Diastolic Heart FailurePhase 2NCT00839228
Perhexiline Therapy in Heart Failure With Preserved Ejection Fraction Syndrome
A Phase 2 study testing Perhexiline in people with Diastolic Heart Failure, run by University of Aberdeen at 1 site in United Kingdom.
Trial summary
Up to half of all patients with clinical features of heart failure are found to have normal heart pumping function. Recently the investigators have shown that a drug called perhexiline markedly improved exercise capacity and symptoms in patients with heart failure associated with impaired cardiac pump function. Read more about this study
- Who is it for?
- Ages 16 to 90 years
- Conditions the study is for
- Diastolic Heart Failure
- What is being tested?
- Perhexiline
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Mar 2009to Feb 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 16 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- HFpEF will be defined as:
- Clinical features consistent with heart failure
- LVEF ≥ 50%, with no evidence of significant valvular disease
- No hypertrophic cardiomyopathy, and no evidence of pericardial constriction
- Peak VO2 < 80% predicted, with RER>1 and with a pattern of gas exchange on metabolic exercise testing indicating a cardiac cause for limitation)
- Patients recruited will be in sinus rhythm
Who cannot join
- BMI >35
- Objective evidence of lung disease on formal lung function testing
- Reversible myocardial ischaemia on contrast-enhanced myocardial stress Echocardiography, and no evidence of exercise-induced mitral regurgitation (>2+)
- Impaired hepatic function; known hypersensitivity to perhexiline
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Randomised Double Blind Placebo Controlled Trial of Perhexiline in Heart Failure With Preserved Ejection Fraction Syndrome (HFpEF)
Up to half of all patients with clinical features of heart failure are found to have normal heart pumping function. Recently the investigators have shown that a drug called perhexiline markedly improved exercise capacity and symptoms in patients with heart failure associated with impaired cardiac pump function. In this proposal the investigators will assess whether perhexiline has beneficial effects in patients with heart failure and a normal heart pumping function.
Read the detailed description
Up to 50% of patients with symptoms of heart failure have a preserved left ventricular ejection fraction (HFpEF syndrome). Current therapy for systolic heart failure is targeted at inducing vasodilation and counteracting neuro-endocrine activation. There is a lack of a corresponding evidence base for the treatment of HEpEF. We have previously shown that perhexiline, an agent that increases the efficiency of energy production by shifting substrate utilization from free fatty acids towards glucose, was highly effective in improving exercise capacity, symptoms and cardiac function in patients with systolic heart failure. We have also recently shown that energy deficiency plays a major role in the pathophysiology of HFpEF. In this proposal we therefore aim to investigate the effectiveness of perhexiline in 70 HFpEF patients, in a 3 month randomised, double-blind, controlled trial. An interim analysis is planned after 20 patients.
- Conditions
- Diastolic Heart Failure
- Being tested
- Perhexiline, Placebo
- Keywords
- diastolic dysfunction, cardiac energetics, perhexiline, magnetic resonance spectroscopy
- Medical subject headings
- Heart Failure, Diastolic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PerhexilineDrug
Also called Pexsig
100mg o bd for 3 months
PlaceboDrug
Also called Starch Placebo
Placebo one tablet bd for 3 months
| Study group | Role | What they receive |
|---|---|---|
| Perhexiline | Experimental | perhexiline 100mg o bd for 3 months Receives: Drug: Perhexiline |
| Placebo | Placebo comparator | Placebo one tablet bd for 3 months Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Peak oxygen consumption (Vo2max)
Measured: 3 months
Secondary outcomes (5)
Symptomatic Status (Modified Minnesota Living with Heart Failure Questionnaire)
Measured: 3 Months
Resting myocardial energetics by cardiac MR spectroscopy (MRS)
Measured: 3 months
Resting and exercise diastolic function (nuclear studies)
Measured: 3 months
Indirect measures of resting LVEDP (tissue Doppler E/Ea)
Measured: 3 months
Global LV Ejection Fraction (MRI / nuclear studies)
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 posted9 Feb 2009 (estimated)
- Study startMar 2009
- Primary completionFeb 2014
- Study completionFeb 2014
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 Diastolic Heart FailureStudies of the same condition, open and under way.
- 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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Aberdeen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michael P Frenneaux, MBBS MD, Principal investigator, University of Aberdeen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00839228
- Sponsor's protocol ID
- RRK 3147
- Other IDs
- RRK 3147, MREC 08/H1207/84, EudraCT 2006-001109-28
- Status last verified
- Nov 2015
- First submitted
- 6 Feb 2009
- Last updated
- 5 Nov 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Singh S, Beadle R, Cameron D, Rudd A, Bruce M, Jagpal B, Schwarz K, Brindley G, Mckiddie F, Lang C, Dawson D, Frenneaux M. Randomized double-blind placebo-controlled trial of perhexiline in heart failure with preserved ejection fraction syndrome. Future Cardiol. 2014 Nov;10(6):693-8. doi: 10.2217/fca.14.62.
