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.
Interventional
Primary Purpose: Treatment
United States: Federal Government
060029
NCT00332033
May 2006
February 2007
Name | Location |
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National Institutes of Health Clinical Center, 9000 Rockville Pike | Bethesda, Maryland 20892 |