Polycystic Ovary SyndromeNCT00166569
The Genetics of Polycystic Ovarian Syndrome
An observational study of Polycystic Ovary Syndrome, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
Trial summary
The goal of this study is to determine the genetic basis of polycystic ovary syndrome (PCOS). We will first look for genes in the Icelandic population, where large family trees are known and it is easier to search for genes. We will then determine whether these same genes are important in U.S. PCOS patients. Read more about this study
- Who is it for?
- Ages 18 years and older, women onlyHealthy volunteers can take part
- Conditions the study is for
- Polycystic Ovary Syndrome
- How many people
- 2,700 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2003to Dec 2025
- Phase
- Observational
- 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 18 years and older
- Women only
- Healthy volunteers accepted
- Adult, Older adult
Study population: Women with PCOS age 18 to 40 years, sampled from the population and controls without PCOS.
Who may be able to join
- Inclusion Criteria-PCOS Probands: 1) Aged 18 yrs or above 2) Oligomenorrhea or amenorrhea (<9 menses/yr); 3) clinical and/or biochemical evidence of hyperandrogenemia; 4) normal TSH and prolactin <25 ng/mL; 5) on no hormonal or insulin sensitizing medication for at least 3 months and have not taken Provera for at least ten days prior to enrollment. If the subject has previously had hormone levels drawn and processed in the Reproductive Endocrine Laboratory, it is not necessary to discontinue hormonal or insulin sensitizing medication.
- A second group of PCOS probands with a documented diagnosis of PCOS will also be recruited. These subjects will meet all of the criteria above except that they will be on hormonal medication.
- Inclusion Criteria-Female Unaffected Relatives: 1) Aged at 18 yrs or above; 2) Regular menstrual cycles 21-35 days or history of regular menstrual cycles in the past if menopausal; 3) no clinical or biochemical evidence of hyperandrogenism; 4) normal TSH and prolactin <25 ng/mL; 5) on no hormonal or insulin sensitizing medication for at least 3 months.
- Inclusion Criteria-Male Relatives: 1) Aged 18 yrs or above; 2) normal TSH and prolactin <25 ng/mL; 5) on no hormonal or insulin sensitizing medication for at least 3 months.
- Inclusion Criteria-Control Subjects: All control subjects will meet the criteria outlined for the female unaffected relatives. In Iceland, control subjects will be recruited from the Icelandic national registry, matched by age and sex to PCOS subjects and their family members. They will otherwise be recruited at random. In Boston, control subjects will be recruited from email and newspaper listings.
Who cannot join
- Exclusion Criteria PCOS Probands: Subjects will not have 1) late onset congenital adrenal hyperplasia as defined by a fasting 17OH progesterone level <200 ng/mL or a cortrosyn stimulated 17 OH progesterone level <500 ng/mL.
- Exclusion Criteria (Female unaffected relatives): None. Exclusion Criteria Male Relatives: None. Exclusion Criteria-Control Subjects: Control subjects chosen at random will be excluded if they are already participating in the study as a PCOS subject or are a first degree family member of the PCOS subject.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of Utah
Salt Lake City, Utah, 84112
Corrine Welt, MD, 801-585-1875, cwelt@genetics.utah.edu
Corrine K Welt, MD, 801-585-1875 ext. 8015851875, cwelt@genetics.utah.edu
About this study
The goal of this study is to determine the genetic basis of polycystic ovary syndrome (PCOS). We will first look for genes in the Icelandic population, where large family trees are known and it is easier to search for genes. We will then determine whether these same genes are important in U.S. PCOS patients.
Read the detailed description
Polycystic Ovary Syndrome (PCOS) is one of the most common endocrine disorders in women. Its marked phenotypic variability, puzzling list of associated conditions, and hence potential risk to a woman's health over her lifecycle clearly remain a clinical investigational, genetic, and therapeutic challenge. To a great extent, this variability can be attributed to the marked genetic and environmental heterogeneity of the populations studied. We plan to address many of these long-term clinical research obstacles by delineating the genetic basis of PCOS in a unique investigational venue, the Icelandic population. Studying the Icelandic population has the following advantages: a) the population's relative genetic and environmental homogeneity; b) the remarkably rich genealogic database; and c) the centralized healthcare information. In this unusual venue, clustering of large numbers of PCOS families with known relationships is possible. Their relative homogeneity and genealogic characteristics can reduce genetic variance and hence be used to map the genetic basis for PCOS more efficiently than in more heterogeneous populations. As the genes responsible for PCOS emerge, this protocol will also determine phenotype/genotype correlations in Iceland, contrasting them with US women, and assess their long-term medical consequences.
Each female subject will be asked to arrive fasting in the morning. Women with PCOS will be asked to arrive at least 10 days from their last menstrual period, while women with regular menstrual cycles will be seen within the first 14 days after their menstrual period starts. All subjects will be asked to fill out an extensive questionnaire. Subjects will then undergo further history, physical exam and laboratory exams. Blood will be drawn at baseline for DNA (the material in the cell that holds the genes), fasting blood glucose and insulin, HbA1C, total and fractionated cholesterol, triglycerides, testosterone, androstenedione, DHEAS, SHBG and 17-OH Progesterone. Additional blood will be drawn at 10 and 20 minutes for measurement of LH and FSH, which are pulsatile. An oral glucose tolerance test will be optional. A standardized transvaginal ultrasound will be performed to look at the ovaries in all female subjects. This can be done over the abdomen with a full bladder if the patient prefers.
