Kalydeco With "Other" Mutations

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stephen

Guest
I would strongly urge anyone with the D1152H (Class 4) mutation to try to get Kalydeco! It has done wonders for my pulmonary problems thank G-d. (I’ve never had any digestive issues.)


This is my seventh month on Kalydeco. To my initial astonishment, I noticed dramatic changes less than a day after starting it. Within a week my coughing stopped and the mucus disappeared. Because of my mutations, it took several weeks for me to become convinced that the improvements were really due to the Kalydeco.

Kalydeco can definitely work for people with Class 4 and 3 mutations that are not on the “list”.
 

Aboveallislove

Super Moderator
Dear Stephen, thank you for sharing. While this doesn't help our son who is ddf508, I know of others like you were and often say try to get it. The battle is sometimes with the insurances companies, and one thing that helps is published literature. Might I suggest that you ask your doctor to do a case study and attempt to have it published in a peer reviewed journal, because that will help others also get it off label if the insurance is fighting...as well as to educate other doctors as I've also heard of doctors hesitant to try off label. May God continue to bless you.
 
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Elc

Guest
Hi Stephen, thank you for your post. My son (only 20 months old) has d1152h and g85e. I have been following kalydeco for residual function mutations as much as possible. So glad to hear that it is helping you! Take care, Emily
 

ladybird

New member
I asked my doctor about getting Kalydeco for my mutation - R117C and deltaF508 and she said she would look into compassionate use, they will discuss this at next appointment. Not sure what that means but I am hopeful. I really want to get better so I can get off disability and go back to work.
 

MOM247

New member
Question: Do they have the results from the Kayldeco trial done in Denver?

My son has a mutation on the list (1898+1G>A). I'm very curious on what they found. If you know a link to results, could you please share. Thanks!
 

cam

New member
Im so glad people with certain mutations getting results but really need one for the 508 im praying for it so much hope everyone gets better and stays healthy
 
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stephen

Guest
The data in the trial Elc references above appears to be based solely on changes in FEV1.


While I do understand the importance of having measurable criteria in evaluating a study, there should be a way to include the effects of Kalydeco on:


  • Chronic cough. (I went form coughing at least 100 times a day to ZERO!)
  • Chronic mucus production and color. (It went from numerous tea spoons full of green mucus a day to ZERO!)
  • Increase in energy and general good feeling.
  • Drop in resting pulse.
  • Reduction in chronic fever.
  • Reduction in lung inflammation.
  • Reduced need for antibiotics.

These are more than just Quality of Life criteria. They are indicative of the presence, or lack of, infection and progressive lung damage.


It’s hard to understand why more CF doctors are not pro-active in trying to get Kalydeco for their patients who have “other” class 3 and 4 mutations, but are instead waiting for the results of more time consuming studies.

I’m sure there are others like me. As I’ve said before, I saw the effects of Kalydeco in less than a day. Within a week, I was a different person. It took a while before I was convinced the Kalydeco was responsible for my dramatic improvements.
 

Aboveallislove

Super Moderator
Vertex is starting a trial with Kalydeco and 661 (which is like 809 but better), in 2015 for those with a residual function among other things.
 
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lifeisgood729

Guest
Vertex is starting a trial with Kalydeco and 661 (which is like 809 but better), in 2015 for those with a residual function among other things.


Aboveallislove, that is great news! Do you have any further info? I have a residual function mutation and have had good results with Kalydeco alone. I know of several others with the same mutation who are having similar results. I am hopeful that Vertex can move things along and find a way to get Kalydeco approved for the residual function mutations soon.

Martha
46 w/CF
 

Aboveallislove

Super Moderator
hey Martha,
Vertex's press release at vrtx.com summarizes the plans for 661. A few additional points I gleaned from its conference at the NACFC: 1) It sounds like the residual function will have two arms--one with Kalydeco alone and one with Kalydeco plus 661. 2) It sounds like it will be placebo controlled (although not sure with the gating mutation arms), so you wouldn't want to be in it since you'd have to give up Kalydeco and could get placeabo. 3) they are already talking with FDA on study design and it will be 2015 but sounds like they want interim on phase 2b for 661/Kalydeco combo, which is fully enrolled; 4) they are moving very quickly on this and said it won't hold up 809/770 approval so try to get on that when that's approved; 5) they have high hopes based on 661 having no interaction with Kalydeco, Denver results, phase 2 results of 661 and how 809 results panned out showing that short time frame gives indicator. I think there's a few other points but can't remember now and need to play with ds. I'll add if I remember.
 

triples15

Super Moderator
Good to see you around Martha! I'm so hopeful they find a way to add the residual functions to the label soon as well! I never did convince my doctor to write me the prescription off label, even with all the evidence you helped me with. UGH.

Love, thanks for that info. I had read all the press releases etc,. but it sounds like you have more info. So you think the residual function study is going to be one study with two arms? With their wording I was curious as to how that was going to be done. I had assumed two separate studies. I was also confused how they could do a phase 3 of Kalydeco with 661 with the residual functions if a phase 2 hadn't been completed yet?

I've got my fingers crossed that by some stretch of a miracle I could get into the phase 3. But only assuming that the criteria will be the same as the phase 2, and I won't be eligible, even tho I have a residual function mutation that shows it responds VERY well to Kalydeco alone. Ugh again.

Anyway, thanks so much for the info, and let us know if you think of anything else.

Autumn 33 w/cf
 

Aboveallislove

Super Moderator
Phase 2b with 661 is fully enrolled and they likely already have some preliminary results which to me is why they are pushing forward. But they said that they are finalizing study based on fda studies and results of phase 2b (which I bet is dosing mainly). There will be several "arms" of the study, so I think one study technically, but one arm for df508 homozygotes, one for df508 heterozygotes, one for gating, and one for residual function. But they mentioned that residual arm would test both kalydeco solo and combo. I gleaned some of the added detail from the investor conference which you can listen to on line, go to vrtx.com and click on investor events and listen to the last conference. Keep in mind, though, that this is for investors, so CFers sound like $Fer$--just gotta put blinders on to that to get to the nity gritty good stuff! And the details are sprinkled throughout; in fact the very last couple sentences in the full hour, where I got the placebo controlled from, as I was thinking they'd have to do it with 809 combo versus 661, but it sounds like they are planning on starting so quickly that it won't be a problem with enrollment because it could come before FDA approval or approval in Europe, so folks will be willing to do the study even if the option is placebo. Now, I'd think gating has to be different because those folks will already be approved and on Kalydeco. But I don't think they can do that for those who have it off label so they would have to be placebo.
 
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