Examining the Effects of Mitochondrial Oxidative Stress in DCM (MitoDCM)

Double blind, randomised, placebo-controlled trial of MitoQ (mitoquinol mesylate) in 106 patients with dilated cardiomyopathy, examining the effect of reducing mitochondrial oxidative stress on myocardial energetics and myocardial function using 31-phosphorus magnetic resonance spectroscopy and cardiovascular magnetic resonance.

Study Overview

• Status: Completed
• Conditions: Dilated Cardiomyopathy
• Intervention / Treatment: Drug: Placebo; Drug: MitoQ Compound

• Study Type: Interventional
• Enrollment (Actual): 53
• Phase: Phase 2

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom
    • Royal Brompton Hospital

Participation Criteria

Eligibility Criteria

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

Description

Inclusion criteria

1. idiopathic or familial DCM
2. LVEF ≤45% on 2 imaging studies of any modality ≥3 months apart (may include CMR scan at baseline visit)
3. on guideline therapy for ≥3 months as determined by usual clinicians
4. sinus rhythm on 12-lead electrocardiogram
5. plasma NT-pro-BNP >250ng/L for those >65 years and >100ng/L for those aged ≤65 years within the last 6 months (may include sample at baseline visit)

Exclusion criteria

1. current persistent atrial fibrillation
2. contraindication to CMR
3. estimated glomerular filtration rate (eGFR) <30mls/min
4. current or planned pregnancy or current breast-feeding
5. clear environmental trigger such as excess alcohol intake, cardiotoxic chemotherapy or peripartum presentation
6. fibrosis burden >25% on CMR
7. current cancer (other than non-melanoma skin cancers)
8. current use of CoQ10
9. current participation in another randomised controlled trial

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo
Experimental: MitoQ; Mitoquinol mesylate 40mg daily	Drug: MitoQ Compound; Mitoquinol mesylate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Vanguard phase - change in myocardial PCr:ATP	The first 34 patients will enter a 3 months Vanguard phase. Change in myocardial PCr:ATP between baseline and follow-up will be measured using 31P magnetic resonance spectroscopy	3 months
Vanguard phase - change in circulating markers of oxidative stress	The first 34 patients will enter a 3 months Vanguard phase. Change in circulating oxLDL and F2isoprostanes will be measured in plasma samples	3 months
Primary outcome - change in LVESVi	Change in LVESVi (ml/m2) between baseline and follow-up amongst all 106 patients measured using cardiovascular magnetic resonance	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Vanguard phase - change in skeletal muscle PCr recovery	The first 34 patients will enter a 3 months Vanguard phase. Change in skeletal muscle PCr recovery between baseline and follow-up will be measured using 31P magnetic resonance spectroscopy	3 months
Follow-on phase - change in LVEF	Change in LVEF (%) between baseline and follow-up amongst all 106 patients measured using cardiovascular magnetic resonance	12 months
Follow-on phase - change in LVEDVi	Change in LVEDVi (ml/m2) between baseline and follow-up amongst all 106 patients measured using cardiovascular magnetic resonance	12 months
Follow-on phase - change in GLS	Change in GLS between baseline and follow-up amongst all 106 patients measured using cardiovascular magnetic resonance	12 months
Follow-on phase - change in NT-pro-BNP	Change in plasma concentrations of NT-pro-BNP (ng/L) between baseline and follow-up amongst all 106 patients	12 months
Follow-on phase - change in renal function	Change in creatinine and eGFR between baseline and follow-up amongst all 106 patients	12 months
Follow-on phase - change in symptom burden	Change in symptom score between baseline and follow-up amongst all 106 patients	12 months
Follow-on phase - change in exercise capacity	Change in 6 minute walk test distance between baseline and follow-up amongst all 106 patients	12 months

Collaborators and Investigators

• Sponsor: Imperial College London
• Collaborators: British Heart Foundation
• Investigators: Principal Investigator: Brian Halliday, MBChB PhD, Imperial College London and Royal Brompton Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 26, 2022
• Primary Completion (Actual): June 19, 2024
• Study Completion (Actual): June 19, 2024

Study Registration Dates

• First Submitted: June 6, 2022
• First Submitted That Met QC Criteria: June 6, 2022
• First Posted (Actual): June 8, 2022

Study Record Updates

• Last Update Posted (Estimated): September 18, 2025
• Last Update Submitted That Met QC Criteria: September 12, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Oxidative stress; Mitochondria; Energetics
• Additional Relevant MeSH Terms: Laminopathies; Cardiovascular Diseases; Heart Diseases; Genetic Diseases, Inborn; Cardiomyopathies; Cardiomegaly; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cardiomyopathy, Dilated; Substandard Drugs; Pharmaceutical Preparations; Counterfeit Drugs; mitoquinone
• Other Study ID Numbers: 21IC6593

Drug and device information, study documents

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