Clinical trial NCT00290251
Conditions: Leiomyoma
Keywords: Endometrium Progesterone Estrogen Fibroid Hysterectomy Leiomyoma Uterine Fibroids Fibroids
Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Start date: 2006-02-01; Completion date: 2010-08-01
Enrollment: 72 participants; Allocation: RANDOMIZED; Intervention model: CROSSOVER

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

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.

Arms: arm 1: 20 mg daily dose ulipristal acetate for three menstrual cycles or up to 102 days arm 2: 10 mg daily dose ulipristal acetate for three menstrual cycles or up to 102 days arm 3: Placebo taken daily for three menstrual cycles or up to 102 days arm 4: Subjects were studied during one baseline cycle without any intervention before entering ulipristal acetate 10 mg arm arm 5: Subjects were studied during one baseline cycle without any intervention before entering ulipristal acetate 20 mg arm arm 6: Subjects were studied during one baseline cycle without any intervention before entering placebo arm
Interventions: intervention 1: ulipristal acetate 20 mg intervention 2: ulipristal acetate 10 mg intervention 3: placebo
Intervention descriptions: intervention 1: ulipristal acetate at a daily dose of 20 mg, given once daily for three menstrual cycles or 90 - 102 days if amenorrheic intervention 2: 10 mg given daily for three menstrual cycles or 90 - 102 days intervention 3: placebo given once daily for 3 menstrual cycles or 90 - 102 days

Protocol documents: