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LeiomyomaPhase 2NCT00290251

Treatment of Uterine Fibroids With the Selective Progesterone Receptor Modulator CDB-2914

A Phase 2 study testing ulipristal acetate 20 mg and ulipristal acetate 10 mg in people with Leiomyoma, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This study will evaluate whether the experimental drug ulipristal acetate can shrink uterine fibroids in pre-menopausal women. Read more about this study

Who is it for?
Ages 25 to 50 years, women only
Conditions the study is for
Leiomyoma
What is being tested?
ulipristal acetate 20 mg and ulipristal acetate 10 mg
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
72 participants
Where
2 sitesUnited States
Study dates
Feb 2006to Aug 2010
Phase
Phase 2
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 25 to 50 years
  • Women only
  • No healthy volunteers
  • Adult

Who may be able to join

  • Female gender-to evaluate effects in the target population for clinical trials.
  • History of uterine leiomyoma causing symptoms of bleeding, pressure, or pain, as defined by the American College of Obstetrics and Gynecology (ACOG) practice bulletin:
  • Excessive uterine bleeding will be evidenced by either of the following-profuse bleeding with flooding or clots or repetitive periods lasting for more than 8 days; or anemia due to acute or chronic blood loss;
  • OR
  • Pelvic discomfort caused by leiomyomata, either acute and severe or chronic lower abdominal or low back pressure or bladder pressure with urinary frequency not due to urinary tract infection.
  • Uterine leiomyoma(ta) of at least 2 cm size.
Show all 17Show fewer
  • In good health. Chronic medication use is acceptable except for glucocorticoid use. Other chronic medication use may be acceptable at the discretion of the research team. Interval use of over-the-counter drugs is acceptable but must be recorded.
  • Menstrual cycles of 24 - 35 days.
  • Hemoglobin greater than 10 g/dL (for those wishing surgery); iron may be administered to improve red blood cell counts.
  • Willing and able to comply with study requirements.
  • Age 25 to 50.
  • Using mechanical (condoms, diaphragms) sterilization or abstinence methods of contraception for the duration of the study.
  • Negative urine pregnancy test.
  • Body mass index (BMI) less than or equal to 33, if a surgical candidate or less than or equal to 35, if not a surgical candidate.
  • Creatinine less than 1.3 mg/dL.
  • Liver function tests within 130% of upper limit.
  • If interested in hysterectomy, no desire for fertility.

Who cannot join

  • Significant abnormalities in the history, physical or laboratory examination.
  • Pregnancy.
  • Lactation.
  • Use of oral, injectable or inhaled glucocorticoids or megestrol within the last year.
  • Unexplained vaginal bleeding.
  • History of malignancy within the past 5 years.
Show all 18Show fewer
  • Use of estrogen or progesterone-containing compounds, such as oral contraceptives and hormone replacement therapy, within 8 weeks of study entry, including transdermal, injectable, vaginal and oral preparations.
  • Use of agents known to induce hepatic P450 enzymes; use of imidazoles.
  • Current use of Gonadotropin-releasing hormone (GnRH) analogs or other compounds that affect menstrual cyclicity.
  • Follicle stimulating hormone (FSH) greater than 20 IU/mL.
  • Untreated cervical dysplasia.
  • Need for interval use of narcotics.
  • Abnormal adnexal/ovarian mass.
  • Use of herbal medication having estrogenic or antiestrogenic effects within the past 3 months.
  • Contradiction to anesthesia, for women planning surgery.
  • Genetic causes of leiomyomata.
  • Previous participation in the study.
  • Known recent rapid growth of fibroids, defined as a doubling in size in six months.

Trial locations and contacts

2 sites in 1 country.

United States2 sites
  • NIH Clinical Center

    Bethesda, Maryland, 20891

  • National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, 20892

Other trials in United States

About this study

Official title: Evaluation of Whether the Selective Progesterone Receptor Modulator CDB-2914 Can Shrink Leiomyomata

This study will evaluate whether the experimental drug ulipristal acetate can shrink uterine fibroids in pre-menopausal women.

Read the detailed description

Uterine leiomyomata (fibroids) are a common benign tumor of the uterine muscle in pre-menopausal women that may cause bleeding, pelvic pain and pressure. Because fibroids grow in the presence of estrogen, medical therapies that decrease estrogen levels (like Gonadotropin releasing hormone,GnRH analog) cause fibroids to shrink and so may relieve symptoms. However, such medication can only be given short-term and has inconvenient side effects such as hot-flashes. Thus, many women with symptomatic fibroids choose to have them removed surgically, either individually or by removing the uterus via hysterectomy.

