EndometriosisEPiCNCT04046081
A Clinical Trial to Evaluate Dichloroacetate (DCA) as a Treatment for Endometriosis-associated Pain
A study testing Dichloroacetate in people with Endometriosis, run by University of Edinburgh at 1 site in United Kingdom.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Endometriosis
- What is being tested?
- Dichloroacetate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 19 Nov 2019to 30 Sep 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- Willing to comply with the treatment
- Willing to use contraception throughout the trial
- Willing and able to complete informed consent
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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
Read the 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.
- Conditions
- Endometriosis
- Being tested
- Dichloroacetate
- Keywords
- Endometriosis, Dichloroacetate
- Medical subject headings
- Endometriosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DichloroacetateDrug
Also called DCA
6.25 mg/kg BD for 6 weeks increasing to 12.5 mg/kg BD for 6 weeks
| Study group | Role | What they receive |
|---|---|---|
| Dichloroacetate | Other | Open label study Receives: Drug: Dichloroacetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: Throughout the treatment (Week 1- 12)
Secondary outcomes (4)
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
Measured: Week 16
To assess whether dichloroacetate is well-tolerated in women with endometriosis.
Self-reported side effects during and after the treatment
Measured: 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
Measured: 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
Measured: Throughout the treatment (Week 1- 12)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2019
- Study start19 Nov 2019
- Primary completion30 Sep 2021
- Study completion30 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for EndometriosisStudies of the same condition, open and under way.
- More trials run by University of EdinburghThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Edinburgh
- Sponsor type
- University, hospital or other
- Collaborators
- University of Nottingham, Ferring Pharmaceuticals, University of Birmingham
- Responsible party
- Sponsor
- Investigators
- Andrew W Horne, Prof, Study chair, University of Edinburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04046081
- Sponsor's protocol ID
- AC18127
- Acronym
- EPiC
- Status last verified
- Jun 2024
- First submitted
- 31 Jul 2019
- Last updated
- 13 Jun 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Selected anonymised data collected or generated by the study will be shared with commercial collaborators once the study is finished.
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.
PubMed 33712086
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm Open Label Exploratory Clinical Trial to Evaluate Dichloroacetate (DCA) as a Possible Treatment for Endometriosis-associated Pain
- ClinicalTrials.gov ID
- NCT04046081
- Acronym / sponsor ID
- EPiC / AC18127
- Conditions
- Endometriosis
- Keywords
- Endometriosis, Dichloroacetate
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- University of EdinburghUniversity, hospital or other
- Collaborators
- University of Nottingham, Ferring Pharmaceuticals, University of Birmingham
- Study start
- 19 Nov 2019 (actual)
- Primary completion
- 30 Sep 2021 (actual)
- Study completion
- 30 Sep 2021 (actual)
- First posted
- 6 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dichloroacetate | Other | Drug: DichloroacetateOpen label study |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DichloroacetateAlso: DCA6.25 mg/kg BD for 6 weeks increasing to 12.5 mg/kg BD for 6 weeks | Drug | Dichloroacetate |
Primary outcome
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
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
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
4 secondary outcomes
To determine the acceptability to patients of the proposed methods of recruitment, treatment, questionnaires and follow up.
Time frame: Week 16
To assess whether dichloroacetate is well-tolerated in women with endometriosis.
Time frame: Throughout the treatment up to week 16
To determine participants' compliance with treatment and to assess the tools used to measure it.
Time frame: Throughout the treatment (Week 1- 12)
To determine participants' compliance with treatment and to assess the tools used to measure it.
Time frame: Throughout the treatment (Week 1- 12)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2024.
- Derived from this study: PubMed 33712086Leow 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.
- Individual participant dataSharing planned: Yes. Selected anonymised data collected or generated by the study will be shared with commercial collaborators once the study is finished.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andrew W Horne, Prof study chair
University of Edinburgh
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Dichloroacetate in people with Endometriosis, run by University of Edinburgh at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine whether it is possible to achieve acceptable recruitment and retention rates within defined inclusion/exclusion criteria. (time frame: Screening). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04046081, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2024.