Hypertrophic CardiomyopathyPhase 2NCT00011076
Pirfenidone to Treat Hypertrophic Cardiomyopathy
A Phase 2 study testing Pirfenidone in people with Hypertrophic Cardiomyopathy, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
This study will examine the effectiveness of the drug pirfenidone (Deskar) in improving heart function in patients with hypertrophic cardiomyopathy (HCM). Stiffening of the heart muscle in patients with HCM impairs the heart's ability to relax and thus fill and empty properly. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hypertrophic Cardiomyopathy
- What is being tested?
- Pirfenidone
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2001to Apr 2003
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or female patient aged 20 to 75 years.
- Cardiac symptoms: New York Heart Association Functional Class II-IV despite medical therapy.
- Maximum LV wall thickness greater than 15 mm assessed by echo or MRI.
- Mean pulmonary capillary wedge pressure of greater than or equal to 18 mm Hg and/or LV end-diastolic pressure of greater than or equal to 18 mm Hg.
Who cannot join
- LV outflow tract gradient greater than 40 mm Hg by cardiac catheterization.
- Coronary artery disease (greater than 50% arterial luminal narrowing of a major epicardial vessel).
- Chronic atrial flutter/fibrillation
- Pregnancy and breast-feeding.
- Treatment with immunosuppressant medication in last 90 days.
- Active neoplastic disease.
- Significant renal (serum creatinine greater than 1.5 x upper reference limit) or hepatic (transaminases greater than 2 x upper reference limit) dysfunction.
- Use of over-the-counter medications, or herbal therapies that may be cardioactive.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Heart, Lung and Blood Institute (NHLBI)
Bethesda, Maryland, 20892
About this study
Official title: Double-Blind Placebo-Controlled Study of Pirfenidone, A Novel Anti-Fibrotic Drug in Symptomatic Patients With Hypertrophic Cardiomyopathy (HCM) Associated With Left Ventricular Diastolic Function
This study will examine the effectiveness of the drug pirfenidone (Deskar) in improving heart function in patients with hypertrophic cardiomyopathy (HCM). Stiffening of the heart muscle in patients with HCM impairs the heart's ability to relax and thus fill and empty properly. This can lead to heart failure, breathlessness and excessive fatigue. The heart's inability to relax may be due to scarring, or fibrosis, in the muscle wall. This study will test whether pirfenidone can reduce fibrosis, improve heart relaxation and reduce abnormal heart rhythms.
Men and women 20 to 75 years old with HCM may be eligible for this study. Participants will undergo a physical examination, blood tests, and other tests and procedures, described below, to assess heart function. When the tests are completed, patients will be randomly assigned to one of two treatment groups. One group will take a pirfenidone capsule and the other will take a placebo (a look-alike pill with no active ingredient) twice a day with meals for 6 months. For the pirfenidone group, the dose of drug will be increased gradually from 400 to 800 milligrams. At the end of 6 months, all patients will repeat the physical examination and heart tests that were done before starting medication. These include:
- Electrocardiogram (ECG) - electrodes are attached to the heart to record the heart's electrical activity, providing information on the heartbeat.
- Echocardiogram - a probe held against the chest wall uses sound waves to produce images of the heart, providing information on the function of the heart chambers.
- 24-hour Holter monitor - a 24-hour recording of the electrical activity of the heart monitors for abnormal heartbeats or conduction abnormalities.
- Magnetic resonance imaging (MRI) - Radiowaves and a strong magnetic field are used to produce images of the heart, providing information on the thickness and movement of the heart muscle.
- Radionuclide angiogram - a radioactive tracer is injected into a vein and a special camera is used to scan the heart, providing information on the beating motion of the heart. Scans are obtained at rest and after exercise.
- Cardiac (heart) catheterization - a catheter (thin plastic tube) is inserted into a blood vessel in the groin and advanced to the heart to record pressures and take pictures inside the heart.
