Premature PubarcheNCT01316042
Metformin for the Treatment of Premature Pubarche in Girls
A study testing sugar pill and Metformin, glucophage in people with Premature Pubarche, run by Milton S. Hershey Medical Center at 3 sites in United States.
Trial summary
The primary objective of this study is to determine the safety and efficacy of metformin in lowering serum DHEAS levels in girls with premature pubarche and secondary, to observe changes in hormones associated with pubertal development including gonadotropins, sex steroids, insulin, adipocytokines, and growth factors. Read more about this study
- Who is it for?
- Ages 4 to 10 years, women only
- Conditions the study is for
- Premature Pubarche
- What is being tested?
- sugar pill and Metformin, glucophage
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 4 participants
- Where
- 3 sitesUnited States
- Study dates
- May 2011to Apr 2014
- Phase
- Interventional
- 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 4 to 10 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Girls aged 4-10 with pubic hair prior to 8 years of age
- Elevated DHEAS level above age normal levels
- Informed consent from parents and assent from the girl
Who cannot join
- Diagnosis of incomplete precocious puberty, peripheral precocious puberty, or evidence of any abnormal pituitary, hypothalamic, adrenal, thyroid, and gonadal function other than premature secretion of adrenal androgens.
- Chronic illness requiring treatment that may interfere with growth and development, i.e. chronic steroid use, renal failure, etc.
- 21-hydroxylase deficiency or other enzyme deficiency leading to the phenotype of congenital adrenal hyperplasia. 21-hydroxylase deficiency will be excluded in all patients by a fasting 17-hydroxyprogesterone (17-OHP) level < 2 ng/mL. In the case of elevated fasting 17-OHP levels, an ACTH stimulation test will be performed. A 1-hour stimulated value > 10 ng/mL will be an exclusion 82. As 21 hydroxylase deficiency is a congenital condition, any normal level in the past of 17-hydroxyprogesterone allows entry into this study.
- Uncorrected thyroid disease (defined as TSH < 0.2 mIU/ML or > 5.5 mIU/mL). A normal level within the last year is adequate for entry.
- Type I or Type II diabetes (defined as a fasting serum glucose > 125mg/dL on two occasions 83), or patients receiving anti-diabetic medications such as insulin, thiazolidinediones, acarbose, or sulfonylureas; patients currently receiving metformin XR for a diagnosis of Type I or Type II diabetes or for PCOS are also specifically excluded.
- Liver disease defined as AST or ALT > 2 times normal or total bilirubin > 2.5 mg/dL.
Show all 19Show fewer
- Renal disease defined as BUN > 30 mg/dL or serum creatinine > 1.4 mg/dL.
- Significant anemia (Hemoglobin < 10 mg/dL).
- History of deep venous thrombosis, pulmonary embolus, or cerebrovascular accident.
- Known heart disease (New York Heart Association Class II or higher).
- Enrolled simultaneously into other investigative studies that require medications, proscribe the study medications, or otherwise prevent compliance with the protocol. Patients who anticipate taking longer than a one month break during the protocol should not be enrolled.
- Concomitant use other medications known to affect reproductive function or metabolism. These medications include growth hormone, IGF-1, medroxyprogesterone acetate, oral contraceptives, GnRH agonists and antagonists, anti-androgens, gonadotropins, anti-obesity drugs, somatostatin, diazoxide, ACE inhibitors, and calcium channel blockers. The washout period on all these medications will be three months.
- Suspected adrenal or ovarian tumor secreting androgens or other ectopic steroid secreting tumor.
- Suspected Cushing's syndrome.
- Lactose intolerance (the placebo filler is lactose).
- Known hypersensitivity to study medication, including ACTH and GnRH, or their excipients.
- Any concomitant medical condition that in the opinion of the investigator, may expose a subject to unacceptable level of safety risk, or that affects subject compliance.
- Subjects who anticipate having any surgery associated with restricted intake of fluids or radiological studies with contrast dye during the study period.
- Any concomitant medical condition that in the opinion of the investigator, may expose a subject to unacceptable level of safety risk, or that affects subject compliance.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
Riley Hospital for Children, Indiana University School of Medicine
Indianapolis, Indiana, 46202
Children's Hospital of Pittsburgh at the University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
About this study
Official title: A Double Blind Randomized Controlled 12 Month Trial of Metformin for the Treatment of Premature Pubarche in Girls
The primary objective of this study is to determine the safety and efficacy of metformin in lowering serum DHEAS levels in girls with premature pubarche and secondary, to observe changes in hormones associated with pubertal development including gonadotropins, sex steroids, insulin, adipocytokines, and growth factors.