Male family members will also undergo a history, physical and laboratory exams in an identical manner to that of their female family members with the addition that specific notice of their hair distribution will be made. PCOS subjects who are on oral contraceptives or other hormonal medication may have cholesterol, insulin, glucose, triglycerides and HbA1C labs drawn, whereas subjects on insulin sensitizing agents will not have any blood drawn for these tests. These subjects will undergo an ultrasound and DNA sampling.
All hormone blood samples from Icelandic and Boston subjects will be examined in Boston. All DNA testing will be performed in the Genotyping Core of deCODE, a company in Iceland. Therefore, all DNA samples from Iceland and Boston will be analyzed in Iceland. The Boston blood samples will be coded and sent to the DeCODE genotyping facility. The investigators in Boston will retain the link between the code and the subject information in a locked area in the office. The names of subjects will not be disclosed to deCODE.
- Conditions
- Polycystic Ovary Syndrome
- Keywords
- Polycystic Ovary Syndrome
- Medical subject headings
- Polycystic Ovary Syndrome
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2005 (estimated)
- Study startJan 2003
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Polycystic Ovary SyndromeStudies 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 2,700 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Principal investigator, Corrine Welt, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Investigators
- Corrine Welt, MD, Principal investigator
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00166569
- Sponsor's protocol ID
- 5U01HD044417-03
- Status last verified
- Mar 2020
- First submitted
- 9 Sep 2005
- Last updated
- 3 Mar 2020
- Results posted
- No
- Data monitoring committee
- No
Publications
Saxena R, Bjonnes AC, Georgopoulos NA, Koika V, Panidis D, Welt CK. Gene variants associated with age at menopause are also associated with polycystic ovary syndrome, gonadotrophins and ovarian volume. Hum Reprod. 2015 Jul;30(7):1697-703. doi: 10.1093/humrep/dev110. Epub 2015 May 20.
PubMed 25994816Saxena R, Georgopoulos NA, Braaten TJ, Bjonnes AC, Koika V, Panidis D, Welt CK. Han Chinese polycystic ovary syndrome risk variants in women of European ancestry: relationship to FSH levels and glucose tolerance. Hum Reprod. 2015 Jun;30(6):1454-9. doi: 10.1093/humrep/dev085. Epub 2015 Apr 22.
PubMed 25904635Pau C, Saxena R, Welt CK. Evaluating reported candidate gene associations with polycystic ovary syndrome. Fertil Steril. 2013 May;99(6):1774-8. doi: 10.1016/j.fertnstert.2012.12.033. Epub 2013 Jan 30.
PubMed 23375202
Study at a glance
From the registry record, last updated by the sponsor on 3 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Genetics of Polycystic Ovarian Syndrome
- ClinicalTrials.gov ID
- NCT00166569
- Acronym / sponsor ID
- 5U01HD044417-03
- Conditions
- Polycystic Ovary Syndrome
- Keywords
- Polycystic Ovary Syndrome
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 2,700 participants (estimated)
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)US National Institutes of Health
- Study start
- Jan 2003
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 14 Sep 2005 (estimated)
- 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.
The goal of this study is to determine the genetic basis of polycystic ovary syndrome (PCOS). We will first look for genes in the Icelandic population, where large family trees are known and it is easier to search for genes. We will then determine whether these same genes are important in U.S. PCOS patients.
Detailed description
Polycystic Ovary Syndrome (PCOS) is one of the most common endocrine disorders in women. Its marked phenotypic variability, puzzling list of associated conditions, and hence potential risk to a woman's health over her lifecycle clearly remain a clinical investigational, genetic, and therapeutic challenge. To a great extent, this variability can be attributed to the marked genetic and environmental heterogeneity of the populations studied. We plan to address many of these long-term clinical research obstacles by delineating the genetic basis of PCOS in a unique investigational venue, the Icelandic population. Studying the Icelandic population has the following advantages: a) the population's relative genetic and environmental homogeneity; b) the remarkably rich genealogic database; and c) the centralized healthcare information. In this unusual venue, clustering of large numbers of PCOS families with known relationships is possible. Their relative homogeneity and genealogic characteristics can reduce genetic variance and hence be used to map the genetic basis for PCOS more efficiently than in more heterogeneous populations. As the genes responsible for PCOS emerge, this protocol will also determine phenotype/genotype correlations in Iceland, contrasting them with US women, and assess their long-term medical consequences.
