptors, reducing the effects of testosterone, which helps alleviate symptoms like hirsutism and acne. COCs combine estrogen and progestin to regulate menstrual cycles, reduce androgen levels, and prevent endometrial hyperplasia. Metformin, an insulin sensitizer, improves insulin resistance and can help restore regular ovulatory cycles. These treatments are crucial for managing the diverse symptoms of PCOS, including hyperandrogenism, menstrual irregularities, and metabolic issues, thereby improving the overall quality of life for patients.

First-line ovulation induction for polycystic ovary syndrome: an individual participant data meta-analysis.Targets to treat androgen excess in polycystic ovary syndrome.Combined oral contraceptives and/or antiandrogens versus insulin sensitizers for polycystic ovary syndrome: a systematic review and meta-analysis.