- Electrophysiology study - a catheter is inserted into a blood vessel in the groin and advanced to the heart to record electrical activity, providing information on abnormal heart rhythms. This procedure is done at the time of the heart catheterization.
- Cardiac biopsy - a catheter is inserted into a blood vessel in the groin and advanced to the heart to remove a small sample of heart muscle for microscopic examination. This procedure is done at the end of the heart catheterization.
Read the detailed description
Left ventricular (LV) diastolic dysfunction and arrhythmias are important causes of morbidity and mortality in HCM. These abnormalities are believed to be in part due to myocardial fibrosis which frequently complicates HCM. Several studies indicate that pirfenidine can safely inhibit progression or cause regression of fibrotic lesions. We therefore propose to perform a six-month study that examines the ability of pirfenidone to improve LV diastolic dysfunction, exercise performance, and electrophysiologic abnormalities in symptomatic patients with severe HCM.
- Conditions
- Hypertrophic Cardiomyopathy
- Being tested
- Pirfenidone
- Keywords
- Deskar, Fibrosis, Myocardium, Scarring, Arrhythmia, Hypertrophic Cardiomyopathy, Left Ventricular Diastolic Dysfunction, Myocardial Fibrosis
- Medical subject headings
- Cardiomyopathy, Hypertrophic, Fibrosis, Cicatrix, Arrhythmias, Cardiac, Ventricular Dysfunction, Left
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PirfenidoneDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2001 (estimated)
- Study startFeb 2001
- Study completionApr 2003
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)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 National Heart, Lung, and Blood Institute (NHLBI)The 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
- Enrolment
- 50 participants
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00011076
- Sponsor's protocol ID
- 010066
- Other IDs
- 01-H-0066
- Status last verified
- Apr 2003
- First submitted
- 9 Feb 2001
- Last updated
- 4 Mar 2008
- Results posted
- No
Publications
Chang AC, McAreavey D, Fananapazir L. Identification of patients with hypertrophic cardiomyopathy at high risk for sudden death. Curr Opin Cardiol. 1995 Jan;10(1):9-15. doi: 10.1097/00001573-199501000-00003.
PubMed 7787270Bonow RO. Left ventricular diastolic function in hypertrophic cardiomyopathy. Herz. 1991 Feb;16(1):13-21.
PubMed 2026381Beranek JT. Fibrosis in hypertrophic cardiomyopathy: its possible pathogenesis. J Am Coll Cardiol. 1990 Aug;16(2):519. doi: 10.1016/0735-1097(90)90615-v. No abstract available.
PubMed 2373833
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind Placebo-Controlled Study of Pirfenidone, A Novel Anti-Fibrotic Drug in Symptomatic Patients With Hypertrophic Cardiomyopathy (HCM) Associated With Left Ventricular Diastolic Function
- ClinicalTrials.gov ID
- NCT00011076
- Acronym / sponsor ID
- 010066
- Conditions
- Hypertrophic Cardiomyopathy
- Keywords
- Deskar, Fibrosis, Myocardium, Scarring, Arrhythmia, Hypertrophic Cardiomyopathy, Left Ventricular Diastolic Dysfunction, Myocardial Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 50 participants
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- Feb 2001
- Study completion
- Apr 2003
- First posted
- 12 Feb 2001 (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.
This study will examine the effectiveness of the drug pirfenidone (Deskar) in improving heart function in patients with hypertrophic cardiomyopathy (HCM). Stiffening of the heart muscle in patients with HCM impairs the heart's ability to relax and thus fill and empty properly. This can lead to heart failure, breathlessness and excessive fatigue. The heart's inability to relax may be due to scarring, or fibrosis, in the muscle wall. This study will test whether pirfenidone can reduce fibrosis, improve heart relaxation and reduce abnormal heart rhythms.