- Conditions
- Premature Pubarche
- Being tested
- sugar pill, Metformin, glucophage
- Keywords
- Puberty, Hormones
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sugar pillDrug
2 pills per day for 12 months
Metformin, glucophageDrug
Also called metformin
2 212.5mg pills/day for 12 months
| Study group | Role | What they receive |
|---|---|---|
| sugar pill | Placebo comparator | 2 pills per day for 12 months Receives: Drug: sugar pill |
| Metformin | Active comparator | 2 212.5mg pill/day for 12 months Receives: Drug: Metformin, glucophage |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Serum DHEAS Levels
Change in DHEAS level was constructed per subject as the 1-year measurement minus the baseline measurement. Only descriptive statistics are provided, statistical tests were not conducted given the extremely small sample size per group.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2011 (estimated)
- Study startMay 2011
- Primary completionDec 2013
- Study completionApr 2014
Why it stopped: poor recruitment at collaborating sites
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 Premature PubarcheStudies of the same condition, open and under way.
- More trials run by Milton S. Hershey Medical CenterThe 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Milton S. Hershey Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Richard S. Legro, M.D., Milton S. Hershey Medical Center
- Investigators
- Richard S Legro, MD, Principal investigator, Milton S. Hershey Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01316042
- Sponsor's protocol ID
- 35865
- Status last verified
- Apr 2016
- First submitted
- 15 Mar 2011
- Last updated
- 12 May 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 May 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double Blind Randomized Controlled 12 Month Trial of Metformin for the Treatment of Premature Pubarche in Girls
- ClinicalTrials.gov ID
- NCT01316042
- Acronym / sponsor ID
- 35865
- Conditions
- Premature Pubarche
- Keywords
- Puberty, Hormones
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: poor recruitment at collaborating sites
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Milton S. Hershey Medical CenterUniversity, hospital or other
- Study start
- May 2011
- Primary completion
- Dec 2013 (actual)
- Study completion
- Apr 2014 (actual)
- First posted
- 16 Mar 2011 (estimated)
- Results
- Posted 12 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to determine the safety and efficacy of metformin in lowering serum DHEAS levels in girls with premature pubarche and secondary, to observe changes in hormones associated with pubertal development including gonadotropins, sex steroids, insulin, adipocytokines, and growth factors.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| sugar pill | Placebo comparator | Drug: sugar pill2 pills per day for 12 months |
| Metformin | Active comparator | Drug: Metformin, glucophage2 212.5mg pill/day for 12 months |
Who may qualify
- Ages
- 4 to 10 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Girls aged 4-10 with pubic hair prior to 8 years of age
- Elevated DHEAS level above age normal levels
- Informed consent from parents and assent from the girl
Key exclusion criteria (19 listed)
- Diagnosis of incomplete precocious puberty, peripheral precocious puberty, or evidence of any abnormal pituitary, hypothalamic, adrenal, thyroid, and gonadal function other than premature secretion of adrenal androgens.
- Chronic illness requiring treatment that may interfere with growth and development, i.e. chronic steroid use, renal failure, etc.
- 21-hydroxylase deficiency or other enzyme deficiency leading to the phenotype of congenital adrenal hyperplasia. 21-hydroxylase deficiency will be excluded in all patients by a fasting 17-hydroxyprogesterone (17-OHP) level < 2 ng/mL. In the case of elevated fasting 17-OHP levels, an ACTH stimulation test will be performed. A 1-hour stimulated value > 10 ng/mL will be an exclusion 82. As 21 hydroxylase deficiency is a congenital condition, any normal level in the past of 17-hydroxyprogesterone allows entry into this study.
- Uncorrected thyroid disease (defined as TSH < 0.2 mIU/ML or > 5.5 mIU/mL). A normal level within the last year is adequate for entry.
- Type I or Type II diabetes (defined as a fasting serum glucose > 125mg/dL on two occasions 83), or patients receiving anti-diabetic medications such as insulin, thiazolidinediones, acarbose, or sulfonylureas; patients currently receiving metformin XR for a diagnosis of Type I or Type II diabetes or for PCOS are also specifically excluded.
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 |
|---|---|---|
| sugar pill2 pills per day for 12 months | Drug | sugar pill |
| Metformin, glucophageAlso: metformin2 212.5mg pills/day for 12 months | Drug | Metformin |
Primary outcome
Change in Serum DHEAS Levels
Time frame: 1 year
Change in DHEAS level was constructed per subject as the 1-year measurement minus the baseline measurement. Only descriptive statistics are provided, statistical tests were not conducted given the extremely small sample size per group.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 May 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- departmental websiteLink provided by the sponsor.
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.
Richard S Legro, MD principal investigator
Milton S. Hershey Medical Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing sugar pill and Metformin, glucophage in people with Premature Pubarche, run by Milton S. Hershey Medical Center at 3 sites 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 3 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: stopped early, as of the sponsor's last update on 12 May 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 12 May 2016). 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?
Change in Serum DHEAS Levels (time frame: 1 year). 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 12 May 2016.