Hypertrophic CardiomyopathyPhase 2NCT01696370
Trimetazidine Therapy in Hypertrophic Cardiomyopathy
A Phase 2 study testing Trimetazidine in people with Hypertrophic Cardiomyopathy, run by University College, London at 1 site in United Kingdom.
Trial summary
Hypertrophic cardiomyopathy (HCM) is a common inherited heart condition that causes breathlessness, chest pain and fatigue. There are few treatments available. The investigators have recently shown that a drug called perhexiline reduced symptoms and improved exercise capacity in patients with HCM. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hypertrophic Cardiomyopathy
- What is being tested?
- Trimetazidine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Apr 2012to Apr 2014
- Phase
- Phase 2
- 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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Non-obstructive hypertrophic cardiomyopathy (gradient <30 mmHg at rest)
- NYHA (New York Heart Association) Class ≥ 2
- Peak VO2 (maximal oxygen consumption) ≤80% predicted for age and gender
- Heart rate < 90/minute at rest
Who cannot join
- Diabetes Mellitus
- Abnormal renal function (GFR<60ml/min) or hepatic impairment
- Female who is pregnant, lactating or planning pregnancy during the course of the study
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site, 1 recruiting
- Recruiting
The Heart Hospital, UCLH
London, W1G 8PH
About this study
Official title: A Phase 2b Randomised, Double Blind, Placebo-controlled Trial of Trimetazidine Therapy in Patients With Non-obstructive Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy (HCM) is a common inherited heart condition that causes breathlessness, chest pain and fatigue. There are few treatments available. The investigators have recently shown that a drug called perhexiline reduced symptoms and improved exercise capacity in patients with HCM. This change appears to be driven by alterations in myocardial energy metabolism. The aim of this trial is to test a similar drug, trimetazidine, in a group of symptomatic patients with non-obstructive HCM.
HYPOTHESIS: trimetazidine will improve symptoms, peak oxygen consumption, cardiac function and arrhythmia burden in medically refractory symptomatic patients with non-obstructive HCM.
Read the detailed description
BACKGROUND:
Hypertrophic cardiomyopathy (HCM) is a common inherited disorder of heart muscle affecting 1 in 500 individuals worldwide. It is associated with arrhythmias, heart failure and sudden death in young people. In the majority of patients, HCM is caused by mutations in genes encoding cardiac contractile proteins. It has been hypothesised that excessive sarcomeric energy consumption is an important and early factor in the pathophysiology of HCM. Therefore modulation of myocardial metabolism presents a novel target for improving myocardial performance and symptoms in patients with HCM. Trimetazidine is an anti-anginal agent which like perhexiline reduces fatty acid oxidation and increases glucose oxidation, thus increasing the efficiency of energy production. Trimetazidine has been shown to significantly improve exercise performance in patients with stable angina, ischaemic and non ischaemic cardiomyopathy, either as monotherapy or in combination with beta-blockers or calcium channel blockers,
DESIGN: A single centre prospective randomised, double blind, placebo-controlled, trial of trimetazidine therapy.
DOSING: 20 mg Trimetazidine or Placebo three times daily for three months
METHODS: The following assessments will be made at baseline and after 3 months treatment: history and physical examination, Minnesota heart failure questionnaire, fasting blood tests, electrocardiogram, echocardiogram, cardiopulmonary exercise test, six minute walk test, 24 hour ECG Holter monitor.
- Conditions
- Hypertrophic Cardiomyopathy
- Being tested
- Trimetazidine, Placebo capsule
- Keywords
- Trimetazidine
- Medical subject headings
- Cardiomyopathy, Hypertrophic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrimetazidineDrug
Trimetazidine 20mg three times per day for 3 months
Placebo capsuleOther
one capsule three times per day for 3 months
| Study group | Role | What they receive |
|---|---|---|
| Trimetazidine | Active comparator | Not described Receives: Drug: Trimetazidine |
| Placebo capsule | Placebo comparator | Not described Receives: Other: Placebo capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peak oxygen consumption
Measured: 3 months
Secondary outcomes (5)
Left ventricular function
TDI and 2D strain
Measured: 3 months
Symptom status
questionnaire
Measured: 3 months
Arrhythmia
24 Hour Holter
Measured: 3 months
Cardiac biomarkers
Measured: 3 months
Exercise capacity
6 minute walk test
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2012 (estimated)
- Study startApr 2012
- Primary completionApr 2014 (estimated)
- Study completionApr 2014 (estimated)
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 Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- More trials run by University College, LondonThe 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University College, London
- Sponsor type
- University, hospital or other
- Collaborators
- British Heart Foundation
- Responsible party
- Sponsor
- Investigators
- Perry M Elliott, MBBS MD, Principal investigator, University College, London
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01696370
- Sponsor's protocol ID
- 10/0216
- Status last verified
- Aug 2011
- First submitted
- 27 Sep 2012
- Last updated
- 28 Feb 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Coats CJ, Pavlou M, Watkinson OT, Protonotarios A, Moss L, Hyland R, Rantell K, Pantazis AA, Tome M, McKenna WJ, Frenneaux MP, Omar R, Elliott PM. Effect of Trimetazidine Dihydrochloride Therapy on Exercise Capacity in Patients With Nonobstructive Hypertrophic Cardiomyopathy: A Randomized Clinical Trial. JAMA Cardiol. 2019 Mar 1;4(3):230-235. doi: 10.1001/jamacardio.2018.4847.
