New Study for Ivacaftor/lumicaftor

jricci

Super Moderator
PROSPECT study

Just received an e-mail from CFF notifying me of the following study:

PROSPECT
This trial aims to identify biomarkers that show CFTR function and disease progression. It will also look at the effect of ivacaftor/lumicaftor on those who may be prescribed treatment. It is for people ages 12 and older.

https://clinicaltrials.gov/ct2/show/NCT02477319

I am sooo happy to see that the CFF (not just Vertex) is sponsoring studies to help others gain access to ivacaftor/lumicaftor. Inclusion/exclusion criteria is a little different than other Vertex sponsored studies. There is one cohort that will include anyone with a class IV/V mutation (residual function mutation). My mutations are r334w and df508. r334w is a class IV that did not show significant response in vitro. I'm receiving Kalydeco off label and have no doubt that it has slowed my progression. But because of in vitro data, my mutation isn't included in most recent Vertex 661 study. So this study gives me hope that they are willing to look beyond what happens in a lab when looking at who will potentially benefit.
I’m not sure of open label extension when study is over since this is a CFF sponsored study, not Vertex. I’ll call CFF and post if I find out anything.
 

Aboveallislove

Super Moderator
That's awesome and I too am so glad the CFF is funding additional studies that the pharmas aren't and hope they continue to do so!!
Also, for those with Class 1 or other (non df508) Class 2, there is a cohort for those with 2 copies of Class 1/2 mutations so that is a great opportunity for those who might not have an option to try off label with that category!
 

jricci

Super Moderator
Ok- I may have jumped the gun with my excitement. As I'm looking closer, it looks like this is just an observational study (not interventional- no study drug) for those in the first 3 cohorts??? I'm a little confused. Aboveallislove take a look and let me know what you think. At least it looks like they are looking to eventually expand label and studies like this would be the first step.
 

Aboveallislove

Super Moderator
Ugh, I think you are right...just watching a d trying to figure out genotype,sc, phenotype relation ship....but I think you're right ths might be first step to try more broad.y. Why the cff doesn't fund some n of 1 studies fr all the misc. one off mutations is beyond me. I'd think they could do without p,acebo arm and get so e even e for who it works for and doesn't within a short time period and I can't imagine vertex wouldn't provide the drugs free if the cff funded the study component since then they'd be able to have proof it works..
 

jricci

Super Moderator
Agreed. With over 1800 different mutations, I think n-of-1 studies will be the only way to make sure "no mutation is left behind."
 

nocode

New member
Jricci, I am one of the few people who also has the r334w mutation!
I was very disappointed when I found out that it didn't show much benefit in vitro with kalydeco but I also believe that the result could be different if tried on people. Do your doctors have an explanation for the slowing down of your disease progression? Do they believe the kalydeco is helping you?

How did you get it off label? I live in Europe so it's not too relevant for me but would still like to be able to fight for it over here.
 

jricci

Super Moderator
I’m not sure if you also have been following the r334w thread on this forum, but I go into some more detail about my Kalydeco experience on some post on that thread.
http://forum.cysticfibrosis.com/threads/40943-R334w/page2
My insurance covered it, no questions asked! It was prescribed and arrived on my doorstep a few days later. I still can’t believe it at times-miraculous really. I am so very grateful. Because of my unexpected response, I feel very obligated to let others know of my success story. I do hope my experience will help someone at some point. I read through some of your older posts and it looks like you could really benefit from this drug. With a lung functions in the 30’s, I’m guessing your quality of life is impacted by CF on a daily basis and you don’t have the luxury of time to wait as these studies are being done.
I’m not familiar with the steps you would have to take to get it off-label in Europe. I would talk to your clinic about it. Maybe you would qualify for a compassionate use program since your FEV is below 40%. Tell your clinic about my experience. I am very willing to share my medical records if that would help in any way. There are also some journal articles that may be useful to help you make a case for yourself. I will share them with you if you would like. Are you pancreatic sufficient? If you are, this will make your case much stronger as you have proof of residual function. An “n- of- 1” study would make so much sense for you. If they would agree to somehow get you the drug for a month trial, you will know within that month if it is working. If you are like me, you will know in a matter of days! Talk to your clinic and please keep me updated. You can also PM me if you would like.
 

jricci

Super Moderator
Jricci, I am one of the few people who also has the r334w mutation!
I was very disappointed when I found out that it didn't show much benefit in vitro with kalydeco but I also believe that the result could be different if tried on people. Do your doctors have an explanation for the slowing down of your disease progression? Do they believe the kalydeco is helping you?

How did you get it off label? I live in Europe so it's not too relevant for me but would still like to be able to fight for it over here.

Nocode,
I also wanted to mention the possible option of participating in a study. After you complete the study (6 months), you will get the drug because there is an open label extension - at least that’s how the latest Vertex studies have worked. I’m assuming Vertex will continue to do this with all future studies. I just checked and the residual function VTX 661-108 study and it does list the Netherlands as having study sites. https://clinicaltrials.gov/ct2/show/study/NCT02392234?show_locs=Y#locn
Although this particular study won’t be helpful to you because our mutation isn’t included, I’m thinking that the VTX 661-107 study will have sites in the Netherlands as well. “This trial is for people ages 12 years and older with one copy of the F508del-CFTR mutation and a second CFTR Mutation that is not likely to respond to therapy.” http://www.cff.org/Display/dsp_ClinicalResearchHTML.cfm?id=405&CTSubId=90
I was sent this info. from CFF yesterday, but there is no info. on clincaltrials.gov site yet; so I’m not sure of study locations.
What is your FEV1? The inclusion criteria says you must be 40% or greater in order to participate. Although, when I was getting screened for a Vertex study I was told that they do sometimes allow for exceptions if you are below 40% .Your doctor/study coordinator would have to call Vertex and ask.
 

jricci

Super Moderator
Nocode,
I also wanted to mention the possible option of participating in a study. After you complete the study (6 months), you will get the drug because there is an open label extension - at least that’s how the latest Vertex studies have worked. I’m assuming Vertex will continue to do this with all future studies. I just checked and the residual function VTX 661-108 study and it does list the Netherlands as having study sites. https://clinicaltrials.gov/ct2/show/study/NCT02392234?show_locs=Y#locn
Although this particular study won’t be helpful to you because our mutation isn’t included, I’m thinking that the VTX 661-107 study will have sites in the Netherlands as well. “This trial is for people ages 12 years and older with one copy of the F508del-CFTR mutation and a second CFTR Mutation that is not likely to respond to therapy.” http://www.cff.org/Display/dsp_ClinicalResearchHTML.cfm?id=405&CTSubId=90
I was sent this info. from CFF yesterday, but there is no info. on clincaltrials.gov site yet; so I’m not sure of study locations.
What is your FEV1? The inclusion criteria says you must be 40% or greater in order to participate. Although, when I was getting screened for a Vertex study I was told that they do sometimes allow for exceptions if you are below 40% .Your doctor/study coordinator would have to call Vertex and ask.
nocode-
Just thought of something. I'm not sure what your second mutation is. I'm assuming it DF508?
 

nocode

New member
My second mutation is N1303K. The studies want someone with D508F so I wouldn't be able to participate.

My fev1 is 30% and has been between 30% - 37% for the past 8 years.

I have an appointment tomorrow so I'll talk to the doctor a bit more about this.
Thanks for all your help.

Is it possible that you have improved and stabilised with Kalydeco because it is actually acting on your D508F mutation, and not on the R334w?
 
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