Leiomyoma
Conditions
Keywords
A benign tumor derived from smooth uterine muscle tissue.
Brief summary
The purpose of this trial is to determine if low-dose mifepristone benefits women with symptomatic fibroids.
Detailed description
This trial is designed to assess the efficacy and tolerability of mifepristone 5 mg given daily for 6 months to pre-menopausal women with symptomatic fibroids. The primary study outcome will be disease-specific quality of life. Secondary outcome measures include global quality of life, pain, bleeding, potential adverse effects, tumor size, and endometrial effects. The researchers will also examine a the effect of mifepristone on uterine blood flow. Randomization of study subjects will be stratified in order to ensure that equal numbers of women with moderate and severe symptoms will be enrolled in the placebo and control groups.
Interventions
DRUGMifepristone
Mifepristone 5mg/day by mouth for 6 months
DRUGInert Capsule
sugar pill
Sponsors
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
University of Rochester
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
FEMALE
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
* Gender: Female * Age: 18 - premenopausal * Have at least moderate symptoms of menorrhagia or pelvic pain/pressure * Have a total uterine volume greater than or equal to 160 cc by ultrasound measurement and at least one fibroid that is =\> 2.5cm in size * Have a score of equal to or greater than 39 on the Uterine Fibroid Symptom and Quality of Life scale * Declined standard treatment options for symptomatic fibroids * Agree to use a double-barrier method (condoms, diaphragms) or other effective non-hormonal methods of contraception (abstinence, sterilization) throughout participation in the study to prevent pregnancy and to report any exposure to pregnancy to the research staff immediately * Willing and able to give informed consent * Willing and able to comply with study requirements
Exclusion criteria
* Current or planned pregnancy during the study period * Menopausal, as indicated by follicle stimulating hormone (FSH) level of the reference laboratory * Currently breast-feeding * Untreated abnormal pap smear * Presence of conditions other than fibroids contributing to pain and/or bleeding * Hemoglobin \< 9.0 mg/dl * Presence of adnexal masses or tenderness indicating further evaluation or surgery * Grade III or IV hydronephrosis by ultrasound * Severe, active mental health disorder * Active substance abuse or dependence * Presence of any contraindication to mifepristone including: * Adrenal insufficiency by history * Sickle cell disease * Active liver disease (liver function tests greater than 1.5 times upper range of normal) * Severe, respiratory disease (P02 saturation\< 92%) * Renal disease (serum creatinine \> 1.5 mg/dl) * Blood clotting defect. (abnormal PT and PTT) * Thromboembolic disease (history of deep vein thrombosis or pulmonary embolus) * Current or recent (within the past 3 months) use of the following medications: * Oral or systemic corticosteroids * Hormones: estrogens, progestins, oral contraceptives * Danazol, anticoagulants * Herbal or botanical supplements with possible hormonal effects. * Use within the past six months of the gonadotropin releasing hormones (GnRH) analogs or Depo-Provera. * Current or planned use during the study of any of the following medications/or products: * ketoconazole, * itraconazole, * erythromycin, * grapefruit juice, * rifampin, * St John's Wort, * phenytoin, * phenobarbital, or * carbamazepine
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Uterine Volume | 6 months | Uterine volume is measured in mLs |
Countries
United States
Participant flow
Recruitment details
Women were recruited between March 3, 2004 and March 30, 2005 through local media and contacts with community physicians.
Pre-assignment details
38 women were deemed ineligible. Of those exclusion criteria applied to 27 and 11 declined participation.
Participants by arm
| Arm | Count |
|---|---|
| Mifepristone 5 mg. Twenty-two women received 5 mg. mifepristone daily. | 22 |
| Placebo Twenty women received a placebo pill daily. | 20 |
| Total | 42 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 2 | 3 |
Baseline characteristics
| Characteristic | Mifepristone 5 mg. | Placebo | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 22 Participants | 20 Participants | 42 Participants |
| Age, Continuous | 42.8 years STANDARD_DEVIATION 6.2 | 43.2 years STANDARD_DEVIATION 4.7 | 43.0 years STANDARD_DEVIATION 5.5 |
| Sex: Female, Male Female | 22 Participants | 20 Participants | 42 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 22 | 0 / 20 |
| serious Total, serious adverse events | 0 / 22 | 0 / 20 |
Outcome results
Primary
Uterine Volume
Uterine volume is measured in mLs
Time frame: 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Treatment Group | Uterine Volume | 719 mL | Standard Deviation 663 |
| Placebo Group | Uterine Volume | 449 mL | Standard Deviation 236 |