Frequently Asked Questions
About CEDAR-HCM
What are my responsibilities in the clinical trial?
Trial participants who participate in the clinical trial will be responsible for visiting their clinical trial site for screening, trial treatment visits, and follow-up appointments. During the clinical trial period, trial participants will be required to visit their clinical trial site for about 16 clinic visits over the entire study duration, though some participants will have more visits. You will be asked to provide biological samples (such as blood or urine) and undergo procedures that might be different from a regular medical examination. This study will involve physical examinations, vital sign measurements, and tests to see how your heart is working. Any additional responsibilities and clinical trial duration will be explained by the clinical site trial investigator physician.
If you complete Period 1 and Period 2, your participation will involve about 16 visits to the research site and the study will last up to 78 weeks (about 1 year and 6.5 months). Over the entire study, you’ll have periodic:
- Echocardiograms
- Electrocardiograms (ECGs)
- Blood draws
- Questionnaires about your health
You will also have the option to participate in the following:
- Optional genetic sub-study (blood sample collected as part of a study visit blood draw)
- Optional blood collection for future testing (blood sample collected as part of a study visit blood draw)
- Optional pharmacokinetics sub-study (additional testing to measure the amount of aficamten in your blood at various times)
- Optional cardiac MRI sub-study (cardiac MRI to assess the effects of the study medicine dosing on the heart’s structure and function)
Can I leave the clinical trial after I start?
Yes, you may withdraw consent and choose to no longer participate in the clinical trial at any time.
Does this clinical trial offer travel reimbursement?
Yes, the trial participant and up to one caregiver may qualify for reimbursement related to clinical trial visits. Trial participants, or their caregiver, should let their clinical trial physician know if they require assistance.
How do I get started?
Please visit https://clinicaltrials.gov/study/NCT06412666 for more information about this clinical trial, as well as a listing of all clinical trial sites. In addition, you may contact [email protected] or 650-624-2929 for more information.
Participation in a clinical trial is an important decision that is properly considered between the trial volunteer, the treating physician, and the clinical trial site investigator. There are many factors the physician and clinical trial site investigator will take into consideration to determine if participating in a clinical trial is appropriate.
About Clinical Trials
A clinical research study (also called a clinical trial) is a medical study that helps to answer important questions about an investigational drug – these include studying whether an investigational drug has any beneficial effects in treating a certain condition and what the potential side effects are. All medications must be tested in a clinical research study before they can be approved and prescribed to patients.
Clinical trials are conducted to answer research questions and to help researchers learn if a certain investigational drug is safe and effective. The main questions the researchers want to answer are:
Benefits
Being in this study may not help you directly. But your participation in the study may benefit other people in the future by helping us learn more about your medical condition and develop drugs to help other pediatric patients with obstructive HCM.
Possible Side Effects
The most common side effects observed with aficamten in the recently completed Phase 3 clinical trial for adult patients with obstructive HCM were palpitations and hypertension. You may have side effects while in this study. Researchers do not know all of the potential side effects from this investigational agent.
Participating in a clinical trial is a voluntary opportunity, and thus, it is always up to the individual interested in participating to decide if they would like to do so. People participate in clinical trials for different reasons, some of which may be:
- They have not experienced improvement of the condition after using the currently approved drug(s)
- They want access to a new type of drug even if it is not guaranteed to be better or as good as already available drugs
- They want to contribute to the development of a new drug for others with a similar disease
A placebo is a substance designed to look like an investigational drug but contains no active ingredient. Clinical trials will use a placebo to test the safety and effectiveness of an investigational drug. Researchers learn from comparing the results of the placebo to the actual investigational drug.
Participating in a clinical trial involves collection of certain information about you, with your full consent. Medical information privacy is covered by the Privacy Rule and HIPAA; certain other personally identifiable information that you may provide may be covered by state privacy laws.
If you are a resident of certain states, like California (or Colorado, Connecticut, Delaware, Florida, Indiana, Iowa, Kentucky, Maryland, Minnesota, Montana, Nebraska, New Hampshire, New Jersey, Oregon, Tennessee, Texas, Utah, and Virginia), you may have certain privacy rights about your personally identifiable information. To view Cytokinetics’ Privacy Policy, please visit cytokinetics.com/privac y-policy/ or contact [email protected].
Aficamten is an investigational drug and is not currently approved by regulatory agencies, including the FDA, for any use. It is currently being studied in this and other trials to evaluate its safety and effectiveness.