A Clinical Trial to Evaluate Dichloroacetate (DCA) as a Treatment for Endometriosis-associated Pain (EPiC)

A Single-arm Open Label Exploratory Clinical Trial to Evaluate Dichloroacetate (DCA) as a Possible Treatment for Endometriosis-associated Pain

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

Study Overview

• Status: Unknown
• Conditions: Endometriosis
• Intervention / Treatment: Drug: Dichloroacetate

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.

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United Kingdom
  • Edinburgh, United Kingdom
    • Recruiting
    • Royal Infirmary of Edinburgh
    • Contact: Priscilla Fernandez

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Female

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Dichloroacetate; Open label study	Drug: Dichloroacetate; 6.25 mg/kg BD for 6 weeks increasing to 12.5 mg/kg BD for 6 weeks; Other Names: DCA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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	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	Throughout the treatment (Week 1- 12)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Week 16
To assess whether dichloroacetate is well-tolerated in women with endometriosis.	Self-reported side effects during and after the treatment	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	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	Throughout the treatment (Week 1- 12)

Collaborators and Investigators

• Sponsor: University of Edinburgh
• Collaborators: Ferring Pharmaceuticals; University of Nottingham; University of Birmingham

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): November 19, 2019
• Primary Completion (Anticipated): May 19, 2020
• Study Completion (Anticipated): August 31, 2020

Study Registration Dates

• First Submitted: July 31, 2019
• First Submitted That Met QC Criteria: August 2, 2019
• First Posted (Actual): August 6, 2019

Study Record Updates

• Last Update Posted (Actual): December 4, 2019
• Last Update Submitted That Met QC Criteria: December 2, 2019
• Last Verified: December 1, 2019

More Information

Terms related to this study

• Keywords: Endometriosis; Dichloroacetate
• Additional Relevant MeSH Terms: Endometriosis
• Other Study ID Numbers: AC18127

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: Selected anonymised data collected or generated by the study will be shared with commercial collaborators once the study is finished.
• IPD Sharing Time Frame: Completion of the trial, published online, available indefinitely.
• IPD Sharing Access Criteria: Anonymized results will be shared with other researchers.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No