I’m sure many people have been waiting to hear about what it was like being on the Vertex (Orkambi) drug trial. I haven’t been able to post about it due to the strict regulations that were put forth by the trial. Now that I have been officially terminated from the study, I can finally post about my experience!
In 2012, the FDA approved a drug called Kalydeco. It is a pill that corrects the mutation that causes Cystic Fibrosis. However, it only worked for a mutation of CF that accounts for just 4-5% of the CF population. For the 4-5% of CFers, it has been a life-saver… literally. CF patients with the proper CF mutation who were taking Kalydeco reported the successes; weight gain, increased lung function, and simply better and healthier lives. Sounds amazing, right?! Well it is! So the next goal is to take this success and make it possible for the double delta-f508 mutants, the most common CF mutation, to have their “miracle drug” too. That’s exactly what Vertex aimed to do with their study. They took the Ivacaftor drug (Kalydeco) and paired it with another drug called Lumacaftor. The addition of Lumacaftor was what was supposed to make it work for the CF patients with two copies of delta-f508 mutations.
In 2013, I was lucky enough to be able to participate in the Vertex drug trial. When I first enrolled in the study, it was in phase two. This meant that I was either receiving the high dose of the medication, the low dose, or the placebo. This phase lasted for a year. I don’t think that I was on the placebo during phase two of the study because I did notice some minor changes. I was feeling pretty drained (which I just thought was stress for the longest time) and my GI issues, which were never very bad to begin with, were virtually gone. I had a much easier time keeping on weight, even gaining some (thanks to prednisone and lot and lots of puppy chow!), and didn’t get any more pesky stomach aches from forgetting my enzymes. After a year, the study was successful enough to enter into the third phase. This was really exciting because that meant that the study was having success. I chose to roll over to the third phase, which meant there was no chance of a placebo anymore. Everyone was either on the low dose or the high dose of the medication. As the study was coming to an end, we got news that the FDA approved the drug and it was named Orkambi. So I wrapped up on the study and had to decide if I was going to transition to taking Orkambi. I chose just to be terminated from the study and not continue on taking Orkambi. So I had officially had my last dose of the Vertex study drug on September 28, 2015.
What was it like taking the Vertex study drug?
Well, truthfully, it was kind of a hassle. The pills were pretty big, which didn’t phase me because us CFers are absolute pros at popping pills. However, the study visits were often months apart and that meant getting a dozen giant blister packs. There were so many packs and they were so large that they barely fit in a double-bagged grocery bag. Even then they would still rip holes in the grocery bags. Not only did I have to transport these from my appointments, I had to keep track of them the whole time in between my appointments because I had to bring them back to be accounted for. So in the morning I had to take 5 giant pink pills and 4 pink/blue pills at night. They were so big and there were so many that all of my pills barely fit in my weekly pill container! Along with the pills I had to eat a small snack because the medication used the fat from the snack to absorb the medication into my system (or something like that.. sorry, I’m not a doctor and they tell you so much information it’s hard to keep track of the specifics). Luckily, when Orkambi was approved they were able to turn the 4-5 pill dose into a 2 pill dose.
What were the study appointments like?
LONG and FULL of paperwork. When I was enrolled in the study there were pages and pages of consent and information forms that we had to go through and sign. There was all the paperwork that allowed me to get paid. Every time I was admitted into the hospital, it had to be reported within 24 hours and was accompanied by more paperwork. Yay paperwork! For the actual appointments, I would arrive to Children’s early in the morning, usually around 9am. Keep in mind I have a 2 hour drive there, so they were kind in not making it any earlier than 9am when possible. First I would turn in my dozen giant blister packs, and then the research coordinator and the pharmacist would document any missed doses. While they were doing this, I would fill out a survey of how I’ve been feeling and other generic questions. Then all the testing would start, including an EKG, height, weight, and PFTs. After all the testing was done, I would get a visit from the nurse practitioner to do a check-up. Then I would have to pee in a cup because no babies are allowed to participate in this study! The last step would be eating some snacks and taking my morning dose of the study drug. All in all, the appointments would take anywhere from 2-6 hours, depending on exactly how much testing had to be done. So… longggg day.
What was it like being in a drug trial?
It was really exciting to be a part of such a huge step in medicine, but really it wasn’t all that eventful. As I said earlier, it didn’t really do a whole lot for me besides help with some minor GI issues. It certainly was not a “miracle drug”. It was mostly just like being prescribed yet another drug, but one that had a lot of restrictions. One of the most challenging restrictions was that I wasn’t allowed to know what my PFTs are. They didn’t want my PFT results to affect whether I thought I was getting the drug or not. Unfortunately, I got so sick one summer that they allowed me to start seeing my results at my regular clinic check-ups; just not during my study appointments. I also wasn’t allowed to talk about the study or post anything on social media, which is why I haven’t written a post about it until now. The whole study was very hush-hush, probably because it was supposed to be such a big deal. I’m really glad that I had the opportunity to participate, and I’m glad that I was one of the “guinea pigs” that helped bring this drug to the people that needed it.
What has it been like since stopping Vertex?
The first thing I noticed since stopping taking the Vertex drug was how much energy I had. I had spent the last two and half years feeling too lazy to get off the couch and spend time with friends. I just chalked it up to me getting sicker and stress and having to get used to that. However, within days after I took my last dose of the Vertex drug, I had a significant increase in my energy level. I happened to be in the hospital and I felt like I was bouncing off the walls. It was the end of my stay, so that was to be expected, but it was more than that; it was also when I remembered how I thought that maybe I had lost energy after starting drug. I couldn’t wait to get out of the hospital and start doing things again. I didn’t have to be stuck on the couch all the time and stay home missing out on things and friends. The other major change is the stomach aches. I think that I got lazy about taking my enzymes while I was on the study drug because my digestion was so much better. I was forgetting to take enzymes with my little snacks because I wasn’t having any consequences from forgetting. I wasn’t getting stomach aches or losing weight. Now that I’ve been off the study drug, I have to retrain myself to remember those little details. It’s been a little set back, but nothing I can’t conquer. It’s also pretty nice to have all that space in my pill container back!
I am really excited to participate in more studies and help more people (hopefully myself included) find better ways to manage this horrible disease. If there are any more questions please feel free to contact me; the study was two and half years long so there is no way I got to cover my whole experience in this little post.
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