My clinical trial experience
I have been living with metastatic breast cancer (MBC) since the autumn of 2016. During this time, I’ve been enrolled in two clinical trials. One with Genentech, an early biotech pioneer that is now part of pharmaceutical giant Roche. The other was with Olema Pharmaceuticals, a recent biotech startup (founded in 2206) that’s focused on endocrine-driven cancers.
My first one, sponsored by Genentech, was a very positive experience. It was for a selective estrogen receptor degrader for ER+ HER2-negative breast cancer, which is my cancer type. I was in this particular study for three years, with no side effects. Then gradually those tumor markers began trending upward again and my oncologist recommended another option. After the relative success of this first study (I keep reminding myself that with metastatic breast cancer, the goal is to delay disease progression and buy more time), I said I was willing and open to trying another one.
Luckily, I was qualified me to join another open study for a drug from a new pharmaceutical company focused on endocrine-driven cancers, Olema Oncology. Unfortunately, that trial did not turn out so well for me. I experienced some uncomfortable side effects. The pills had to be taken on an empty stomach, to which my stomach responded: I don’t think so! I had trouble swallowing and developed what I can only describe as an unpleasant form of nausea, or maybe the feeling that people get with acid reflux. I probably would have been willing to power through that discomfort if the drug was getting the job done, but once again, the tumor markers started heading in the wrong direction.
If another clinical trial opportunity arises, I am ready and willing to give another go. For stage 4 cancers, clinical trials could be the best option and the only hope for stopping disease progression and giving us more time.
I am quite confident that I am still alive and doing well because of pharmaceutical advances enabled by clinical trials. That’s why I’m writing this article, to help other advanced cancer patients understand the basics of what’s involved and also address two concerns many people fear:
Clinical trial fear and hesitancy
In my experience, people have some typical responses to clinical trials. Some people express open curiosity, others are hesitant, and a few people display resistance that sometimes borders on outright hostility. The hesitancy comes from two major concerns:
- Getting a placebo instead of actual treatment.
- Being a guinea pig in an experiment that could go awry.
I’ll give longer responses later, in a Q&A section, but since these are the proverbial elephants in the room, I thought I’d get to it sooner rather than later. The short answer to number 1 is that it’s unlikely you will get a placebo. The short answer to number 2 is that you are not a guinea pig or a lab rat; you are a human being receiving an early stage treatment possibility while contributing to the knowledge that will be needed to bring that treatment to market. That said, it’s a very personal choice, and one I hope you will at least explore and consider.
Clinical trial jargon
Let’s start with a review of the variables and language of clinical trials, or studies as I’ll sometimes call them.
Main study types
- Interventional studies (better known as clinical trials) test or try a drug, medical device, activity, or procedure in people to evaluate direct impacts of treatment or preventive measures on disease. Each study design has specific outcome measures.
- Observational studies (also called epidemiological studies and including patient registries), are those where the investigator is not acting upon study participants, but instead observing natural relationships between factors and outcomes.
Recruitment status
| Not yet recruiting | The study has not begun recruiting participants. |
| Recruiting | The study is currently recruiting participants. |
| Enrolling by invitation | The study is selecting participants from a particular group of people, decided in advance by the researchers. |
| Active, not recruiting | The study is ongoing, but potential participants are not currently being recruited or enrolled. |
| Suspended | The study has stopped early but may start again. |
| Terminated | The study has stopped early and will not start again. |
| Completed | The study has ended normally. |
| Withdrawn | The study stopped early, before enrolling its first participant. |
| Unknown | The status has not been verified within the past 2 years. |
Study start date
You may see either an actual date, which is when the first participant was enrolled, or an estimated date when the researchers expect to begin.
Primary completion date
Again, it could be the actual or estimated date on which the last participant was examined or received an intervention to collect final data for the primary outcome measure.
Outcomes
| Primary Outcome Measures | Secondary Outcome Measures |
| The planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. | A planned outcome measure not as important as the primary outcome measure, but still of interest to researchers. Most clinical studies have more than one secondary outcome measure. |
Study completion date
This is the last participant’s last visit, either an actual or estimated date on which the last participant was (or will be) examined or received an intervention to collect final data for the primary outcome measures, secondary outcome measures, and adverse events.
In most of the trial options I’ve seen, the primary completion date and study completion date have been the same.
Arm
An arm is a subgroup of participants that receives a specific intervention or treatment, according to the trial’s protocol. In one of my studies, I was in the arm that also included palbociclib, which is a standard treatment for metastatic breast cancer marketed as IBRANCE.
Randomization and masked studies
Randomization and masking (or blinding) are processes used to prevent bias in the interpretation of clinical trial outcomes.
Eligibility criteria
All the requirements for participation. Some are general, like age or gender, and some are very specific, requiring you to know the particulars of your cancer.
