Open Label Trial of Thalidomide in the Treatment of Women with Chronic Pelvic Pain associated with Endometriosis

In order to participate an individual had to be:

  1. Over the age of 18
  2. Diagnosed with endometriosis by surgery
  3. Premenopausal and NOT pregnant
  4. Willing to use birth control methods

 

Study Overview

  • The study involved the use of FDA-approved, investigational drug for the treatment of pelvic pain caused by endometriosis
  • Required up to 8 visits to UNC Hospitals over the course of 6 months

 

Purpose

The purpose of this research study is to see if treatment with thalidomide reduces chronic pelvic pain in women with endometriosis who have not experienced relief of their symptoms with standard treatment. The study will also be looking to see if thalidomide treatment lowers the levels of TNF-α, IL-6, IL-1 Beta, IL-10, and IL13 in the blood. The levels of these pro-inflammatory factors in the blood of women with endometriosis are often higher than average and may contribute to chronic pelvic pain. Previous research has suggested that Thalidomide can reduce levels of TNF-α and IL-6 in the blood. Finally, the study will see if the participants’ overall quality of life and functionality is improved while being treated with thalidomide.

In 1957, thalidomide was marketed world wide as a sedative and was widely used for pregnant women with morning sickness. In 1961 researchers discovered that thalidomide caused severe birth defects and the drug was removed for the market. In 1998 thalidomide was approved by the Food and Drug Administration (FDA) for a leprosy skin condition called erythema nodosum leprosum (ENL). Thalidomide has been used by over 200,000 patients since 1998 primarily in the treatment of cancer, dermatologic disorders, rheumatology, HIV, and Crohns disease.

Recently it was suggested that thalidomide may have activity in “Complex Regional Pain Syndrome”, a severe neuropathic pain disorder that can develop as a consequence of trauma affecting limbs without nerve damage. In this syndrome, TNF-α levels are thought to play a key role in causing pain because of TNF-α’s effect at the site of nerve damage. Today there are several active research studies assessing thalidomide’s activity in pain disorders such as fibromyalgia, chronic fatigue syndrome, interstitial cystitis, radiculopathy and Complex Regional Pain Syndrome.

Similar to these studies, Thalidomide may have the potential to provide local and systemic pain relief in women with endometriosis by decreasing TNF-α levels. Thalidomide has never been used to treat chronic pelvic pain in women with endometriosis. In this study, the use of thalidomide is considered investigational.

The primary goals of this study were:

  • To measure the reduction of chronic pelvic pain in women with endometriosis during treatment with thalidomide
  • To measure the average thalidomide dose needed to reduce chronic pelvic pain in women with endometriosis
  • To estimate the change in levels of TNF-alpha, IL-6, IL-1 Beta, IL-10, and IL13 in the blood during the course of treatment with thalidomide
  • To measure changes in depression, coping and quality of life of women with chronic pelvic pain associated with endometriosis

 

Results

We are currently conducting data analysis for the results of this study.