PCOSPhase 2/3NCT03981861
Metabolic and Neuro-Endocrine Effect of Treating PCOS in Adolescents
A Phase 2/3 study testing Metformin and Spironolactone in people with PCOS, run by Children's Mercy Hospital Kansas City at 1 site in United States.
Trial summary
To exam in the effect of a combination of low dose Metformin and Spironolactone on functional brain MRI, menstrual regulation and metabolism in adolescents with PCOS. Read more about this study
- Who is it for?
- Ages 13 to 21 years, women only
- Conditions the study is for
- PCOS
- What is being tested?
- Metformin and Spironolactone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jul 2016to 5 Jun 2019
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 21 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Generally healthy
- Meeting Androgen Excess Society (AES) diagnostic criteria of PCOS: Menstrual Dysfunction or PCO ovaries on ultrasound AND clinical or biochemical hyperandrogenism
- Normal liver and kidney function
- No chronic illnesses except for stable, treated hypothyroidism
Who cannot join
- Use of metformin and/or spironolactone within the last 6 months
- Currently on either oral hormonal contraception or other forms of hormonal contraception such as Depo-Provera, NuvaRing
- Current or past pregnancy
- Currently sexually active
- Psychiatric disorder based on self/parental report
- Type 2 diabetes (blood glucose > 200mg/dl on OGTT)
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- Anemia (Hct < 35)
- Impaired kidney function (Baseline creatinine > 1.0 mg)
- Abnormal liver transaminases > 2 x the upper limit of normal range
- Potassium elevated outside the reference range (in non-hemolyzed blood sample)
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Mercy Hospital
Kansas City, Missouri, 64108
About this study
To exam in the effect of a combination of low dose Metformin and Spironolactone on functional brain MRI, menstrual regulation and metabolism in adolescents with PCOS.
Read the detailed description
Polycystic Ovarian Syndrome (PCOS) is the most common endocrinopathy in adolescent females and also one of the most complex. Patients experience an exaggerated ovarian/adrenal androgen production in response to physiologic and supra-physiologic elevations in insulin. The hormonal dysregulation is not only associated with acne, hirsutism, and menstrual irregularity, but also with perpetuated insulin resistance, central adiposity, and clinical depression.
In the proposed study, we aim to treat a hormonally and metabolically well-defined group of adolescent girls with PCOS with a combination of two pharmacological agents: metformin (insulin sensitizer) and spironolactone (anti-androgen) for 6 months. Although hyperandrogenism is a fundamental component of PCOS and responsible for the perpetuation of insulin resistance, adiposity and anovulation, there are few pediatric studies that have examined the benefits of treating both insulin resistance and hyperandrogenism simultaneously. Preliminary studies in adults, however, suggest synergistic effect of both spironolactone and metformin (spiro-met) with near normalization of the metabolic and ovulatory dysfunction. Therefore, we hypothesize that spiro-met will improve adolescent metabolism, body composition, ovarian morphology/function. We anticipate that our study will generate key pilot data for further randomized, double blind placebo controlled trials using both agents.
We also plan to examine functional brain MRI before and after the spiro-met intervention. This will allow us to inspect the effects of the hyperinsulinemic/androgenic hormonal milieu in PCOS on structural and functional brain MRI. We hypothesis, that the hormonal environment in PCOS affects centers of appetite and mood.
- Conditions
- PCOS
- Being tested
- Metformin and Spironolactone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Metformin and SpironolactoneDrug
Oral Metformin tablet (500 mg/tablet) twice daily for 6 months. Oral Spironolactone tablet (50 mg/tablet) once daily for 6 months.
| Study group | Role | What they receive |
|---|---|---|
| Overall Study | Experimental | Treatment with Metformin and Spironolactone Receives: Drug: Metformin and Spironolactone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Oral Glucose Tolerance Test
Measurement of glucose and insulin at baseline and 2 hours after 75g of glucola.
Measured: Change Measures: Baseline & 6 Months
Total Testosterone
Total Testosterone measured in ng/dL
Measured: Change Measures: Baseline & 6 Months
Free Testosterone
Free Testosterone measured in ng/dL
Measured: Change Measures: Baseline & 6 Months
Dehydroepiandrosterone Sulfate (DHEAS)
DHEAS measured in mcg/dL
Measured: Change Measures: Baseline & 6 Months
Body Mass Index (BMI)
Body Mass Index measured in kg/m2
Measured: Change Measures: Baseline & 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jun 2019
- Study start2 Jul 2016
- Primary completion9 Jul 2018
- Study completion5 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for PCOSStudies of the same condition, open and under way.
