Trial of Mifepristone for Fibroids

• Sponsor: University of Rochester (Other)
• Overall Status: Completed
• CT.gov ID: NCT00133705
• Collaborator: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (NIH)
• Enrollment: 70
• Location: 1
• Arms: 2
• Duration (Months): 83
• Patients Per Site Per Month: 0.8

Study Details

Study Description

Brief Summary

The purpose of this trial is to determine if low-dose mifepristone benefits women with symptomatic fibroids.

Condition or Disease	Intervention/Treatment	Phase
Leiomyoma	Drug: Mifepristone; Drug: Inert Capsule	Phase 3

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 70 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: Randomized Control Trial Of Mifepristone for Fibroids
• Study Start Date: Jul 1, 2003
• Actual Primary Completion Date: Jun 1, 2010
• Actual Study Completion Date: Jun 1, 2010

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Mifepristone Mifepristone 5 MG capsule taken once daily by mouth	Drug: Mifepristone; Mifepristone 5mg/day by mouth for 6 months; Other Names: RU486
Placebo Comparator: Inert capsule Placebo (for Mifepristone) capsule of nearly identical color, size, and weight taken once daily by mouth	Drug: Inert Capsule; sugar pill

Outcome Measures

Primary Outcome Measures

1. Uterine Volume [6 months]

   Uterine volume is measured in mLs

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: Female
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Rochester School of Medicine & Dentistry	Rochester	New York	United States	14642

Sponsors and Collaborators

• University of Rochester
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Investigators

• Principal Investigator: Kevin Fiscella, MD, MPH, University of Rochester School of Medicine & Dentistry

Study Documents (Full-Text)

None provided.

More Information

Publications

• 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.
• 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.

• Responsible Party: Kevin Fiscella, Tenured Professor, University of Rochester
• ClinicalTrials.gov Identifier: NCT00133705
• Other Study ID Numbers: RO1-HD042578-2
• First Posted: Aug 23, 2005
• Last Update Posted: Mar 14, 2014
• Last Verified: Feb 1, 2014

• Keywords provided by Kevin Fiscella, Tenured Professor, University of Rochester: A benign tumor derived from smooth uterine muscle tissue.
• Additional relevant MeSH terms: Leiomyoma; Mifepristone

Study Results

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 Detail	38 women were deemed ineligible. Of those exclusion criteria applied to 27 and 11 declined participation.

Arm/Group Title	Treatment Group	Placebo Group
Arm/Group Description	This group will receive 5 mg. capsules to be taken once daily.	These women received placebo capsules identical in appearance and weight to the 5 mg. mifepristone capsule to be taken once daily.
Period Title: Overall Study
STARTED	22	20
COMPLETED	20	17
NOT COMPLETED	2	3

Baseline Characteristics

Arm/Group Title	Mifepristone 5 mg.	Placebo	Total
Arm/Group Description	Twenty-two women received 5 mg. mifepristone daily.	Twenty women received a placebo pill daily.	Total of all reporting groups
Overall Participants	22	20	42
Age (Count of Participants)
<=18 years	0 0%	0 0%	0 0%
Between 18 and 65 years	22 100%	20 100%	42 100%
>=65 years	0 0%	0 0%	0 0%
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	42.8 (6.2)	43.2 (4.7)	43.0 (5.5)
Sex: Female, Male (Count of Participants)
Female	22 100%	20 100%	42 100%
Male	0 0%	0 0%	0 0%

Outcome Measures

1. Primary Outcome

Title	Uterine Volume
Description	Uterine volume is measured in mLs
Time Frame	6 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Treatment Group	Placebo Group
Arm/Group Description	This group will receive 5 mg. capsules to be taken once daily.	These women received placebo capsules identical in appearance and weight to the 5 mg. mifepristone capsule to be taken once daily.
Measure Participants	22	20
Mean (Standard Deviation) [mL]	719 (663)	449 (236)

Adverse Events

Arm/Group Title	Treatment Group		Placebo Group
Arm/Group Description	This group will receive 5 mg. capsules to be taken once daily.		These women received placebo capsules identical in appearance and weight to the 5 mg. mifepristone capsule to be taken once daily.
All Cause Mortality
	Treatment Group		Placebo Group
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)
Serious Adverse Events
	Treatment Group		Placebo Group
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/22 (0%)		0/20 (0%)
Other (Not Including Serious) Adverse Events
	Treatment Group		Placebo Group
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/22 (0%)		0/20 (0%)

Limitations/Caveats

There were no adverse events in this clinical trial.

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.