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

Uterine Leiomyoma Clinical Trial

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

Status Completed

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Fibroids
• Leiomyoma
• Uterine Leiomyoma

• NCT number: NCT00332033
• Study type: Interventional
• Source: National Institutes of Health Clinical Center (CC)
• Status: Completed
• Phase: Phase 2
• Start date: May 25, 2006
• Completion date: February 16, 2007