- 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
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Tania Burgert, MD, Principal investigator, Children's Mercy Hospital Kansas City
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03981861
- Sponsor's protocol ID
- 16010020
- Status last verified
- Feb 2021
- First submitted
- 7 Jun 2019
- Last updated
- 23 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Metabolic and Neuro-Endocrine Effect of Treating PCOS in Adolescents
- ClinicalTrials.gov ID
- NCT03981861
- Acronym / sponsor ID
- 16010020
- Conditions
- PCOS
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Children's Mercy Hospital Kansas CityUniversity, hospital or other
- Study start
- 2 Jul 2016 (actual)
- Primary completion
- 9 Jul 2018 (actual)
- Study completion
- 5 Jun 2019 (actual)
- First posted
- 11 Jun 2019 (actual)
- Results
- Posted 23 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To exam in the effect of a combination of low dose Metformin and Spironolactone on functional brain MRI, menstrual regulation and metabolism in adolescents with PCOS.
Detailed description
Polycystic Ovarian Syndrome (PCOS) is the most common endocrinopathy in adolescent females and also one of the most complex. Patients experience an exaggerated ovarian/adrenal androgen production in response to physiologic and supra-physiologic elevations in insulin. The hormonal dysregulation is not only associated with acne, hirsutism, and menstrual irregularity, but also with perpetuated insulin resistance, central adiposity, and clinical depression.
In the proposed study, we aim to treat a hormonally and metabolically well-defined group of adolescent girls with PCOS with a combination of two pharmacological agents: metformin (insulin sensitizer) and spironolactone (anti-androgen) for 6 months. Although hyperandrogenism is a fundamental component of PCOS and responsible for the perpetuation of insulin resistance, adiposity and anovulation, there are few pediatric studies that have examined the benefits of treating both insulin resistance and hyperandrogenism simultaneously. Preliminary studies in adults, however, suggest synergistic effect of both spironolactone and metformin (spiro-met) with near normalization of the metabolic and ovulatory dysfunction. Therefore, we hypothesize that spiro-met will improve adolescent metabolism, body composition, ovarian morphology/function. We anticipate that our study will generate key pilot data for further randomized, double blind placebo controlled trials using both agents.
We also plan to examine functional brain MRI before and after the spiro-met intervention. This will allow us to inspect the effects of the hyperinsulinemic/androgenic hormonal milieu in PCOS on structural and functional brain MRI. We hypothesis, that the hormonal environment in PCOS affects centers of appetite and mood.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Overall Study | Experimental | Drug: Metformin and SpironolactoneTreatment with Metformin and Spironolactone |
Who may qualify
- Ages
- 13 to 21 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Generally healthy
- Meeting Androgen Excess Society (AES) diagnostic criteria of PCOS: Menstrual Dysfunction or PCO ovaries on ultrasound AND clinical or biochemical hyperandrogenism
- Normal liver and kidney function
- No chronic illnesses except for stable, treated hypothyroidism
Key exclusion criteria (10 listed)
- Use of metformin and/or spironolactone within the last 6 months
- Currently on either oral hormonal contraception or other forms of hormonal contraception such as Depo-Provera, NuvaRing
- Current or past pregnancy
- Currently sexually active
- Psychiatric disorder based on self/parental report
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 |
|---|---|---|
| Metformin and SpironolactoneOral Metformin tablet (500 mg/tablet) twice daily for 6 months. Oral Spironolactone tablet (50 mg/tablet) once daily for 6 months. | Drug | Overall Study |
Primary outcome
Oral Glucose Tolerance Test
Time frame: Change Measures: Baseline & 6 Months
Measurement of glucose and insulin at baseline and 2 hours after 75g of glucola.
Total Testosterone
Time frame: Change Measures: Baseline & 6 Months
Total Testosterone measured in ng/dL
Free Testosterone
Time frame: Change Measures: Baseline & 6 Months
Free Testosterone measured in ng/dL
Dehydroepiandrosterone Sulfate (DHEAS)
Time frame: Change Measures: Baseline & 6 Months
DHEAS measured in mcg/dL
Body Mass Index (BMI)
Time frame: Change Measures: Baseline & 6 Months
Body Mass Index measured in kg/m2
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for PCOS
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Efficacy of Injectable Vitamin D Supplementation in Females With Polycystic Ovary Syndrome
Anovulatory Infertility
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The Value of LC-MS/MS Detection of Androgen Profiles in the Diagnosis of PCOS: A Prospective, Multicenter Real-world Study
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The Effects of the Mediterranean Diet and Synbiotics in Polycystic Ovary Syndrome
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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.
Tania Burgert, MD principal investigator
Children's Mercy Hospital Kansas City
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Metformin and Spironolactone in people with PCOS, run by Children's Mercy Hospital Kansas City 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 4 inclusion and 10 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: finished, as of the sponsor's last update on 23 Feb 2021. 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 23 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Oral Glucose Tolerance Test (time frame: Change Measures: Baseline & 6 Months). The study lists 5 primary outcomes in all. 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 23 Feb 2021.