Development of a Non-Invasive Treatment for Uterine Leiomyoma (Fibroids)
Condition: Uterine Leiomyoma; Fibroids
MeSH terms: Leiomyoma; Myofibroma
Study type: Interventional
Phase: Phase 2

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.
    

Detailed description: 
      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.
    

Intervention name(s): Pirfenidone
Intervention type(s): Drug
Intervention MeSH terms: Pirfenidone
Primary purpose: Treatment
Keywords: Fibroids; Leiomyoma; Pirfenidone; Misoprostol; Non-Surgical; Collagen; Uterus; Uterine Fibroids; Uterine Leiomyoma