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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
30
6.25 mg/kg BD for 6 weeks increasing to 12.5 mg/kg BD for 6 weeks
Royal Infirmary of Edinburgh
Edinburgh, United Kingdom
To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria.
The proportion of screened women who are eligible for the trial determined from the screening logs
Time frame: Screening
To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria.
The proportion of eligible patients recruited to the study recorded on the screening logs
Time frame: Screening
To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria.
The proportion of recruited patients who answer their average NRS scores at visits 3 and 5, complete the assessment tools (questionnaires) and attend for designated blood testing
Time frame: Throughout the treatment (Week 1- 12)
To determine the acceptability to patients of the proposed methods of recruitment, treatment, questionnaires and follow up.
Assessed by acceptability questionnaire at the end of study asking questions about participants' satisfaction with the methods of recruitment, treatment, questionnaires and follow up
Time frame: Week 16
To assess whether dichloroacetate is well-tolerated in women with endometriosis.
Self-reported side effects during and after the treatment
Time frame: Throughout the treatment up to week 16
To determine participants' compliance with treatment and to assess the tools used to measure it.
Assessed by self-report using treatment diaries to measure number of doses taken
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Time frame: Throughout the treatment (Week 1- 12)
To determine participants' compliance with treatment and to assess the tools used to measure it.
Assessed by measuring levels of systemic dichloroacetate from blood samples using mass spectrometry
Time frame: Throughout the treatment (Week 1- 12)