About to start Vertex trial-quetion for those that have been through Kalydeco trial

[Irishgal]

Hey everyone?
I am about to start a vertex-661 trail.
My question is: For anyone that has been enrolled in any of the vertex studies and have RECEIVED the drug, what happens when the trial is over and you stop taking the drug? does your lung function drop/you go back to feeling how you did before receiving the MED, etc?
For those that did the Kalydeco study and have GG51D mutation, once the Kalydeco study was done & it became FDA approved for public use, and you tarting taking it again, did it effect you just as well as it did while you were on it during the study? For example, are you feeling as good as you did while enrolled in the study? Are you having the same results as far as lung function, less exacerbations, etc?
Thank you for any input you can give.

 

[hammerpocket]

As Rachie mentioned, after the trial is over you should be able to stay on the drug while waiting for FDA approval.

BTW, the Vertex stuff doesn't work for everyone. I was excited to be in a trial earlier this year, but ended up having to stop taking the drug because it was actually harming my lung function. Good luck to you!

 

[Irishgal]

Thanks guys. I did not know about the roll-over option, so I'll be sure to ask. "Hammerpocket," I'm sorry to hear that you did worse-you had a vertex drug, not a placebo, is that right?

 

[CFcarebear]

Once my husband started the Kalydeco trial in 2011, he never got off of the med. He was able to continue it all the way through being approved by the FDA. Also, FYI, he just received a double lung transplant in May and he's still taking Kalydeco!

 

[brad.johns]

If I am not mistaken, 661 has not made it as far as the 809 trial. I believe it is only in phase 2. Open label or being able to stay on the drug after you finish the study only occurs after phase 3. I do not believe you will be able to stay on the drug after the trial. You should ask the doctor to be sure though.

 

[hammerpocket]

"Hammerpocket," I'm sorry to hear that you did worse-you had a vertex drug, not a placebo, is that right?

The first part of the trial was randomized with placebo. I didn't notice any change during that time and I hoped (because of the lack of improvement) I was being given placebo. When I rolled over into the next part of the study, I was given known study drug. I noticed a negative effect on my breathing immediately -- within hours of the first "real" dose, but I had heard that people taking Kalydeco often feel worse right after starting it, so that didn't seem alarming. It was only after we saw that my lung function was continuing to decline for a few weeks that the study doctor and I agreed I should stop.

 

[CFParent2]

Exactly the same happened to me, it made me so tight i went from 45% to 35% was so disappointed. I have had to discontinue, some people don't get this side effect but it was just too much for me.

Interesting, I am guessing that there is a lower limit of lung function where the initial shock to the system of releasing all of the build up in the lungs is too much for the body to take. I wonder if this is not just a dosing issue. I don't know if changing the dosage would lessen the intial shock to the system or is it an all or nothing fix? Unfortunately, a drug regiment cannot be taylored to the patient during a drug study. That said maybe your doctor could do off label dosing not part of the study since it is over.