Clinical trial questions for newbie

Whether or not one wants to pursue a clinical trial option depends on many things.

1. First and foremost would be whether or not there is a viable treatment option for your condition. Back in the dark ages of melanoma (before 2011) when there were NO FDA approved treatment options other than interferon (which did absolutely nothing to improve OS and made you sick as a dog) or IL2 which as administered back then was a stay in the intensive care unit and a 10% response rate and given its toxicity was not generally an option unless you were Stage IV with inoperable disease – we all wanted a chance at a treatment via a clinical trial as ipi and nivo were cooking along there and outcomes to them were looking better than the options noted. So….if you have viable treatment options with odds that are as good or better than any current trial drugs – regular standard therapy is generally preferred.

2. Personality and money. Clinical trials are not for the faint of heart. They are their own crazy in addition to the treatment. Plus, they can be expensive. Most are not “free” as many may think. Pricing is variable, but for my nivo trial in 2010 the only thing that was “given” to me was the trial drug and the labs THEY wanted to draw for the study. All other expenses (from nursing in the unit, to tubing, to doctor visits, to REQUIRED for participation scans and labs – not to mention travel to the location) were my problem. If my insurance company covered them great. But if they didn’t and/or costs like deductibles and patient percentages not covered – they were mine to pay. Further, most trial paper work comes with a disclaimer that states if you grow three heads, have side effects, or any other untoward event – the coverage for diagnosing and treating that event is NOT the responsibility of the doc or institution running the trial nor the drug manufacturer. So, that’s fun. You really have to pay attention to the fine print.

3. Understanding what you are offered. Phase 1 trials are basically dosing trials. They are not technically done to see who they cure, rather they are done to determine the amount of med needed to get a response versus the amount of drug that makes people freak. Using my phase 1 nivo trial as an example – we were divided into 3 arms of Stage IV patients with active disease and 3 arms of Stage IV patients who had their disease removed via surgery or radiation. The first arm got only 1mg/kg of the drug. When we did not spontaneously combust, the next got 3mg/kg. And the third got 10mg/kg. The results of this and other studies at that time led to the use of the 3mg/kg dosing schedule which has now been rounded to the 240 mg dose every 2 weeks or the 480 mg dose every 4. Additionally, the drugs used in Phase 1 trials are relatively new with little data in humans to back up their use. So, there is a risk that they will not benefit the rattie as much as desired. HOWEVER, the good thing about Phase 1 trials is that everyone gets the drug. In Phase 2/3 trials – the trial drug is often randomized. With some arms getting nothing or a lesser drug. The recent/ongoing trials for ipi/nivo as adjuvant for melanoma are a pretty good deal because the participants get either the desired combo or nivo alone. So even if you get “only” the nivo arm, you are still getting a good treatment. Unfortunately, not all trial options are that good. I’ve ranted for years about everybody and his brother putting valid trial drugs for melanoma up against dacarbazine (a drug known to have little effect). Something that I feel at this point in time is simply unconscionable!!! I digress, but you get the idea.

4. Your doc’s role. Your doctor, if they deserve the license under which they practice ABSOLUTELY SHOULD discuss trial options with their patients when appropriate and help find the one that will best suit their patient’s needs!!!!!!!!!!!! However, the odds of attaining this service with a local one is small. If things get beyond the treatment options they have available, they will usually just send the patient to a bigger facility that does more research – which I guess is okay. The patient can then meet with docs who are more specialized and who have access to clinical trials. NOW – THOSE docs should still be frank about how good a fit the trials at their institution may or MAY NOT be for the particular patient. Some are. Some aren’t. I’ve experienced both. Sadly, many will simply try to enroll you in what they are running, rather than telling you about trial options elsewhere that may actually be better for you. I can say with complete authority that Jeff Weber, currently of NYU is a straight shooter whom I have known to tell patients when appropriate, that what he has open currently is not really their best bet, BUT the trial being run by so-and-so would be a better option and hook them up with the other physician.

5. Trial navigators can be used to help patients find trials they need. I have mixed opinions on this one, because – like the docs – some do a really good job and some do not. Many institutions and foundations have this service. In fact, I believe MRF has some.

6. There is no advocate for the patient better than the patient – when you or a family member is able physically and mentally to do the task. Searching for trials is absolutely daunting. But, it is doable for many. This site: https://clinicaltrials.gov/ – is fairly navigable. You can put in your disease and stage. You can put in a particular drug if you like. You can put in “recruiting” as a filter. For instance – here is what it looks like for Stage IV melanoma, actively recruiting trials: https://clinicaltrials.gov/ct2/results?recrs=ab&cond=Stage+IV+Melanoma&term=&cntry=&state=&city=&dist= 198 trials are listed. You can click on each one. When you do that you will see the drugs used and the arms offered. You can google the drugs as needed to see what sort they are. Below that, inclusion and exclusion criteria are listed. Below that, all locations that the trial is being offered is noted – across the globe – not just the US. And often a trial coordinator is listed as well. I always tell folks – if you are at all interested in a trial – CALL!!!! Even if you are not certain you WANT to do it or if you actually qualify. It never hurts to ask questions and learn more. My husband found all the trial options I ever looked at via a trip to the institution at which they were offered including the Phase 1 nivo trial I participated in. HOWEVER, he found the nivo trial by calling regarding a different trial entirely!!!!

7. This may have been way more than you were interested in and your eyes may well have glazed over by now!!! I am still here because of my participation in my trial. I am certain of it. However, it is important to know what you are getting into. Hope this has been of some use.

yours, celeste

Hi John, this Melanoma Mike that you’ve heard of in round about topics like Melanoma, or cancer & such haha, nice to meet ya…So, your asking about Trials?, I see our sister Celeste gave you an awesome answer, Trials are Night & Day compared to regular FDA approved meds, Clinical Trials are a pain in the ass! & That’s putting it lightly, they stand a good chance of working don’t get me wrong but, try and use the top of the line immunotherapys FIRST, plus there’s other avenues to, radiation, surgery’s, TIL shots, Vaccines etc. assuming your eligible. There’s so many Trials for “Solid Tumors” (that’s what ours is in a nutshell) Trials may not nessesarilly name Melanoma as one or more cancers to be treated, look for the name Solid Tumors when looking, you’ll see that mostly, especially in phase 1, 1b trials. As trials progress to phase 2 then you’ll see Melanoma listed a lot more cuz actual data is avail on Mel.,.Haven’t you recently started Yervoy?….In your bio, unless you got the dates wrong, you stated in late November that your team will test for the B-RAF Gene, you where diagnosed in March? And their just now testing that? Just curious bro!….my doctor, Dr. Hamid seems to have taken the reigns in searching for the best trials, so YES, your doc & team should help you….