For example, for one of the studies I participated in, I needed to meet the following criteria to be included:
“Histologically documented advanced or metastatic solid tumor with KRAS G12C mutation.”
And if I had had any of the following, I would have been excluded:
- Active brain metastases.
- Malabsorption or other condition that interferes with enteral absorption.
- Clinically significant cardiovascular dysfunction or liver disease.
Contacts and locations
This is where you can find the name of the drug company (that’s generally what it will be) sponsoring the study and basic contact information like a phone number and email. You will also see the study locations, but if you don’t see your cancer center on the list, it doesn’t mean you can’t gain access elsewhere. When I looked at the study locations for the two trials I was on, my hospital, the University of Colorado, was not listed for either of them, so don’t let that be a deterrent.
Clinical trial questions and concerns
What about privacy and confidentiality?
You are not identified in a clinical trial by your name or Social Security number. Each participant is assigned a unique identifying code. In addition, physicians must obtain written HIPAA authorization before publishing papers or making presentations containing protected health information (PHI)
Will I be compensated in some way?
Generally, no. It’s possible that you will get reimbursed for some travel expenses like lodging and transportation, and you should by all means inquire about this. Otherwise, if this lack of remuneration seems unfair, put on your altruistic hat and think about future generations of cancer patients you could be helping. That’s how I view it. I may as well find some good in this disease.
Are placebos used in cancer clinical trials?
According to Cancer.net, the use of placebos (an inactive substance) in cancer clinical trials is rare. The stakes are too high! If a placebo is part of the study, the study must follow certain rules to monitor and protect patient health and ensure patients receive outstanding supportive care.
Will I just be a guinea pig for the pharma industry?
I’ve heard people say they would never go on a clinical trial because they don’t want to be a guinea pig. All I can say is that I have never felt that way. I was treated with the utmost care and respect during both the clinical trials I’ve been in so far, not to mention all my appointments and refills were done for me! Plus I had no extra out-of-pocket costs for the study drug. And while every trial is different, the sponsor often pays for research-related expenses like travel and hotel expenses.
One last thought: maybe what I really feel is that all of us dealing with advanced cancer are guinea pigs, are we not? After all, it’s a whole new cancer arena we’re in. I prefer to look at it in a positive light: I am helping other people with metastatic breast cancer that are coming right behind. Cancer patients before me have paved a road for me to follow; I want to continue building those roads into a future where treatment is replaced by vaccination and other preventative measures, or better yet, the eradication of cancer.
Why would I want to support a process that likely involved living creatures held captive in lab cages?
It’s true that many rats and mice have sacrificed freedom, comfort, and maybe even their lives in the name of clinical research. You may not want to support the process, and that is your choice and your right. Just know that any in-market drug, whether prescription or over-the-counter, likely began with research using rats or mice, which make up the vast majority of laboratory animals. Rodents have genetic, biological and behavior characteristics that closely resemble those of humans, and many symptoms of human conditions can be replicated in mice and rats.
To end this on a more positive note and with greater hope for these creatures:
- Researchers are expected to follow NIH policies on the humane care and use of laboratory animals.
- Perhaps new technologies like artificial intelligence will make rodent testing obsolete one day.
Bottom line about clinical trials
Clinical trials are a source of treatment and hope for advanced cancer patients. In my view, they also add greater meaning and purpose to my metastatic cancer experience. I take pride in knowing that I have (and likely will again) contribute to a body of knowledge that will help researchers continue to innovate and develop and refine new treatments.
Hopefully, one day, reach that holy grail we all want–a cure or some other end to cancer.
My clinical trial advice:
- If your oncologist suggests one, be open to it.
- If your oncologist doesn’t offer clinical trials as a treatment option, ASK!
- Do research of your own (with the help of your medical record for the particulars of your disease) and discuss with your oncologist.
- Know that you can end your clinical trial participation at any time, for any reason.
How to find suitable clinical trial options
Clinical trial participation is often initiated by oncologists, but it’s becoming increasingly important for patients to be actively engaged in their treatment options. It may seem like a daunting task, but the following two online resources make the process much easier:
- Clinicaltrials.gov is a good first place to start your research. As a resource provided by the U.S. National Library of Medicine, it’s a database of privately and publicly funded clinical studies around the world.
- TrialJectory is an excellent resource developed by a private company. It taps into the clinicaltrials.gov database and other sources, but it’s more user friendly, presenting you with a series of questions you can answer with a click of a mouse to narrow the results for the best fit for your situation.
- More helpful and easy information on clinical trials from the US National Institutes of Health.
If you are dealing with a stage 4 cancer diagnosis, you may be presented with the option of joining a clinical trial, if not, you should definitely ask your oncologist and consider it. The life you save (or extend) may be your own, not to mention you may be giving hope and treatment options to future patients.