Recruiting
Early Phase 1

Spironolactone

Sponsor:

University of Virginia

Code:

NCT04075149

Conditions

Polycystic Ovary Syndrome

Puberty Disorders

Ovulation Disorder

Eligibility Criteria

Sex: Female

Age: 13 - 19

Healthy Volunteers: Not accepted

Interventions

Spironolactone

Study Details

Brief summary:

Adolescent girls with androgen excess have a higher rate of irregular periods and decreased ovulation rates compared to normal girls, and are considered at-risk for developing polycystic ovary syndrome (PCOS). This pilot study will look at whether giving spironolactone might improve ovulation rates in girls with androgen excess, ages 13-19. If this is true, spironolactone treatment to young girls might prevent PCOS from developing and avoid future infertility.

Conditions

Polycystic Ovary Syndrome

Puberty Disorders

Ovulation Disorder

Study ID

NCT04075149

Start date

Dec 18, 2019

Status verified date

Oct, 2023

Completion date

Sep 1, 2024

Anticipated

Primary completion date

Sep 1, 2024

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 13 - 19

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Girls age 13 - 19 years, with clinical or biochemical HA (defined as hirsutism and/or elevated Tanner stage-specific free testosterone concentrations) that are 4-6 years post-menarche
  • Screening labs within age-appropriate normal range
  • Volunteers who are 18-19 y old must be willing and able to provide written informed consent.
  • When the subject is a minor (i.e., age < 18 y), the subject and custodial parents must be willing and able to provide written informed assent and consent, respectively.
  • Willingness to strictly avoid pregnancy (using non-hormonal methods) during the time of study.

Exclusion Criteria:

  • Age < 13 or > 19 y
  • Girls ≤ 3 years and ≥ 7 years post-menarche will be excluded
  • Being a study of androgen excess in adolescent girls with HA, men and boys are excluded
  • Inability to comprehend what will be done during the study or why it will be done
  • Precocious puberty (breast development before age 7)
  • Primary amenorrhea (no menses by age 16)
  • BMI-for-age < 5th percentile
  • Patients currently enrolled in another research protocol will be excluded, except for those enrolled in IRB-HSR 17633 DENND1A
  • Obesity due to genetic syndrome (e.g. Prader-Willi syndrome)
  • Cushing syndrome
  • Positive pregnancy test or lactation. Subjects with a positive pregnancy test will be informed of the result by the screening physician. Under Virginia law, parental notification is not required for minors. However, the screening physician will encourage them to tell their parent(s) and counsel them about the importance of appropriate prenatal care and counseling. We will arrange follow-up for them at the Teen Health Clinic at the University of Virginia or their primary care physician's office in a timely manner.
  • Diabetes mellitus
  • History of congenital adrenal hyperplasia or 17-hydroxyprogesterone > 300 ng/dL in the follicular phase, which suggests the possibility of congenital adrenal hyperplasia. NOTE: If a 17-hydroxyprogesterone > 300 ng/dL is confirmed on repeat testing, and ACTH-stimulated 17-hydroxyprogesterone < 1000 ng/dL will be required for study participation.
  • Total testosterone > 150 ng/dL
  • Abnormal thyroid stimulating hormone (TSH) for age. Subjects with adequately treated hypothyroidism, reflected by normal TSH values, will not be excluded.
  • Abnormal sodium, potassium, or bicarbonate concentrations, or elevated creatine concentration (confirmed on repeat)
  • Subjects must not take exogenous steroids or any medications known to affect the reproductive axis or glucose metabolism for 3 months prior to the study (or in the 2 months prior to screening). Such medications include oral contraceptives, progestins, metformin, glucocorticoids, and antipsychotic medications
  • If sexually active, subjects will be required to abstain and/or use barrier forms of contraception during the study.

Note: Abnormal laboratory studies may be confirmed by repeat testing to exclude laboratory error.

Study Design

Enrollment

24 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Spironolactone

16 weeks without medication, then 16 weeks with medication, then 12 months without medication; spironolactone 50 mg tablets: 50-100 mg orally twice daily (1.7-3.3 mg/kg/24 hr)

Interventions

Spironolactone

Spironolactone is an androgen-receptor commonly used (off-label) for hyperandrogenism in girls and women. It is used in this study to determine if androgen blockade can improve ovulation rates in girls with androgen excess, who often have low rates of ovulation in the years following menarche. The spironolactone dose will be as follows: If ≤ 60 kg, 50 mg twice daily; if > 60 kg, 100 mg twice daily.

Primary outcome measure

  • Ovulation Rate Changes [ Time Frame: Rates of ovulation during weeks 17-32 (on medication) and weeks 33-48 (after medication) compared to weeks 1-16 (baseline, prior to medication) ]

Central Contacts and Locations

Central contacts

Christine Burt Solorzano, MD

434-243-6911pcos@virginia.edu

Locations

University of Virginia Center for Research in Reproduction

Recruiting

Charlottesville, Virginia, United States, 22908

Contacts

Principal Investigator:

Christine Burt Solorzano, MD

More Information

Sponsor

University of Virginia

Last update posted

Oct 27, 2023

Last verified

Oct, 2023

Keywords

  • PCOS
  • Polycystic Ovary Syndrome
  • ovulation
  • puberty
  • hyperandrogenemia
  • androgen excess

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by University of Virginia on 2023-10-27.