Who may participate
Joining a clinical trial is an important and personal decision. Your doctor may have mentioned the REALM-DCM study to you, or perhaps you found your way here by yourself. Either way, thank you for your interest.
This study might be right for you if:
- you are aged 18 years and older,
- you have symptomatic dilated cardiomyopathy (DCM),
- your DCM is due to a mutation in the LMNA gene, which encodes the lamin A/C protein (Participants without a pre-identified LMNA gene mutation will be tested for the mutation during the screening phase of the study), and
- you have an implanted defibrillator (ICD or CRT-D).
If you are interested in participating, you’ll review the details with the study team and ask any questions you have. If you choose to participate, you will sign the informed consent document and continue with screening, to confirm whether you meet the eligibility criteria and are able to participate.
Each clinical trial has its own guidelines for who can participate, called eligibility criteria. However, only the research study staff can determine if this study is right for you.
What to expect
Before you begin the study, you will have medical tests and procedures to help the study doctor decide if this study is right for you. If this study is right for you, you will return to the study site for a baseline visit.
After the baseline visit, you will return to the study clinic at weeks 4, 8, 12 and 24, and then every 12 weeks until the end of the study. At some clinic visits (including the screening and baseline visits) you will be asked to complete a test to see how far you can walk in 6 minutes. Additionally, the study doctor will review your medical history, give you a physical exam and conduct some tests, including taking blood and urine samples. This is a two-part study: Part 1 is made up of a randomized and blinded period while Part 2 is made up of an open-label period. Participation in the study will last at least 24 weeks (6 months).
Additional clinic visits every 12 weeks until study completion
About Dilated Cardiomyopathy
Dilated cardiomyopathy (DCM) affects the heart's ability to pump blood efficiently. The disease often starts in the heart's main pumping chamber, the left ventricle. The heart muscle begins to dilate, stretch and become thinner. As the heart becomes weaker, heart failure can occur.
Often, the cause of DCM is unknown, referred to as idiopathic DCM. There is growing evidence that most idiopathic DCM is genetic and can run in families. This study is evaluating how effective a new study drug is for treating adults with dilated cardiomyopathy due to a genetic mutation called Lamin A/C (LMNA).
Frequently Asked Questions
How common is DCM?
DCM is one of the leading causes of heart failure worldwide. It accounts for about 30% of all heart failure hospitalizations in the United States. DCM is also the most common indication for needing a heart transplant.
What are the symptoms of DCM?
If you have DCM, you're likely to have signs and symptoms of heart failure or arrhythmias caused by your condition. Signs and symptoms include:
• Shortness of breath (dyspnea) when you're active or lying down
• Reduced ability to exercise
• Swelling (edema) in your legs, ankles and feet
• Swelling of your abdomen due to fluid buildup (ascites)
• Chest pain
• Extra or unusual sounds heard when your heart beats (heart murmurs)
How can DCM affect my quality of life?
Symptoms vary from individual to individual. As the disease advances, individuals with DCM may report impairments to their quality of life and psychological well-being due to heart failure related symptoms.
What is LMNA-related DCM?
Lamins are important proteins that provide structural and functional support for all cells in the body. The lamin A/C (LMNA) gene is responsible for making sure lamins are properly made to support our cell’s functions. Mutations in the Lamin A/C (LMNA) gene has been associated with DCM. Specifically, when LMNA mutations occur, lamin proteins are not made correctly and cannot provide the necessary support for cardiac cells. Heart cells with LMNA mutations can become inflamed and damaged, weakening the heart and resulting in LMNA-related DCM.
How is LMNA-related DCM diagnosed?
LMNA-DCM should be suspected in individuals with DCM (enlarged left ventricle and/or reduced heart function of unknown origin). Genetic screening for LMNA mutations can be used to confirm LMNA-related DCM. Importantly, not all carriers of an LMNA mutation will develop DCM and the disease may develop at different ages.
Can LMNA-related DCM be passed on to children? Are my chances of LMNA-related DCM increased if I have family members with the diagnosis?
LMNA mutations can be passed on to children and the chances of LMNA-related DCM increases if you have a family member with the diagnosis. Because of that, molecular genetic testing can be offered to at-risk relatives once a family member has been identified with LMNA-related DCM. This enables prompt diagnosis, surveillance, and treatment for those in whom the LMNA pathogenic variant has been detected. If genetic testing is not possible, it is recommended that first-degree relatives are evaluated periodically to determine the presence of DCM. It is important to note that only certain features of the disease may be present in some individuals even if they share the same mutation.
Why is this study drug being studied to treat LMNA-related DCM?
As of today, no therapies have been developed to specifically treat LMNA-related DCM. Despite advances in medical therapies, there is still high unmet need to treat individuals with LMNA-related DCM. The main purpose of this research study is to evaluate the effect of the study drug on your ability to walk over a 6-minute period (as measured by the 6-minute walk test) compared to a placebo (study drug without active ingredients.)
Is the study recruiting and how do I find a site conducting the study?
How do I get on a list for entry into the study?
Pfizer does not maintain a list of candidates seeking to participate in this study. Each study site will manage recruitment and enrollment activities. We encourage potential participants to work with their doctor to identify the best site to work with.
How can I get more information as this point in time?
What if we have several people with LMNA-related DCM in our family?
Any person interested in participating in this study may learn more about the study via the contact information above. If you are already participating in the study and have family members interested in learning more about the study, please consult your Study Doctor.
If multiple people from the same family participate to the study, will they be co-randomized?
Prior to participating in the study, each person must consent to be part of the study. Once consented, participants will be evaluated to determine if they meet all the requirements to be part of the study. If all requirements are met, each participant will then be individually randomized (like flipping a coin) to receive either study drug or placebo.
Why is there a placebo group?
Our mission is to research and develop medicines that are safe and effective. Placebo-controlled studies are a rigorous design to evaluate the safety and efficacy of a new medicine/new mode of action. It removes the potential bias that often occurs when there is hope that an experimental treatment might be beneficial.
Participants randomized to the placebo group at the start of our study are not excluded from the study. After all participants complete the first 24 weeks, participants in the placebo group will have the opportunity to receive study drug if they meet eligibility criteria. In other terms, participants in the placebo group experience a delay before receiving the study drug.
What happens once I complete the study?
Availability of the study drug following the study's closure through either a separate rollover extension protocol or expanded access/compassionate use is at the discretion of the Sponsor and subject to local conditions and regulations.
Will you be accepting international/cross-border participants?
Pfizer is not involved in study participant selection. This should be discussed between potential participants and study sites. Sites should ensure that decisions about enrolling patients from other countries are aligned with all relevant regulatory laws/regulations.
Will priority be given to those individuals who live closer to a research site; i.e., will there be geographical preferences?
Pfizer will not control enrollment or selection for the study and there are no rules associated with the study about how close to the site a participant must live. Each site will manage its own enrollment. Participation in the study will be a discussion between potential participants and the study doctor.
Is there an idea about the possible price of treatment?
We do not have that information at the moment. Pfizer will continue to work with all stakeholders, including regulators and payers, hoping to bring this potential therapy to the broad LMNA-DCM patient population.
What can I expect my out of pocket expenses to be if I participate in this trial?
Some out of pocket expenses may be reimbursed; however, this would need to be discussed with your study doctor.