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Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)

Phase 2
35 Years
50 Years
Not Enrolling
Uterine Leiomyoma, Fibroids

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Trial Information

Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)

Uterine leiomyomas (fibroids) represent a major public health problem with few effective
therapies. Currently, the only definitive treatment is hysterectomy and women are demanding
alternative therapies to surgery. We have developed a new approach to the treatment of
uterine fibroids based on collaborative laboratory research into the molecular,
ultra-structural, and histopathologic changes that occur with the transformation of normal
uterine myocytes into abnormal myocytes comprising uterine fibroids. We have confirmed that
excessive, dysregulated collagen production (fibrosis) and abnormal collagen deposition is
an underlying etiology in the pathogenesis of leiomyoma. We will test the hypothesis that an
anti-tumor drug (Pirfenidone) will decrease the size of clinically relevant leiomyomas by
30%. The specific aim is to compare the effects of pirfenidone with placebo on uterine
leiomyoma volume. Thirty-two (32) women will be randomized in a double-blinded treatment
design. Inclusion criteria include women that have completed child-bearing, who are
candidates for hysterectomy, are using effective contraceptive, and have at least one
uterine leiomyoma greater than 4 cm diameter confirmed by ultrasound. Women will be excluded
if they have a body mass index greater than 33 kg/m(2), other gynecological diseases, and
history of cardiovascular disease or smoking. Response in each treatment group will be
assessed by T-2 weighted magnetic resonance imaging (MRI) and 3-D ultrasound imaging studies
during the enrollment period. To our knowledge, this will be the first study to document the
response of large fibroids to a short-term trial of an anti-tumor drug. The data will be
used to further define the role of fibrosis in leiomyoma and establish other clinical trials
to thoroughly evaluate such therapeutic approaches for uterine leiomyomas.

Inclusion Criteria


- Parous and non-parous women who have completed child-bearing (age criteria of greater
than 35 years-old, and less than 50 years-old).

- Currently practicing or willing to practice contraception throughout the duration of
the study.

- Women with at least one fibroid greater than 4 cm diameter on ultrasound examination.

- Women who were planning to undergo hysterectomy for treatment of symptomatic fibroids
may also participate if they are willing to defer the operation until the study is
completed. The patients will understand that they may choose to have surgery or any
other non-study treatment at any time after enrollment and end study participation.


- Inability to comply with study visits and requirements.

- BMI greater than 33kg/m(2)

- History of a serious adverse reaction to study medication.

- Inability to tolerate MRI and 3D ultrasound procedures (i.e. presence of an
intra-uterine device (IUD), pacemakers, aneurismal clips or other metallic devices
that are not compatible with magnetic resonance imaging).

- Medical problems including: genetic diseases that cause fibroids, history of
thromboembolic (blood clot) events or need for anticoagulation (Coumadin, Heparin,

- A history of cancer within the past 5 years.

- Abnormal liver function tests (typically, will be greater than 20% elevation). Mild
elevations will be at the discretion of the investigators, but undiagnosed liver
conditions will represent an exclusion criterion.

- Pregnancy or lactation. Pregnancy will be evaluated by (Beta)hCG test every 30 days.
Patients will be asked to use non-hormonal contraception methods while on study drug.

- Severe anemia (hct. Less than 30). Mild anemia is common in women with fibroids.

- Recent rapid growth of fibroids (i.e. doubling in size within one-six months).

- Use any of the following medications:

1. Oral, injectable, or inhaled steroids or megesterol within the past year

2. Estrogen or progesterone-containing compounds (including oral contraceptives,
hormone replacement therapy, transdermal/injectable/vaginal/oral preparations,
herbal medications with estrogenic or anti-estrogenic effects) within the past
8-12 weeks; or GnRH analogs (Danazol) or other compounds that affect menstrual

3. Agents like Imidazoles, due to possible interference with metabolism.

- Unevaluated gynecologic abnormalities (unexplained vaginal bleeding, cervical
dysplasia, or abnormal adnexal/ovarian mass).

Type of Study:


Study Design:

Primary Purpose: Treatment


United States: Federal Government

Study ID:




Start Date:

May 2006

Completion Date:

February 2007

Related Keywords:

  • Uterine Leiomyoma
  • Fibroids
  • Fibroids
  • Leiomyoma
  • Pirfenidone
  • Misoprostol
  • Non-Surgical
  • Collagen
  • Uterus
  • Uterine Fibroids
  • Uterine Leiomyoma
  • Leiomyoma
  • Myofibroma



National Institutes of Health Clinical Center, 9000 Rockville PikeBethesda, Maryland  20892