I have debated long and hard whether to disclose this information. I understand as the Founder and CEO of the Hypertrophic Cardiomyopathy Association (HCMA) that my actions have the potential to have an impact on others also living with a diagnosis of hypertrophic cardiomyopathy. Those of you who have followed the HCMA as well as my personal journey with HCM have known me to be very candid and direct about my own health. So when I was faced with the opportunities to participate in clinical trials as well as my new role as a co-principal investigator on a clinical trial in HCM I wondered what my personal role was going to be. I debated whether to share my personal experience with my own individual participation in a clinical trial or to withhold that information for fear others would not do their due diligence and only follow my lead.
I have spent many hours debating with myself and trusted friends and colleagues do I withhold this information or do I live completely transparently. A friend of mine encouraged me to “live your life ‘raw’”; I think that is excellent advice.
For the next few blog entries, I will be discussing my individual choice to participate in a specific clinical trial. It is my hope that this blog will inspire you to think about the possibility of participating in a clinical trial. I was going to withhold the name of the trial that I am participating in for fear that others would say if Lisa did it, it must be okay so I am going to do it to. Please do not put that responsibility and burden on me. I cannot state firmly enough that my decision may not be the right decision for anyone else. And for all I know I may be making a mistake for myself however, even if I am making a mistake for myself, I feel I am contributing to the overall knowledge and understanding of this particular research question. You must educate yourself and with your own doctors and families decide if these trials fit into your own individual life plan. Neither the HCMA nor do I advocate for specific trials except that they are the progressive pathways to potentially controlling and curing HCM. However, we do advocate for individual choice and that is what this blog is about.
If I am going to do this, share my experience in a clinical trial with all of you, it is important to understand my history with clinical trials. My first trial was in the late 1980s at the National Institutes of Health. It was a positive experience. Afterwards I then participated in more trials. Sadly, these were not good experiences. As many of you know, I lost my sister due to the actions of a researcher at the National Institute of Health, in 1995. It is incredibly important to say corrective action has been taken regarding his behavior and he is no longer a participant in HCM clinical trials nor is he employed any longer with the National Institutes of Health. One bad apple does not spoil the bunch. I did participate in the M-PATHY trial in the late 1990’s as an attempt to better understand the true role of DDD pacing in HCM, but nothing since. Later I participated in a small drug study, of a drug that was already on market but its usefulness in HCM was unclear. I also have given my blood for genetic research. These were small studies and the impact on the overall HCM community was important but this is a new day and the study designs are more substantial and the implications potentially much further reaching. It is with a great deal of personal reflection and the knowledge and understanding that we have very few treatment options available that are scientifically validated with clinical trials in hypertrophic cardiomyopathy. I know this is not an acceptable future for us, it is time we all stepped up to participate actively in HCM research. That is what I am choosing to do.
I began writeing this blog entry July 2, 2015. As of this day there are a number of functioning clinical trials. I am going to take you through my decision tree on why I have chosen not to participate in a few studies and then I will lead to the study that I was enrolled into this week.
Step one is to visit the HCMA website and review the current link to clinical trials.gov. This website will list all of the open and recruiting trial that are available. Upon review of this list I quickly found I was not a candidate for a few studies based on the following reasons.
As a matter of complete transparency, I work with some of the researchers and know them very well. I understand not all patients have personal contact regularly with them , however, through the HCMA and Clinicaltrials.gov, you can see that hopefully I can help give you assurances that the researchers and study coordinators are accessible and willing to speak to you and help you understand the process should you have any questions. Reach out to them they WILL get back to you!
Lisa’s personal decision tree on available trials: (for the sake of length I have identified why I do not qualify for the study and not each inclusion criteria – you can however use the links provided and see all of the inclusion and exclusion criteria for each – in cases where I qualify I have gone over all points.)
The VANISH trial principal investigator Dr. Carolyn Ho and colleagues. I personally am not a candidate for this trial because I have advanced hypertrophic cardiomyopathy that has moved into a low ejection fraction HCM. I am also too old – and that is not fun to admit! Therefore, my decision tree ends there – I am disqualified.
HCMR – principal investigator Dr. Chris Kramer and colleagues. I am not a candidate for this trial because it includes a cardiac MRI or CMR and due to my implantable defibrillator, I cannot have the diagnostic tool that is part of this trial. Therefore, my decision tree ends there – I am disqualified.
LIVE-HCM principal investigator Dr. Rachel Lampert and colleagues, of which I am one, as a co-principal investigator. As a co-principal investigator, I am not qualified to participate in a trial that I am in investigator on. Therefore, my decision tree ends there – I am disqualified.
Phase 1 study MyoKardia – study director Dr. Jonathan Fox. The inclusion criteria for this study states you must have a normal ejection fraction and the New York heart Association class I or II, meaning minimally symptomatic. Unfortunately, my HCM profile does not qualify for this study. Therefore, my decision tree and there – I am disqualified.
Liberty-HCM principal investigator Dr. Martin Maron. Eligibility criteria New York heart Association class II or higher, I qualify. Screening at baseline with peak VO2 under 80% of protected for age sex and weight, (previously I had had this test and know my scores – I have scored 17.2 – 18 predicted for my demographic 30, my percentage 74-76%), and sadly I qualify. Ability to perform an upgrade treadmill cardiopulmonary exercise test, I qualify.
Exclusion criteria known aortic valve stenosis, I do not have, therefore I qualify. Known coronary artery disease, I do not have, therefore I qualify. Left ventricular systolic dysfunction with an ejection fraction of under 50%, I do not have, therefore I qualify. Recent septal reduction procedure, I have not had, therefore I qualify.
