Clinical trial NCT04046081: A Clinical Trial to Evaluate Dichloroacetate (DCA) as a Treatment for Endometriosis-associated Pain
Official title: A Single-arm Open Label Exploratory Clinical Trial to Evaluate Dichloroacetate (DCA) as a Possible Treatment for Endometriosis-associated Pain
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Endometriosis
Keywords: Endometriosis
Dichloroacetate
Lead sponsor: University of Edinburgh
Sponsor class: OTHER
Collaborators: name: University of Nottingham
name: Ferring Pharmaceuticals
name: University of Birmingham
First posted: 2019-08-06
Enrollment: 30
Sex: FEMALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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

Detailed description: 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.

Eligibility criteria: 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

Primary outcomes:
measure: To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria.
timeFrame: Screening
measure: To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria.
timeFrame: Screening
measure: To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria.
timeFrame: Throughout the treatment (Week 1- 12)
Secondary outcomes:
measure: To determine the acceptability to patients of the proposed methods of recruitment, treatment, questionnaires and follow up.
timeFrame: Week 16
measure: To assess whether dichloroacetate is well-tolerated in women with endometriosis.
timeFrame: Throughout the treatment up to week 16
measure: To determine participants' compliance with treatment and to assess the tools used to measure it.
timeFrame: Throughout the treatment (Week 1- 12)
measure: To determine participants' compliance with treatment and to assess the tools used to measure it.
timeFrame: Throughout the treatment (Week 1- 12)
Other outcomes:
Locations:
city: Edinburgh
country: United Kingdom
facility: Royal Infirmary of Edinburgh
Study officials:
affiliation: University of Edinburgh
name: Andrew W Horne, Prof
role: STUDY_CHAIR
References:
citation: Leow HW, Koscielniak M, Williams L, Saunders PTK, Daniels J, Doust AM, Jones MC, Ferguson GD, Bagger Y, Horne AW, Whitaker LHR. Dichloroacetate as a possible treatment for endometriosis-associated pain: a single-arm open-label exploratory clinical trial (EPiC). Pilot Feasibility Stud. 2021 Mar 12;7(1):67. doi: 10.1186/s40814-021-00797-0.