PubMed 25495811
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomised Double Blind Placebo Controlled Trial of Perhexiline in Heart Failure With Preserved Ejection Fraction Syndrome (HFpEF)
- ClinicalTrials.gov ID
- NCT00839228
- Acronym / sponsor ID
- RRK 3147
- Conditions
- Diastolic Heart Failure
- Keywords
- diastolic dysfunction, cardiac energetics, perhexiline, magnetic resonance spectroscopy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- University of AberdeenUniversity, hospital or other
- Study start
- Mar 2009
- Primary completion
- Feb 2014 (actual)
- Study completion
- Feb 2014 (actual)
- First posted
- 9 Feb 2009 (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.
Up to half of all patients with clinical features of heart failure are found to have normal heart pumping function. Recently the investigators have shown that a drug called perhexiline markedly improved exercise capacity and symptoms in patients with heart failure associated with impaired cardiac pump function. In this proposal the investigators will assess whether perhexiline has beneficial effects in patients with heart failure and a normal heart pumping function.
Detailed description
Up to 50% of patients with symptoms of heart failure have a preserved left ventricular ejection fraction (HFpEF syndrome). Current therapy for systolic heart failure is targeted at inducing vasodilation and counteracting neuro-endocrine activation. There is a lack of a corresponding evidence base for the treatment of HEpEF. We have previously shown that perhexiline, an agent that increases the efficiency of energy production by shifting substrate utilization from free fatty acids towards glucose, was highly effective in improving exercise capacity, symptoms and cardiac function in patients with systolic heart failure. We have also recently shown that energy deficiency plays a major role in the pathophysiology of HFpEF. In this proposal we therefore aim to investigate the effectiveness of perhexiline in 70 HFpEF patients, in a 3 month randomised, double-blind, controlled trial. An interim analysis is planned after 20 patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Perhexiline | Experimental | Drug: Perhexilineperhexiline 100mg o bd for 3 months |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo one tablet bd for 3 months |
Who may qualify
- Ages
- 16 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- HFpEF will be defined as:
- Patients recruited will be in sinus rhythm
Key exclusion criteria (4 listed)
- BMI >35
- Objective evidence of lung disease on formal lung function testing
- Reversible myocardial ischaemia on contrast-enhanced myocardial stress Echocardiography, and no evidence of exercise-induced mitral regurgitation (>2+)
- Impaired hepatic function; known hypersensitivity to perhexiline
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 |
|---|---|---|
| PerhexilineAlso: Pexsig100mg o bd for 3 months | Drug | Perhexiline |
| PlaceboAlso: Starch PlaceboPlacebo one tablet bd for 3 months | Drug | Placebo |
Primary outcome
Change in Peak oxygen consumption (Vo2max)
Time frame: 3 months
5 secondary outcomes
Symptomatic Status (Modified Minnesota Living with Heart Failure Questionnaire)
Time frame: 3 Months
Resting myocardial energetics by cardiac MR spectroscopy (MRS)
Time frame: 3 months
Resting and exercise diastolic function (nuclear studies)
Time frame: 3 months
Indirect measures of resting LVEDP (tissue Doppler E/Ea)
Time frame: 3 months
Global LV Ejection Fraction (MRI / nuclear studies)
Time frame: 3 months
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 5 Nov 2015.
- Derived from this study: PubMed 25495811Singh S, Beadle R, Cameron D, Rudd A, Bruce M, Jagpal B, Schwarz K, Brindley G, Mckiddie F, Lang C, Dawson D, Frenneaux M. Randomized double-blind placebo-controlled trial of perhexiline in heart failure with preserved ejection fraction syndrome. Future Cardiol. 2014 Nov;10(6):693-8. doi: 10.2217/fca.14.62.
Related trials for Diastolic Heart Failure
Not yet recruitingPhase 2
A Study of Bimagrumab in Participants With HFpEF
Heart Failure, Diastolic, Diastolic Heart Failure
RecruitingPhase 1
A Study of LY3971297 in Participants With Heart Failure
Heart Failure, Heart Failure, Diastolic
Not yet recruiting
Importance of Point-of-Care Ultrasound for Early Detection of Valvular and Cardiac Diseases (IMPROVE)
Valvular Heart Disease Patients, Aortic Stenosis
Recruiting
PrEcision Pacing in HFpEF: the PEP-HFpEF Trial
Heart Failure and Preserved Ejection Fraction, Heart Failure New York Heart Association Class III
Recruiting
Assessment of CCM in HF With Higher Ejection Fraction
Heart Failure, Heart Failure With Preserved Ejection Fraction
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.
Michael P Frenneaux, MBBS MD principal investigator
University of Aberdeen
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 Perhexiline in people with Diastolic Heart Failure, run by University of Aberdeen 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 6 inclusion and 4 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 5 Nov 2015. 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?
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?
Change in Peak oxygen consumption (Vo2max) (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 NCT00839228, 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 5 Nov 2015.