Ninerafaxstat 4 nonobstructive HCM

Posted by hncm @hncm, 5 days ago

Since ninerafaxstat has good reports from Phase 1 and 2 clinical trials, does anyone know when and where Phase 3 will be held and how to volunteer? It's for nonobstructive (HNCM) Also is anyone now on Camzyos for HNCM? Any improvements?

Interested in more discussions like this? Go to the Hypertrophic Cardiomyopathy (HCM) Support Group.

Justin McClanahan, Moderator | @JustinMcClanahan | 5 days ago

Hello @hncm, here is a very brief overview of the research you are referring to:

"Safety and Efficacy of Metabolic Modulation With Ninerafaxstat in Patients With Nonobstructive Hypertrophic Cardiomyopathy"
- https://www.jacc.org/doi/10.1016/j.jacc.2024.03.387
---------
"Ninerafaxstat in the Treatment of Diabetic Cardiomyopathy and Nonobstructive Hypertrophic Cardiomyopathy"
- https://pubmed.ncbi.nlm.nih.gov/39194232/
---------
As you mentioned, the study concluded that it be moved to a Phase 3, but finding exactly when is unclear. Have you had a chance to discuss this research with your provider to inquire about whether or not you can become part of the research?

hncm | @hncm | 5 days ago

Thank you! I actually have read those two articles and they are excellent pieces of research-valid, reliable and have given me hope. In fact everything I've read recommends Phase 3 for HNCM and Ninerafaxstat, but so far there's no info on how and when to sign up to volunteer for clinical trials. BTW, I seem to know more about HNCM, HOCM, Ninerafaxstat and Mavacamten then my local cardiologists here on the Central Gulf Coast of FL! Also, my primary care doc seem to know very little and is unmotivated and even uncooperative to help with signing up for genetic testing--I've moving forward with that process--very slowly, on my own. I am 1.5 hrs away from a Center of Excellence (CEO) in Orlando and from May to November I'll be 1.5 hours away from Mayo in Rochester MN. I suspect I'll probably be working with Mayo next summer and fall to get my answers. I am a member of 4HCMA.org and maybe I'll get help with info there.

boatsforlife | @boatsforlife | 5 days ago

Hello @hncm . When I started camzyos, I understood that it was ONLY for obstructive (hon-obstructive was a show stopper), and only if a handful of other criteria were met as well. It's certainly possible that the obstructive/non-obstructive distinction may have changed - it's still a pretty new medication, and I expect there are all kinds of new lessons being learned as time goes on. It must be frustrating to have a member of your medical team being uncooperative when you are trying to get things sorted out. Keep the faith! I applaud your courage in being willing to be part of a phase 3 study of a potential new medication. Many people are justly nervous about being "guinea pigs", but these new medications will only become available with people like you being willing to put themselves on the line. Kudos!

hncm | @hncm | 5 days ago

Thanks for that . . . So far the side effects of Ninerafaxstat were "tolerable" in Phases 1 and 2, but of course the sample size of volunteers was small and there is no longitudinal data. The benefit (so far) is increased ability to exercise more efficiently. If I found out I have nHCM then I don't have any other meds currently available, so nothing to lose!