A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM)
This study is gathering information on people living with a heart condition called obstructive hypertrophic cardiomyopathy (HCM). Researchers want to understand how this condition is treated in everyday medical practice, particularly looking at a new medication called mavacamten, compared to other common treatments, or no treatment if previous options haven't worked. They are interested in seeing how patients' health changes over time, their experience with different treatments, and how safe and effective these treatments are. The study is particularly looking at this in the US and Europe to gather real-world data and help doctors make the best treatment choices for patients with HCM.
At a glance
What is this study about?
This study is exploring a heart condition called obstructive hypertrophic cardiomyopathy, or obstructive HCM for short. This is when the heart muscle becomes unusually thick, making it harder for the heart to pump blood effectively to the rest of the body. This can cause symptoms like shortness of breath, chest pain, or feeling lightheaded.
The main goal of this research is to understand how people with obstructive HCM are treated in their everyday lives, outside of strict research settings. Doctors want to see how patients respond to a newer medication called mavacamten, as well as to more traditional treatments or even when no treatment is being given due to side effects or if other options haven't helped. By collecting this 'real-world' information, the study aims to get a clearer picture of who gets which treatments, how well these treatments work over time, and if there are any safety concerns.
Ultimately, the information gathered from this study will help healthcare professionals better understand the journey of patients with obstructive HCM. This knowledge can then be used to improve treatment plans, guide important decisions for patients, and ensure they receive the most appropriate and effective care tailored to their individual needs.
Key takeaways
- This study gathers real-world information on living with obstructive HCM.
- It compares a new medicine (mavacamten) with other common treatments.
- The goal is to understand treatment patterns, how patients feel, and long-term results.
- Participation involves sharing medical information from your routine care.
- It helps doctors make better treatment choices for patients with HCM.
- Your medical care will not be affected by joining the study.
Who may be eligible?
To join this study, you need to be at least 18 years old and willing to provide your consent to take part. This study is specifically for people who have been diagnosed with obstructive hypertrophic cardiomyopathy (HCM).
In the UK, this diagnosis would typically follow guidelines from leading heart organisations. Your heart's main pumping chamber (left ventricle) would need to be thicker than usual, and there would be a blockage making it harder for blood to leave your heart (known as a 'gradient'). Your heart's pumping ability should be generally strong, and you should be experiencing symptoms like shortness of breath or chest pain.
As for your current treatment, you might already be taking medicines like beta-blockers, calcium channel blockers, or disopyramide, or starting mavacamten. You might also be eligible if you're not currently on treatment because previous medicines haven't worked for you or you couldn't tolerate them.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years or older?
- Do you have a diagnosis of obstructive hypertrophic cardiomyopathy (HCM)?
- Are you experiencing symptoms related to your HCM?
- Are you currently on treatment for HCM (like beta-blockers, calcium channel blockers, disopyramide, or mavacamten) or unable to tolerate previous treatments?
What does participation involve?
As a 'registry study', participation primarily involves allowing researchers to collect information from your routine medical records over time. This means there likely aren't extra visits or procedures specifically for the study. Instead, researchers would track your health status, how your obstructive HCM is managed, the treatments you receive (like mavacamten or other medications), and how your condition progresses during your regular hospital or clinic appointments. This allows them to understand real-world treatment patterns and outcomes without directly interfering with your care. The total duration of your participation would depend on how long the study continues to collect follow-up data.
Potential risks and benefits
Locations (99)
- Alaska Heart InstituteVerified postcodeAnchorage, United States· Recruiting
- Pima Heart and VascularVerified postcodeTucson, United States· Recruiting
- UAMSVerified postcodeLittle Rock, United States· Recruiting
- UC San Diego School of MedicineVerified postcodeLa Jolla, United States· Recruiting
- Keck School of Medicine of USC-UscVerified postcodeLos Angeles, United States· Recruiting
- Stanford Health Care Hospital & ClinicsVerified postcodePalo Alto, United States· Recruiting
- University Of California San Francisco Medical CenterVerified postcodeSan Francisco, United States· Recruiting
- UC Denver, AMCVerified postcodeAurora, United States· Recruiting
- Hartford HealthCareVerified postcodeHartford, United States· Recruiting
- MedStar Washington Hospital CenterVerified postcodeWashington D.C., United States· Recruiting
- Uf Health JacksonvilleVerified postcodeJacksonville, United States· Recruiting
- University of Hawaii Cancer CenterVerified postcodeHonolulu, United States· Recruiting
Common questions
What is obstructive hypertrophic cardiomyopathy (HCM)?
It's a heart condition where the heart muscle is unusually thick, making it harder for your heart to pump blood effectively and causing symptoms like breathlessness.
What is mavacamten?
Mavacamten is a newer medicine specifically designed to treat obstructive HCM.
Will I have to change my current treatment if I join?
No, this study observes how you are treated in real life. Your doctor will continue to make all decisions about your care and medication.
Will I have extra appointments?
Because this is a registry study, it's unlikely you'll have extra appointments. Information will mostly be collected from your routine medical visits.
Is this a trial where I might get a placebo?
No, this is not a traditional drug trial where you might get a placebo. It's an observational study looking at existing real-world treatments.
How to find out more
BMS Study Connect Contact Center www.BMSStudyConnect.com
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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