This is a single-arm open label exploratory clinical trial to evaluate dichloroacetate (DCA) as a possible treatment for treatment of endometriosis-associated pain

Endometriosis is a chronic condition usually affecting women throughout their reproductive lives. It is defined as a growth of endometrial-like tissue (womb lining) outside the uterus (womb) and is associated with chronic pelvic pain that can be frequent and severe, resulting in tiredness, lower quality of life and difficulties in getting pregnant. Current treatments are unsatisfactory and there is an unmet need for new medical treatment for endometriosis. Research findings from our laboratory have shown that women with endometriosis have more lactate in their pelvis. In laboratory models of endometriosis, we have tested dichloroacetate (DCA), a compound used to treat metabolic disorders in children. Our results showed that DCA could stop the growth and survival of endometriosis cells and reduce lactate production. In our study we plan to investigate if we can we can recruit and retain women into a trial using this treatment. We will recruit 30 women aged 18 or over, with pelvic pain and a diagnosis of endometriosis within the last three years. Participants will complete informed consent, be willing to comply with the treatment and use contraception throughout the trial. We will recruit patients over six months at Royal Infirmary of Edinburgh. Women who consent will take a daily dose of DCA capsules for 12 weeks.

Inclusion Criteria:

• Women aged 18 or over
• Weight between 50 and 100kg
• Pre-menopausal
• Superficial peritoneal endometriosis (ASRM Stage I or II) at laparoscopy, performed within the last three years (and >2 weeks from surgery)
• Pelvic pain for longer than six months
• Average pain score of ≥ 4 over the four weeks prior to treatment
• Willing to comply with the treatment
• Willing to use contraception throughout the trial
• Willing and able to complete informed consent

Exclusion Criteria:

• Evidence of ovarian endometrioma or deep endometriosis (based upon current surgical staging or most recent imaging)
• Women who are pregnant or actively trying to get pregnant
• Known allergy or hypersensitivity to any excipient of DCA
• Breastfeeding
• Clinical evidence of pre-existing neuropathy
• Diabetes
• History of liver disease
• History of kidney disease
• Taking part in a CTIMP or other interventional non-CTIMP studies
• Patient on combination antiretroviral therapy
• History of malabsorption syndrome or substantial amount of small bowels or stomach removed

Selected anonymised data collected or generated by the study will be shared with commercial collaborators once the study is finished.

Completion of the trial, published online, available indefinitely.

Anonymized results will be shared with other researchers.