PubMed 30725091
Study at a glance
From the registry record, last updated by the sponsor on 28 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b Randomised, Double Blind, Placebo-controlled Trial of Trimetazidine Therapy in Patients With Non-obstructive Hypertrophic Cardiomyopathy
- ClinicalTrials.gov ID
- NCT01696370
- Acronym / sponsor ID
- 10/0216
- Conditions
- Hypertrophic Cardiomyopathy
- Keywords
- Trimetazidine
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- University College, LondonUniversity, hospital or other
- Collaborators
- British Heart Foundation
- Study start
- Apr 2012
- Primary completion
- Apr 2014 (estimated)
- Study completion
- Apr 2014 (estimated)
- First posted
- 1 Oct 2012 (estimated)
- 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.
Hypertrophic cardiomyopathy (HCM) is a common inherited heart condition that causes breathlessness, chest pain and fatigue. There are few treatments available. The investigators have recently shown that a drug called perhexiline reduced symptoms and improved exercise capacity in patients with HCM. This change appears to be driven by alterations in myocardial energy metabolism. The aim of this trial is to test a similar drug, trimetazidine, in a group of symptomatic patients with non-obstructive HCM.
HYPOTHESIS: trimetazidine will improve symptoms, peak oxygen consumption, cardiac function and arrhythmia burden in medically refractory symptomatic patients with non-obstructive HCM.
Detailed description
BACKGROUND:
Hypertrophic cardiomyopathy (HCM) is a common inherited disorder of heart muscle affecting 1 in 500 individuals worldwide. It is associated with arrhythmias, heart failure and sudden death in young people. In the majority of patients, HCM is caused by mutations in genes encoding cardiac contractile proteins. It has been hypothesised that excessive sarcomeric energy consumption is an important and early factor in the pathophysiology of HCM. Therefore modulation of myocardial metabolism presents a novel target for improving myocardial performance and symptoms in patients with HCM. Trimetazidine is an anti-anginal agent which like perhexiline reduces fatty acid oxidation and increases glucose oxidation, thus increasing the efficiency of energy production. Trimetazidine has been shown to significantly improve exercise performance in patients with stable angina, ischaemic and non ischaemic cardiomyopathy, either as monotherapy or in combination with beta-blockers or calcium channel blockers,
DESIGN: A single centre prospective randomised, double blind, placebo-controlled, trial of trimetazidine therapy.
DOSING: 20 mg Trimetazidine or Placebo three times daily for three months
METHODS: The following assessments will be made at baseline and after 3 months treatment: history and physical examination, Minnesota heart failure questionnaire, fasting blood tests, electrocardiogram, echocardiogram, cardiopulmonary exercise test, six minute walk test, 24 hour ECG Holter monitor.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Trimetazidine | Active comparator | Drug: Trimetazidine |
| Placebo capsule | Placebo comparator | Other: Placebo capsule |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Non-obstructive hypertrophic cardiomyopathy (gradient <30 mmHg at rest)
- NYHA (New York Heart Association) Class ≥ 2
- Peak VO2 (maximal oxygen consumption) ≤80% predicted for age and gender
- Heart rate < 90/minute at rest
Key exclusion criteria (3 listed)
- Diabetes Mellitus
- Abnormal renal function (GFR<60ml/min) or hepatic impairment
- Female who is pregnant, lactating or planning pregnancy during the course of the study
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 |
|---|---|---|
| TrimetazidineTrimetazidine 20mg three times per day for 3 months | Drug | Trimetazidine |
| Placebo capsuleone capsule three times per day for 3 months | Other | Placebo capsule |
Primary outcome
Peak oxygen consumption
Time frame: 3 months
5 secondary outcomes
Left ventricular function
Time frame: 3 months
Symptom status
Time frame: 3 months
Arrhythmia
Time frame: 3 months
Cardiac biomarkers
Time frame: 3 months
Exercise capacity
Time frame: 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Feb 2013.
- Derived from this study: PubMed 30725091Coats CJ, Pavlou M, Watkinson OT, Protonotarios A, Moss L, Hyland R, Rantell K, Pantazis AA, Tome M, McKenna WJ, Frenneaux MP, Omar R, Elliott PM. Effect of Trimetazidine Dihydrochloride Therapy on Exercise Capacity in Patients With Nonobstructive Hypertrophic Cardiomyopathy: A Randomized Clinical Trial. JAMA Cardiol. 2019 Mar 1;4(3):230-235. doi: 10.1001/jamacardio.2018.4847.
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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.
Perry M Elliott, MBBS MD principal investigator
University College, London
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Trimetazidine in people with Hypertrophic Cardiomyopathy, run by University College, London 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 4 inclusion and 3 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: status not confirmed, as of the sponsor's last update on 28 Feb 2013. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Perry M Elliott, MBBS MD (020 3456 7898, p.elliott@ucl.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Peak oxygen consumption (time frame: 3 months). 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 NCT01696370, 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 28 Feb 2013.