Women between 25 and 50 years of age who have regular menstrual cycles and a history of uterine fibroids that cause heavy bleeding, pressure, or pain may be eligible for this study. Candidates are screened with a medical history and physical examination, including breast and pelvic examination, blood and urine tests, a quality-of-life questionnaire, and a home urine test for leuteinizing hormone (LH) surge. They are given a diary to record the LH surge, days of vaginal spotting or bleeding, and symptoms. Participation includes the following:

Baseline Studies (First Menstrual Cycle)

  • Magnetic resonance imaging (MRI): The subject lies in the MRI scanner, a narrow cylinder with a strong magnetic field, for imaging the uterus.
  • Saline hysterosonogram: This is an ultrasound examination of the uterus. A speculum is placed in the vagina and a small amount of liquid is inserted into the uterus. A probe is then inserted into the vagina. The probe emits and receives sound waves that are used to visualize the fibroids and surrounding structures.

Study Drug Phase (Second through Fourth Menstrual Cycles)

  • Subjects are randomly assigned to take ulipristal acetate or placebo (inactive compound) once a day by mouth on an empty stomach for three menstrual cycles or up to 102 days if menstrual cycles are irregular or stop.
  • Pregnancy test on first or second day of every menstrual cycle.
  • Blood tests every 2 weeks to measure effects of study medication on hormones, blood count, blood chemistries and liver function.
  • 24-hour urine collections three times during the study, about once a month, to measure cortisol and check adrenal gland function.
  • Repeat transvaginal ultrasound after 4-6 weeks of study drug to check fibroid growth.
  • Repeat hysterosonogram and MRI within 2 weeks of surgery to count and measure the fibroids. or placebo (inactive compound) once a day by mouth on an empty stomach for three menstrual cycles or up to 102 days if menstrual cycles are irregular or stop.
  • Pregnancy test on first or second day of every menstrual cycle.
  • Blood tests every 2 weeks to measure effects of study medication on hormones, blood count, blood chemistries and liver function.
  • 24-hour urine collections three times during the study, about once a month, to measure cortisol and check adrenal gland function.
  • Repeat transvaginal ultrasound after 4-6 weeks of study drug to check fibroid growth.
  • Repeat hysterosonogram and MRI within 2 weeks of surgery to count and measure the fibroids. or placebo (inactive compound) once a day by mouth on an empty stomach for three menstrual cycles or up to 102 days if menstrual cycles are irregular or stop.
  • Pregnancy test on first or second day of every menstrual cycle.
  • Blood tests every 2 weeks to measure effects of study medication on hormones, blood count, blood chemistries and liver function.
  • 24-hour urine collections three times during the study, about once a month, to measure cortisol and check adrenal gland function.
  • Repeat transvaginal ultrasound after 4-6 weeks of study drug to check fibroid growth.
  • Repeat hysterosonogram and MRI within 2 weeks of surgery to count and measure the fibroids.
Conditions
Leiomyoma
Being tested
ulipristal acetate 20 mg, ulipristal acetate 10 mg, placebo
Keywords
Endometrium, Progesterone, Estrogen, Fibroid, Hysterectomy, Leiomyoma, Uterine Fibroids, Fibroids
Medical subject headings
Leiomyoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ulipristal acetate 20 mgDrug

    Also called VA2914; CDB-2914

    ulipristal acetate at a daily dose of 20 mg, given once daily for three menstrual cycles or 90 - 102 days if amenorrheic

  • ulipristal acetate 10 mgDrug

    Also called VA2914; CDB2914

    10 mg given daily for three menstrual cycles or 90 - 102 days

  • placeboDrug

    placebo given once daily for 3 menstrual cycles or 90 - 102 days

Study groups in NCT00290251
Study groupRoleWhat they receive
ulipristal acetate -20 mgActive comparator20 mg daily dose ulipristal acetate for three menstrual cycles or up to 102 days
Receives: Drug: ulipristal acetate 20 mg
ulipristal acetate - 10 mgActive comparator10 mg daily dose ulipristal acetate for three menstrual cycles or up to 102 days
Receives: Drug: ulipristal acetate 10 mg
PlaceboPlacebo comparatorPlacebo taken daily for three menstrual cycles or up to 102 days
Receives: Drug: placebo
Pre-ulipristal acetate 10 mgNo interventionSubjects were studied during one baseline cycle without any intervention before entering ulipristal acetate 10 mg arm
Pre-ulipristal acetate 20 mgNo interventionSubjects were studied during one baseline cycle without any intervention before entering ulipristal acetate 20 mg arm
Pre-placeboNo interventionSubjects were studied during one baseline cycle without any intervention before entering placebo arm

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Shrinkage of Fibroids - Size of Fibroids

    The primary outcome, fibroid volume, was calculated by an ellipsoid formula (π/6xd1xd2xd3) using orthogonal three-dimensional measurements taken from pelvic MRI scan. Individual volumes were summed to assess total fibroid volume for each woman, which were log-transformed before analysis. Women with paired MRI results were included in this intent to treat analysis, even if they did not take all study medication. Fibroids were included if they were seen on both studies.The absolute change in cm3 between baseline and end of treatment was calculated and its log was used for statistics and reporting the results in the data table below.

