Clinical trial NCT00133705: Trial of Mifepristone for Fibroids
Official title: Randomized Control Trial Of Mifepristone for Fibroids
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Leiomyoma
Keywords: A benign tumor derived from smooth uterine muscle tissue.
Lead sponsor: University of Rochester
Sponsor class: OTHER
Collaborators: name: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
First posted: 2005-08-23
Enrollment: 70
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Uterine Volume
timeFrame: 6 months
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: University of Rochester School of Medicine & Dentistry
state: New York
Study officials:
affiliation: University of Rochester School of Medicine & Dentistry
name: Kevin Fiscella, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References:
citation: Eisinger SH, Bonfiglio T, Fiscella K, Meldrum S, Guzick DS. Twelve-month safety and efficacy of low-dose mifepristone for uterine myomas. J Minim Invasive Gynecol. 2005 May-Jun;12(3):227-33. doi: 10.1016/j.jmig.2005.01.022.
citation: Eisinger SH, Meldrum S, Fiscella K, le Roux HD, Guzick DS. Low-dose mifepristone for uterine leiomyomata. Obstet Gynecol. 2003 Feb;101(2):243-50. doi: 10.1016/s0029-7844(02)02511-5.
citation: Fiscella K, Eisinger SH, Meldrum S, Feng C, Fisher SG, Guzick DS. Effect of mifepristone for symptomatic leiomyomata on quality of life and uterine size: a randomized controlled trial. Obstet Gynecol. 2006 Dec;108(6):1381-7. doi: 10.1097/01.AOG.0000243776.23391.7b.