Each female subject will be asked to arrive fasting in the morning. Women with PCOS will be asked to arrive at least 10 days from their last menstrual period, while women with regular menstrual cycles will be seen within the first 14 days after their menstrual period starts. All subjects will be asked to fill out an extensive questionnaire. Subjects will then undergo further history, physical exam and laboratory exams. Blood will be drawn at baseline for DNA (the material in the cell that holds the genes), fasting blood glucose and insulin, HbA1C, total and fractionated cholesterol, triglycerides, testosterone, androstenedione, DHEAS, SHBG and 17-OH Progesterone. Additional blood will be drawn at 10 and 20 minutes for measurement of LH and FSH, which are pulsatile. An oral glucose tolerance test will be optional. A standardized transvaginal ultrasound will be performed to look at the ovaries in all female subjects. This can be done over the abdomen with a full bladder if the patient prefers.
Male family members will also undergo a history, physical and laboratory exams in an identical manner to that of their female family members with the addition that specific notice of their hair distribution will be made. PCOS subjects who are on oral contraceptives or other hormonal medication may have cholesterol, insulin, glucose, triglycerides and HbA1C labs drawn, whereas subjects on insulin sensitizing agents will not have any blood drawn for these tests. These subjects will undergo an ultrasound and DNA sampling.
All hormone blood samples from Icelandic and Boston subjects will be examined in Boston. All DNA testing will be performed in the Genotyping Core of deCODE, a company in Iceland. Therefore, all DNA samples from Iceland and Boston will be analyzed in Iceland. The Boston blood samples will be coded and sent to the DeCODE genotyping facility. The investigators in Boston will retain the link between the code and the subject information in a locked area in the office. The names of subjects will not be disclosed to deCODE.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Women with PCOS age 18 to 40 years, sampled from the population and controls without PCOS.
Key inclusion criteria (5 listed)
- Inclusion Criteria-PCOS Probands: 1) Aged 18 yrs or above 2) Oligomenorrhea or amenorrhea (<9 menses/yr); 3) clinical and/or biochemical evidence of hyperandrogenemia; 4) normal TSH and prolactin <25 ng/mL; 5) on no hormonal or insulin sensitizing medication for at least 3 months and have not taken Provera for at least ten days prior to enrollment. If the subject has previously had hormone levels drawn and processed in the Reproductive Endocrine Laboratory, it is not necessary to discontinue hormonal or insulin sensitizing medication.
- A second group of PCOS probands with a documented diagnosis of PCOS will also be recruited. These subjects will meet all of the criteria above except that they will be on hormonal medication.
- Inclusion Criteria-Female Unaffected Relatives: 1) Aged at 18 yrs or above; 2) Regular menstrual cycles 21-35 days or history of regular menstrual cycles in the past if menopausal; 3) no clinical or biochemical evidence of hyperandrogenism; 4) normal TSH and prolactin <25 ng/mL; 5) on no hormonal or insulin sensitizing medication for at least 3 months.
- Inclusion Criteria-Male Relatives: 1) Aged 18 yrs or above; 2) normal TSH and prolactin <25 ng/mL; 5) on no hormonal or insulin sensitizing medication for at least 3 months.
- Inclusion Criteria-Control Subjects: All control subjects will meet the criteria outlined for the female unaffected relatives. In Iceland, control subjects will be recruited from the Icelandic national registry, matched by age and sex to PCOS subjects and their family members. They will otherwise be recruited at random. In Boston, control subjects will be recruited from email and newspaper listings.
Key exclusion criteria (2 listed)
- Exclusion Criteria PCOS Probands: Subjects will not have 1) late onset congenital adrenal hyperplasia as defined by a fasting 17OH progesterone level <200 ng/mL or a cortrosyn stimulated 17 OH progesterone level <500 ng/mL.
- Exclusion Criteria (Female unaffected relatives): None. Exclusion Criteria Male Relatives: None. Exclusion Criteria-Control Subjects: Control subjects chosen at random will be excluded if they are already participating in the study as a PCOS subject or are a first degree family member of the PCOS subject.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Mar 2020.
- Derived from this study: PubMed 25994816Saxena R, Bjonnes AC, Georgopoulos NA, Koika V, Panidis D, Welt CK. Gene variants associated with age at menopause are also associated with polycystic ovary syndrome, gonadotrophins and ovarian volume. Hum Reprod. 2015 Jul;30(7):1697-703. doi: 10.1093/humrep/dev110. Epub 2015 May 20.
- Derived from this study: PubMed 25904635Saxena R, Georgopoulos NA, Braaten TJ, Bjonnes AC, Koika V, Panidis D, Welt CK. Han Chinese polycystic ovary syndrome risk variants in women of European ancestry: relationship to FSH levels and glucose tolerance. Hum Reprod. 2015 Jun;30(6):1454-9. doi: 10.1093/humrep/dev085. Epub 2015 Apr 22.
- Derived from this study: PubMed 23375202Pau C, Saxena R, Welt CK. Evaluating reported candidate gene associations with polycystic ovary syndrome. Fertil Steril. 2013 May;99(6):1774-8. doi: 10.1016/j.fertnstert.2012.12.033. Epub 2013 Jan 30.
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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.
Corrine Welt, MD principal investigator
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Polycystic Ovary Syndrome, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 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 2 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 3 Mar 2020. 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.
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 Corrine Welt, MD (801-585-1875, cwelt@genetics.utah.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00166569, which also lists 3 linked publications.
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 3 Mar 2020; AzTrials last refreshed its copy on 8 Nov 2026.