    Measured: 3 months (baseline to end of treatment)

Secondary outcomes (1)
  1. Short Form-36 and Uterine Fibroid Symptom Quality of Life

    The short form-36 (SF-36)and Uterine Fibroid Symptom Quality of Life (UFS-QOL) questionnaires were given before and at treatment end with scales of 0 - 100. SF-36 scales = mental and physical well-being. The UFS subscales are symptom severity, concern, activities, energy and mood, control, self-consciousness, sexual functioning compiled into an overall QOL score. Higher results indicate better QOL on all but symptom severity (higher = worse). The change in scores from baseline to end of treatment was calculated.

    Measured: 3 months (Baseline to end of treatment 1)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Feb 2006 (estimated)
  2. Study startFeb 2006
  3. Primary completionJul 2009
  4. Study completionAug 2010

Resources

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
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Model description
Subjects were studied during a single baseline menstrual cycle followed by a cross-over into three arms (active treatment at one of two doses, or placebo) given for three menstrual cycles
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
72 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00290251
Sponsor's protocol ID
060090
Other IDs
1ZIAHD000637-17, 06-CH-0090
Status last verified
Jul 2024
First submitted
10 Feb 2006
Last updated
15 Jul 2024
Data monitoring committee
Yes

Publications

  • Batista MC, Cartledge TP, Zellmer AW, Merino MJ, Axiotis C, Loriaux DL, Nieman LK. Delayed endometrial maturation induced by daily administration of the antiprogestin RU 486: a potential new contraceptive strategy. Am J Obstet Gynecol. 1992 Jul;167(1):60-5. doi: 10.1016/s0002-9378(11)91627-5.

    PubMed 1442957
  • Burroughs KD, Howe SR, Okubo Y, Fuchs-Young R, LeRoith D, Walker CL. Dysregulation of IGF-I signaling in uterine leiomyoma. J Endocrinol. 2002 Jan;172(1):83-93. doi: 10.1677/joe.0.1720083.

    PubMed 11786376
  • Cadepond F, Ulmann A, Baulieu EE. RU486 (mifepristone): mechanisms of action and clinical uses. Annu Rev Med. 1997;48:129-56. doi: 10.1146/annurev.med.48.1.129.

    PubMed 9046951
  • Levens ED, Wesley R, Premkumar A, Blocker W, Nieman LK. Magnetic resonance imaging and transvaginal ultrasound for determining fibroid burden: implications for research and clinical care. Am J Obstet Gynecol. 2009 May;200(5):537.e1-7. doi: 10.1016/j.ajog.2008.12.037. Epub 2009 Mar 9.

    PubMed 19268886
  • Nieman LK, Blocker W, Nansel T, Mahoney S, Reynolds J, Blithe D, Wesley R, Armstrong A. Efficacy and tolerability of CDB-2914 treatment for symptomatic uterine fibroids: a randomized, double-blind, placebo-controlled, phase IIb study. Fertil Steril. 2011 Feb;95(2):767-72.e1-2. doi: 10.1016/j.fertnstert.2010.09.059. Epub 2010 Nov 5.

    PubMed 21055739
  • Parikh TP, Malik M, Britten J, Aly JM, Pilgrim J, Catherino WH. Steroid hormones and hormone antagonists regulate the neural marker neurotrimin in uterine leiomyoma. Fertil Steril. 2020 Jan;113(1):176-186. doi: 10.1016/j.fertnstert.2019.08.090.

    PubMed 32033718
  • Lewis TD, Malik M, Britten J, Parikh T, Cox J, Catherino WH. Ulipristal acetate decreases active TGF-beta3 and its canonical signaling in uterine leiomyoma via two novel mechanisms. Fertil Steril. 2019 Apr;111(4):806-815.e1. doi: 10.1016/j.fertnstert.2018.12.026. Epub 2019 Mar 11.

    PubMed 30871768
  • Ng SSM, Jorge S, Malik M, Britten J, Su SC, Armstrong CR, Brennan JT, Chang S, Baig KM, Driggers PH, Segars JH. A-Kinase Anchoring Protein 13 (AKAP13) Augments Progesterone Signaling in Uterine Fibroid Cells. J Clin Endocrinol Metab. 2019 Mar 1;104(3):970-980. doi: 10.1210/jc.2018-01216.

    PubMed 30239831
  • Levens ED, Potlog-Nahari C, Armstrong AY, Wesley R, Premkumar A, Blithe DL, Blocker W, Nieman LK. CDB-2914 for uterine leiomyomata treatment: a randomized controlled trial. Obstet Gynecol. 2008 May;111(5):1129-36. doi: 10.1097/AOG.0b013e3181705d0e.

    PubMed 18448745

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ulipristal acetate 20 mg and ulipristal acetate 10 mg in people with Leiomyoma, run by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) at 2 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 17 inclusion and 18 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 15 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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.

What does this 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.

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 18 Dec 2012). 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?

Shrinkage of Fibroids - Size of Fibroids (time frame: 3 months (baseline to end of treatment)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00290251, which also lists 9 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 15 Jul 2024.

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