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

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ClinicalTrials.gov Identifier: NCT04046081
Recruitment Status : Recruiting
First Posted : August 6, 2019
Last Update Posted : December 4, 2019
Sponsor:
Collaborators:
University of Nottingham
Ferring Pharmaceuticals
University of Birmingham
Information provided by (Responsible Party):
University of Edinburgh

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

Condition or disease Intervention/treatment Phase
Endometriosis Drug: Dichloroacetate Not Applicable

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: A Single-arm Open Label Exploratory Clinical Trial to Evaluate Dichloroacetate (DCA) as a Possible Treatment for Endometriosis-associated Pain
Actual Study Start Date : November 19, 2019
Estimated Primary Completion Date : May 19, 2020
Estimated Study Completion Date : August 31, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Endometriosis

Arm Intervention/treatment
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 Name: DCA




Primary Outcome Measures :
  1. To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria. [ Time Frame: Screening ]
    The proportion of screened women who are eligible for the trial determined from the screening logs

  2. To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria. [ Time Frame: Screening ]
    The proportion of eligible patients recruited to the study recorded on the screening logs

  3. To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria. [ Time Frame: Throughout the treatment (Week 1- 12) ]
    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


Secondary Outcome Measures :
  1. To determine the acceptability to patients of the proposed methods of recruitment, treatment, questionnaires and follow up. [ Time Frame: Week 16 ]
    Assessed by acceptability questionnaire at the end of study asking questions about participants' satisfaction with the methods of recruitment, treatment, questionnaires and follow up

  2. To assess whether dichloroacetate is well-tolerated in women with endometriosis. [ Time Frame: Throughout the treatment up to week 16 ]
    Self-reported side effects during and after the treatment

  3. To determine participants' compliance with treatment and to assess the tools used to measure it. [ Time Frame: Throughout the treatment (Week 1- 12) ]
    Assessed by self-report using treatment diaries to measure number of doses taken

  4. To determine participants' compliance with treatment and to assess the tools used to measure it. [ Time Frame: Throughout the treatment (Week 1- 12) ]
    Assessed by measuring levels of systemic dichloroacetate from blood samples using mass spectrometry



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04046081


Contacts
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Contact: Ann M Doust 01312429492 Ann.Doust@ed.ac.uk
Contact: Magda Koscielniak, PhD 01312429492 M.Koscielniak@ed.ac.uk

Locations
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United Kingdom
Royal Infirmary of Edinburgh Recruiting
Edinburgh, United Kingdom
Contact: Priscilla Fernandez         
Sponsors and Collaborators
University of Edinburgh
University of Nottingham
Ferring Pharmaceuticals
University of Birmingham
Investigators
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Study Chair: Andrew W Horne, Prof University of Edinburgh
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Responsible Party: University of Edinburgh
ClinicalTrials.gov Identifier: NCT04046081    
Other Study ID Numbers: AC18127
First Posted: August 6, 2019    Key Record Dates
Last Update Posted: December 4, 2019
Last Verified: December 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Selected anonymised data collected or generated by the study will be shared with commercial collaborators once the study is finished.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: Completion of the trial, published online, available indefinitely.
Access Criteria: Anonymized results will be shared with other researchers.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by University of Edinburgh:
Endometriosis
Dichloroacetate
Additional relevant MeSH terms:
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Endometriosis
Genital Diseases, Female