Polycystic Ovary SyndromeEarly Phase 1CBS009NCT04075149
Does Treatment of Androgen Excess Using Spironolactone Improve Ovulatory Rates in Girls With Androgen Excess?
A Early Phase 1 study testing Spironolactone in people with Polycystic Ovary Syndrome and Puberty Disorders, run by University of Virginia at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 13 to 19 years, women only
- Conditions the study is for
- Polycystic Ovary Syndrome, Puberty Disorders, Ovulation Disorder
- What is being tested?
- Spironolactone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Dec 2019to 1 Sep 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 19 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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)
Show all 19Show fewer
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of Virginia Center for Research in Reproduction
Charlottesville, Virginia, 22908
Melissa Gilrain, 434-243-6911, pcos@virginia.edu
Christine Burt Solorzano, MD
About this study
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.
Read the detailed description
This is a non-randomized, clinical pilot study testing whether 16 weeks of spironolactone improves ovulation rates in post-menarcheal girls with androgen excess. The investigators will recruit girls ages 13-19 with clinical or biochemical androgen excess (defined as hirsutism and/or elevated Tanner stage-specific free testosterone concentrations) who are 4 to 6 years post-menarche. Girls will keep a menstrual diary and collect twice weekly saliva samples for progesterone and estradiol for 16 weeks before and for 16 weeks during spironolactone (if ≤ 60 kg, 50 mg twice daily; if > 60 kg, 100 mg twice daily [1.7-3.3 mg/kg/24 hr]) use. Girls will be asked to continue to collect saliva twice weekly for progesterone and estradiol testing for an additional 16 weeks after discontinuation of spironolactone and to continue menstrual diaries off of study medication for 12 additional months to assess persistence of any spironolactone effect. Ovulation events will be counted per 100 days based on definitive rises in salivary progesterone. The primary outcome will be changes in ovulation rate during spironolactone use compared to baseline. Changes in menstrual regularity via inter-menstrual interval will also be assessed as a secondary outcome of spironolactone administration. Another secondary outcome will be changes in acne and/or hirsutism.
- Being tested
- Spironolactone
- Keywords
- PCOS, Polycystic Ovary Syndrome, ovulation, puberty, hyperandrogenemia, androgen excess
- Medical subject headings
- Polycystic Ovary Syndrome, Hyperandrogenism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SpironolactoneDrug
Also called Aldactone
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.
| Study group | Role | What they receive |
|---|---|---|
| Spironolactone | Experimental | 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) Receives: Drug: Spironolactone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ovulation Rate Changes
Ovulation rate during and after spironolactone compared to before medication. Ovulation will be counted based on salivary progesterone levels. The rate of ovulation will be defined as the number of ovulatory cycles per 100 days of follow-up.
Measured: Rates of ovulation during weeks 17-32 (on medication) and weeks 33-48 (after medication) compared to weeks 1-16 (baseline, prior to medication)
Secondary outcomes (3)
Menstrual Regularity Changes
Menstrual length during and after spironolactone compared to before medication. Changes in menstrual regularity will be assessed using inter-menstrual interval (IMI)
Measured: IMI for weeks 1-16 (before medication, baseline), weeks 17-32 (on medication), and weeks 33-52 (after medication)
Acne
Changes in acne before vs. after spironolactone treatment assessed using the Global Acne Grading System
Measured: weeks 16 and 32
Hirsutism
Changes in hirsutism before vs. after spironolactone assessed using a modified Ferriman-Gallwey scoring system
Measured: weeks 16 and 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2019
- Study start18 Dec 2019
- Primary completion1 Sep 2024 (estimated)
- Study completion1 Sep 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycystic Ovary SyndromeStudies of the same condition, open and under way.
- More trials run by University of VirginiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- non-randomized cross-sectional, before and after intervention
- Masking
- None (open label)
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Virginia
- Sponsor type
- University, hospital or other
- Collaborators
- The Waterloo Foundation
- Responsible party
- Principal investigator, Christine Burt Solorzano, University of Virginia
- Investigators
- Christine Burt Solorzano, MD, Principal investigator, University of Virginia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04075149
- Sponsor's protocol ID
- 21768
- Acronym
- CBS009
- Status last verified
- Oct 2023
- First submitted
- 29 Aug 2019
- Last updated
- 27 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Does Treatment of Androgen Excess Using Spironolactone Improve Ovulatory Rates in Girls With Androgen Excess?
- ClinicalTrials.gov ID
- NCT04075149
- Acronym / sponsor ID
- CBS009 / 21768
- Keywords
- PCOS, Polycystic Ovary Syndrome, ovulation, puberty, hyperandrogenemia, androgen excess
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- University of VirginiaUniversity, hospital or other
- Collaborators
- The Waterloo Foundation
- Study start
- 18 Dec 2019 (actual)
- Primary completion
- 1 Sep 2024 (estimated)
- Study completion
- 1 Sep 2024 (estimated)
- First posted
- 30 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a non-randomized, clinical pilot study testing whether 16 weeks of spironolactone improves ovulation rates in post-menarcheal girls with androgen excess. The investigators will recruit girls ages 13-19 with clinical or biochemical androgen excess (defined as hirsutism and/or elevated Tanner stage-specific free testosterone concentrations) who are 4 to 6 years post-menarche. Girls will keep a menstrual diary and collect twice weekly saliva samples for progesterone and estradiol for 16 weeks before and for 16 weeks during spironolactone (if ≤ 60 kg, 50 mg twice daily; if > 60 kg, 100 mg twice daily [1.7-3.3 mg/kg/24 hr]) use. Girls will be asked to continue to collect saliva twice weekly for progesterone and estradiol testing for an additional 16 weeks after discontinuation of spironolactone and to continue menstrual diaries off of study medication for 12 additional months to assess persistence of any spironolactone effect. Ovulation events will be counted per 100 days based on definitive rises in salivary progesterone. The primary outcome will be changes in ovulation rate during spironolactone use compared to baseline. Changes in menstrual regularity via inter-menstrual interval will also be assessed as a secondary outcome of spironolactone administration. Another secondary outcome will be changes in acne and/or hirsutism.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- non-randomized cross-sectional, before and after intervention
| Arm | Type | Receives |
|---|---|---|
| Spironolactone | Experimental | Drug: Spironolactone16 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) |
Who may qualify
- Ages
- 13 to 19 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (19 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SpironolactoneAlso: AldactoneSpironolactone 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. | Drug | Spironolactone |
Primary outcome
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)
Ovulation rate during and after spironolactone compared to before medication. Ovulation will be counted based on salivary progesterone levels. The rate of ovulation will be defined as the number of ovulatory cycles per 100 days of follow-up.
3 secondary outcomes
Menstrual Regularity Changes
Time frame: IMI for weeks 1-16 (before medication, baseline), weeks 17-32 (on medication), and weeks 33-52 (after medication)
Acne
Time frame: weeks 16 and 32
Hirsutism
Time frame: weeks 16 and 32
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christine Burt Solorzano, MD principal investigator
University of Virginia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Spironolactone in people with Polycystic Ovary Syndrome and Puberty Disorders, run by University of Virginia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 27 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Melissa Gilrain, BS (434-243-6911, pcos@virginia.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2023.