1. < Hypertrophic Cardiomyopathy (HCM)

Clinical trial

Posted by rmhc123 @rmhc123, Nov 25, 2024

Hi, my name is Rachelle and I’m unsure if I am already a member of this group. I have nHCM (non-obstructive) and currently a part of a clinical trial study at Emory University in Atlanta for the drug Aficamten. Is anyone else here taking part in this trial? It’s a little frustrating because it is a double blind study.

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

hillbilly1065 | @hillbilly1065 | Jan 24 5:35pm

I was never told which I had. Even to this day I don't know which one I took. I will eventually find out, they will send out the study. When the double-blind study was over we started the actual drug trial. I was just told I could continue if I wanted.

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rmhc123 | @rmhc123 | Jan 24 8:27pm

So you were given the actual drug to take? I’ve not been told that I will be given the drug when it is over. I was told there were no guarantees.

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Mentor
Debra, Volunteer Mentor | @karukgirl | Jan 25 4:49pm
In reply to @hillbilly1065 "A little about my health. I have HCM with obstruction, I have had 3 ablations over..." + (show)
@hillbilly1065

A little about my health. I have HCM with obstruction, I have had 3 ablations over the past few years. I have an ICD. I started the doubled-blind trial on 01 March 2023. Then started the active drug (aficamten) on 19 February 2024. I haven't got the double-blind results back yet but I believe I was on the Placebo. Prior to taking the active drug my life style was very limited. Since taking the active drug I have noticed great improvement. As I a stated in my previous post I was at my hospital yesterday for my 12 week follow up. I was going approximately once a month last year but now the visit are getting further apart. I was notified yesterday that I can now televisit if no issues but will have to come in 6 months. I was also told that they will be adding an additional 900 people for the trial worldwide and in hopes of the FDA approving aficamten this fall. I'm not sure that will happen but I think they are working toward that date. Even though I am feeling better and in my opinion doing well the echo yesterday showed my gradient level had increased. My doctor had concerned about this and mentioned the septal myectomy . I still have to process that though. I am currently taking 20 mg per day with no side-effects for myself.

I am very new this this forum stuff, so if I have left any thing out anyone wishes to know please just tell me. If I provided to much information let me know that as well. If I can provide anyone with more information just ask, I have found it is hard to find information on HCM especially personal details that are not readily available.

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Welcome to Mayo Clinic Connect @hillbilly1065! I'm glad you found this site and made your first post. We were all new here at one time, and I think you did an outstanding job describing your situation and being willing to share with others.
You never know who may be reading your posts and learn something they did not know before, so thank you!
I second the thoughts of @boatsforlife...many members will be curious to hear all about your adventures in clinical trials!
I had to have open heart surgery for my HOCM, and in my opinion it takes more courage to be in a clinical trial then have a septal myectomy! I do appreciate not having to take any medications anymore, and I got it over with in one fell swoop.
You are in the right place to share and learn from others just like you. That is what makes Mayo Connect so beneficial. Patients can talk to patients and share their knowledge and experience with each other. That is a blessing! Because many (as in most all) healthcare providers have no idea what it is like to live with HOCM.
So again...welcome aboard! And feel free to share whatever you wish!

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walkinggirl | @walkinggirl | Jan 26 7:25am

During the 1990's I was in a double blind study, the goal was to replicate a study conducted in Denmark (?) for Tamoxifen (a breast cancer drug), to study its preventative attributes (it does have them). I did not know if I was using the real thing or a placebo, no reactions. I was surprised when told I was on it, the staff administering the study thought it was hilarious that the placebo people noted more side effects. I think that when a drug makes it to the trials, much has been established for safety.

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rmhc123 | @rmhc123 | Jan 26 8:00am
In reply to @walkinggirl "During the 1990's I was in a double blind study, the goal was to replicate a..." + (show)
@walkinggirl

During the 1990's I was in a double blind study, the goal was to replicate a study conducted in Denmark (?) for Tamoxifen (a breast cancer drug), to study its preventative attributes (it does have them). I did not know if I was using the real thing or a placebo, no reactions. I was surprised when told I was on it, the staff administering the study thought it was hilarious that the placebo people noted more side effects. I think that when a drug makes it to the trials, much has been established for safety.

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That is very interesting. I am truly clueless as to whether I am on the drug or the placebo.
So you were eventually told? Also, you were given the option to take the drug when the study was over?
And wow- you have battled both breast cancer and now HCM?!?

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1 reply
walkinggirl | @walkinggirl | Jan 26 9:12am
In reply to @rmhc123 "That is very interesting. I am truly clueless as to whether I am on the drug..." + (show)
@rmhc123

That is very interesting. I am truly clueless as to whether I am on the drug or the placebo.
So you were eventually told? Also, you were given the option to take the drug when the study was over?
And wow- you have battled both breast cancer and now HCM?!?

Jump to this post

Yes, when the 5 year study was over, we were told. I was on it. The placebo people were given a choice - take Tamoxifen for 5 years or take part in a new study comparing Tamoxifen and another effective drug to see which was better. No, I did not have breast cancer, the study was to find out if it prevented breast cancer. One of the qualifications to take part was having a close relative diagnosed with it, it was my mother who eventually died from it. I was glad to be able to contribute to the body of knowledge in the mid 1990's. Mom's side is cancer, Dad's side is hearts.

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rmhc123 | @rmhc123 | Jan 26 10:06am

Wow. Thank you for sharing.

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