Men and women 20 to 75 years old with HCM may be eligible for this study. Participants will undergo a physical examination, blood tests, and other tests and procedures, described below, to assess heart function. When the tests are completed, patients will be randomly assigned to one of two treatment groups. One group will take a pirfenidone capsule and the other will take a placebo (a look-alike pill with no active ingredient) twice a day with meals for 6 months. For the pirfenidone group, the dose of drug will be increased gradually from 400 to 800 milligrams. At the end of 6 months, all patients will repeat the physical examination and heart tests that were done before starting medication. These include:
- Electrocardiogram (ECG) - electrodes are attached to the heart to record the heart's electrical activity, providing information on the heartbeat.
- Echocardiogram - a probe held against the chest wall uses sound waves to produce images of the heart, providing information on the function of the heart chambers.
- 24-hour Holter monitor - a 24-hour recording of the electrical activity of the heart monitors for abnormal heartbeats or conduction abnormalities.
- Magnetic resonance imaging (MRI) - Radiowaves and a strong magnetic field are used to produce images of the heart, providing information on the thickness and movement of the heart muscle.
- Radionuclide angiogram - a radioactive tracer is injected into a vein and a special camera is used to scan the heart, providing information on the beating motion of the heart. Scans are obtained at rest and after exercise.
- Cardiac (heart) catheterization - a catheter (thin plastic tube) is inserted into a blood vessel in the groin and advanced to the heart to record pressures and take pictures inside the heart.
- Electrophysiology study - a catheter is inserted into a blood vessel in the groin and advanced to the heart to record electrical activity, providing information on abnormal heart rhythms. This procedure is done at the time of the heart catheterization.
- Cardiac biopsy - a catheter is inserted into a blood vessel in the groin and advanced to the heart to remove a small sample of heart muscle for microscopic examination. This procedure is done at the end of the heart catheterization.
Detailed description
Left ventricular (LV) diastolic dysfunction and arrhythmias are important causes of morbidity and mortality in HCM. These abnormalities are believed to be in part due to myocardial fibrosis which frequently complicates HCM. Several studies indicate that pirfenidine can safely inhibit progression or cause regression of fibrotic lesions. We therefore propose to perform a six-month study that examines the ability of pirfenidone to improve LV diastolic dysfunction, exercise performance, and electrophysiologic abnormalities in symptomatic patients with severe HCM.
Study design
- Primary purpose
- Treatment
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male or female patient aged 20 to 75 years.
- Cardiac symptoms: New York Heart Association Functional Class II-IV despite medical therapy.
- Maximum LV wall thickness greater than 15 mm assessed by echo or MRI.
- Mean pulmonary capillary wedge pressure of greater than or equal to 18 mm Hg and/or LV end-diastolic pressure of greater than or equal to 18 mm Hg.
Key exclusion criteria (8 listed)
- LV outflow tract gradient greater than 40 mm Hg by cardiac catheterization.
- Coronary artery disease (greater than 50% arterial luminal narrowing of a major epicardial vessel).
- Chronic atrial flutter/fibrillation
- Pregnancy and breast-feeding.
- Treatment with immunosuppressant medication in last 90 days.
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 |
|---|---|---|
| Pirfenidone | Drug | – |
Primary outcome
Not reported in the source record.
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 4 Mar 2008.
- Background: PubMed 7787270Chang AC, McAreavey D, Fananapazir L. Identification of patients with hypertrophic cardiomyopathy at high risk for sudden death. Curr Opin Cardiol. 1995 Jan;10(1):9-15. doi: 10.1097/00001573-199501000-00003.
- Background: PubMed 2026381Bonow RO. Left ventricular diastolic function in hypertrophic cardiomyopathy. Herz. 1991 Feb;16(1):13-21.
- Background: PubMed 2373833Beranek JT. Fibrosis in hypertrophic cardiomyopathy: its possible pathogenesis. J Am Coll Cardiol. 1990 Aug;16(2):519. doi: 10.1016/0735-1097(90)90615-v. No abstract available.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 Pirfenidone in people with Hypertrophic Cardiomyopathy, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
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 8 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 4 Mar 2008. 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 States. 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.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00011076, which also lists 3 linked publications.
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 4 Mar 2008.