Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)
Status:
Completed
Trial end date:
2007-02-16
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 2
Details
Lead Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)