I now move on to the next phase. I contacted my physician and I said I am interested in participating in this trial.
Morristown Medical Center – New Jersey
The research coordinator then contacted me. She asked me what a convenient time would be for me to come in for baseline evaluation testing and made an appointment.
My echocardiogram – to enter the trial (all photos are selfies)
We sat down and discussed the research protocol in detail. I was given ample time to review the informed consent documents and ask any questions that I may have. I was concerned about one item in the research outline. They want me to wear a ZIO patch heart monitor for two weeks straight. I have sensitive skin and I have found that even EKG electrodes causes skin irritation.
We discussed how long the ZIO patch had to stay on and my research coordinator asked the drug company what the requirements were and they advised to wear it as long as possible but if I couldn’t wear for the full two weeks they would take that under consideration, likely not a disqualification. I was presented with a second research informed consent document to consider requesting the review of biomarkers in the blood samples that I was giving I took some time to consider that as well. Ultimately, I decided that I would permit them to search for biomarkers and I signed that consent form.
Not my most attractive moment getting an ECG, however I did promise transparency didn’t I?
Just a little stick and the blood work is done!
Going through my list of things to do to participate in the study frankly was not a whole lot different from my annual exam from HCM anyway. I needed an echocardiogram, a VO2 stress test, blood work, air analysis, EKG and to complete the documentation with the research coordinator. After review of all of my test results, I will be advised whether I can proceed to the next level of the study.
Vo2 stress test – a perfect time for a selfie
On June 30th I spent the day at Morristown Medical Center and had all my testing completed and was fitted with my Zio patch.
The next day off to work with my Zio Patch – you can hardly see it.
A few days later and the Zio Patch was fine.
I have an appointment in three weeks to find out if I have been accepted into the study or not. If I am to be accepted into the study based upon my test results, I will take either a placebo or a drug that is in the classification of a sodium channel blocker for 24 weeks and I will repeat my exercise VO2 test and see if there has been any improvement, in addition I will complete another questionnaire.
I have opted for complete transparency here regarding my participation in this clinical trial and my reasons for not participating in other trials for one reason: I want to STOP HCM from taking from us all. My complete transparency includes some less than flattering pictures, which I hope do not come back and haunt me someday!
As most of you know I have dedicated my life in an effort to help improve and save the lives of others battling with hypertrophic cardiomyopathy. I have given my time, my intellect, my passion and my creativity to build the HCMA and to broaden our network of researchers and interested physicians however now is the time to put action in front of passion. The only way our currently reality of living with HCM is going to change in any meaningful way in the future for ourselves, for our children and for our children’s children is to have better treatments and ultimately one day a cure. We cannot reach better treatments or the ultimate Holy Grail of a “cure” without the participation of qualified patients for research trials.
I am updating this blog post on July 22, 2015. Today I received the complete results of my pre-testing and was fully admitted into the study. Todays appointment included a blood draw, a physical, urinalysis, interrogating my ICD, a second Zio Patch (updated Holtor Monitor type device) and my first dose of either a placebo or medication. A few hours after my first dose a second blood draw was done and I left. I am officially IN LIBERTY-HCM effective TODAY.
First dose of either placebo or medication. The starting dose is 5 pills, then one a day
A few weeks ago on Facebook I posed a question. I asked what would interest you in participating in a clinical trial? Some of the answers received were, frankly, unrealistic. Some examples are, “I want to guarantee it will work”, “I want free medical care for year”, “and I don’t want anything bad to happen to me”. Frankly responses like those made me giggle a little, to guarantee a drug (or device or test) is “going to work in everyone is not realistically an endpoint. Nothing in life is life is truly guaranteed… Nothing. Free medical care for year imagine how expensive all research would be if each trial participant had complete free healthcare for a year, a tad bit unrealistic to expect that kind of reimbursement. Nobody wants anything bad to happen to participants including the researchers and all of those involved in the Internal Review Board (IRB) process of conducting a medical trial and all reasonable safety precautions have been taken to ensure your safety. However, nothing is 100% safe when it comes to medical care and we all need to be realistic about that.
I read some other comments that were reasonable and practical statements such as:
- I would like my travel reimbursed.
- I would like convenient scheduling.
- It would be helpful if I could do it on a weekend.
- If my family can all participated the same time.
- Then there were those of you who said things like – I would be happy to help in any way I can to make the future better for my family and the rest of my HCM family.
Most of the comments here are very reasonable and when working with the study coordinators, most of these can be accommodated. Each study is a little bit different so travel reimbursement does vary but is available for most studies. Flexible scheduling is available, in most studies family participation can get a little tricky because of scheduling but it is probably possible. To all of you who want to give back to others I applaud you and I thank you.
So today, I embark on a new part of my journey! I am a research subject in a clinical trial, which aims to identify a new treatment for hypertrophic cardiomyopathy, and may in turn help others. I accept the risks personally and while I seek no benefit for myself, I remain hopeful that I might find one.
Please do not think, as I stated above, Lisa vetted this trial thereby it is the one I want to participate in. What is right for me may not be right, and in fact may be very wrong, for you. Please speak to the study coordinators and your personal physician to discuss participation in these and other clinical trials we need you, you need these clinical trials the question is… are you willing to get involved?
Onward and upward to a brighter tomorrow… and no laughing at the pictures of me participating in the trial! I will admit the study team and I did have a few laughs along the way and I am happy and excited to be a part of the future in a whole new way!
From my big heart to yours…. Stay well!
Your bighearted blogger