If you qualify and choose to participate, you will be asked to:
Your study team will explain each step and answer your questions.
Ninerafaxstat is an investigational drug being studied as a possible treatment for nHCM. It is designed to help the heart use energy more effectively without affecting pulse rate, blood pressure, or heart pumping function.
Because it is still being studied, ninerafaxstat has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory agency. It is only available through clinical trials.
No. This is a “double-blind” clinical study. Neither you nor the study team will know whether you are receiving ninerafaxstat or a placebo.
Participation lasts about 20 weeks. Joining the study is entirely voluntary. You may withdraw consent and choose to no longer participate in the trial at any time, for any reason, with no impact on your regular medical care.
Yes, participants may qualify for reimbursement related to clinical trial visits. Participants should let their clinical trial physician know if they require assistance.
Yes. You can continue seeing your usual healthcare providers. The study team may coordinate with them to help ensure your care is safe and consistent.
Yes. Your personal information will be kept strictly confidential. Only authorized study personnel will see your data, and any shared results will not contain participant names or identifying information.
If you are interested, please:
Participation in a clinical trial is an important decision involving many factors that should be carefully considered and discussed between the trial volunteer, the treating physician, and the clinical trial site investigator.
A clinical trial is a research study that helps scientists understand if a treatment is safe, how it works, and who it might help. This information is required before a regulatory agency can approve a treatment for broader use.
A placebo looks like the study medication but has no active ingredients. It helps researchers clearly compare results between groups.
Clinical trials are monitored by:
These groups help protect participant safety.
You have the right to:
These rights are protected by ethical and legal standards.
Ninerafaxstat is an investigational drug and not approved for any indication by regulatory agencies like the FDA. Its safety and effectiveness have not been established. Participation in FORTITUDE-HCM is limited to individuals who meet study eligibility criteria. All potential risks and benefits will be explained during the informed consent process.
