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

NCT04046081 | Completed

• 1 other identifier: AC18127
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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

Conditions

Endometriosis

Keywords

Endometriosis; Dichloroacetate

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (4)

• To determine the acceptability to patients of the proposed methods of recruitment, treatment, questionnaires and follow up.

  Week 16

• To assess whether dichloroacetate is well-tolerated in women with endometriosis.

  Throughout the treatment up to week 16

• To determine participants' compliance with treatment and to assess the tools used to measure it.

  Throughout the treatment (Week 1- 12)

• To determine participants' compliance with treatment and to assess the tools used to measure it.

  Throughout the treatment (Week 1- 12)

Study Arms (1)

Dichloroacetate

OTHER

Open label study

Drug: Dichloroacetate

Interventions

Dichloroacetate DRUG

6.25 mg/kg BD for 6 weeks increasing to 12.5 mg/kg BD for 6 weeks

Also known as: DCA

Dichloroacetate

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Edinburgh: lead
• University of Nottingham: collaborator
• Ferring Pharmaceuticals: collaborator
• University of Birmingham: collaborator

Study Sites (1)

Royal Infirmary of Edinburgh

Edinburgh, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 33712086 DERIVED

MeSH Terms

Conditions

Endometriosis

Interventions

Dichloroacetic Acid

Condition Hierarchy (Ancestors)

Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases

Intervention Hierarchy (Ancestors)

Chloroacetates; Acetates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Hydrocarbons, Chlorinated; Hydrocarbons, Halogenated; Hydrocarbons

Study Officials

• Andrew W Horne, Prof

  University of Edinburgh

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 31, 2019
• First Posted: August 6, 2019
• Study Start: November 19, 2019
• Primary Completion: September 30, 2021
• Study Completion: September 30, 2021
• Last Updated: June 13, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Completion of the trial, published online, available indefinitely.
• Access Criteria: Anonymized results will be shared with other researchers.

Locations

GB (1)