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Thread: Kalydeco and Class IV Mutations

  1. #131
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    Hey jricci,
    I am so sorry. I hope that isn't accurate and she is just misinformed. That really stinks. :-(. But if it is true, remember there is another arm just for heteros who aren't suppose to benefit from Kalydeco. I'd think if they don't list your mutation for residual function then you would fit that other arm.
    Also, I'm sorry if you've said in other posts, but have your tried to get off label? PM me if you want more info on that. Hugs and prayers,
    Love

  2. #132
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    Quote Originally Posted by lifeisgood729 View Post
    From Facebook this morning:

    Our clinic is going to be doing the 661- Ivacaftor study for residual function, starting this summer. At Stanford / LPCH in California. Woohoo!
    Got some paperwork on the trial today. Interesting lay out, it's a 32 week study. 4 wks pre study drug screening, 8 weeks on drug, 8 wks washout. 8 wks on drug 4 wk post study screening.
    There are 6 different senario, 8 weeks on 8 weeks off 8 wks on a different combo.
    These are the sequences you will randomly be place in one.
    1. 8 wks 661w/ Ivacaftor (washout 8wks) 8 wks ivacaftor
    2. 8 wks Ivacaftor (washout 8 wks). 8 wks 661/ Ivacaftor
    3. 8 wks 661/Ivacaftor (washout 8 wks) 8 wks placebo
    4. 8 wks Placebo (washout 8 wks) 8 wks 661/Ivacaftor
    5. 8 wks Ivacaftor (washout 8 wks) 8 wks placebo
    6. 8 wks placebo (washout 8 wks) 8 wks Ivacaftor.
    Depending on the results, if they are good there will be an extension for everyone on drugs

    Thanks Martha! Looks a lot like Denver. Except Denver was 4 weeks on placebo or drug, then 4 week washout, then 4 weeks on placebo or drug etc... I think it was 26 weeks. There was of course, no 661 though..

  3. #133
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    Quote Originally Posted by jricci View Post
    I found out some information, but still don't know how "residual function" will be defined. According to one of the research nurses that is involved with this study, it will be difficult to get into this particular study if your CF center is not participating because the enrollment is limited to 100 people in the United States. The trial says they are enrolling 300, but this is worldwide. I have not seen any of this in writing. This is just as per a conversation I had with a research nurse. Centers will start filling spots with those eligible from their center and if spots are still available, they will accept people from other centers. Vertex will compensate for travel, according to the person I spoke with.
    Given this information, I would call as many centers that are feasible for you to travel to and give them your information, including medical records (i.e. your mutation, evidence of pancreatic function, sweat chloride value), so that they will contact you if their clinicís enrollment is not complete.

    Also, I found out that those who culture Mycobacterium, Burkholderia cepacia, or Burkholderia dolosa will be excluded.
    I'll post as I find out more.
    Wow, that's a bummer for me to hear that it will probably be hard to get into. I've contacted Houston and Dallas, the only other feasible center would be Tyler so maybe I'll contact them to.

    It's also a bummer to hear they are going straight off the residual function list. I, like others, had hoped it would be a bit more broad. I am lucky in that I still qualify based on my mutation, but bummed for others.

    Well, thanks for the info and the update!

    Autumn

  4. #134
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    Quote Originally Posted by Aboveallislove View Post
    Hey jricci,
    I am so sorry. I hope that isn't accurate and she is just misinformed. That really stinks. :-(. But if it is true, remember there is another arm just for heteros who aren't suppose to benefit from Kalydeco. I'd think if they don't list your mutation for residual function then you would fit that other arm.
    Also, I'm sorry if you've said in other posts, but have your tried to get off label? PM me if you want more info on that. Hugs and prayers,
    Love
    Thanks aboveallislove,
    I am one of the lucky ones that is receiving it off-label. Kalydeco alone has done wonders. I wasn't trying to get into the 661 study at this time. I'm just so upset for the other 163 people that share my mutation because once the study is done and it is officially approved (hopefully!)., my mutation wouldn't be included as one of the approved mutations.

  5. #135
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    Oh, I'm so glad you are, but I agree...it is horrible for those who can't get! I can't keep everyone straight, but is your doctor the one doing the case study? That could help them. Also, if they all tried to get in the "hetero" arm (non residual mutation arm), they could really scew the results favorably since they will all be benefiting from your mutation and could get it available for all heteros! (because I really think the mutation is only part of it and as Jenny's pictures show, it helps those even it wasn't "suppose" to help!!). As an aside, are you going to try to get 809/combo when it's approved since 809 and 661 work the same way?

  6. #136
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    Quote Originally Posted by Aboveallislove View Post
    Oh, I'm so glad you are, but I agree...it is horrible for those who can't get! I can't keep everyone straight, but is your doctor the one doing the case study? That could help them. Also, if they all tried to get in the "hetero" arm (non residual mutation arm), they could really scew the results favorably since they will all be benefiting from your mutation and could get it available for all heteros! (because I really think the mutation is only part of it and as Jenny's pictures show, it helps those even it wasn't "suppose" to help!!). As an aside, are you going to try to get 809/combo when it's approved since 809 and 661 work the same way?
    Sorry, you lost me on the 661 study. What arm are you talking about? Is it a current study or one that is expected soon?
    To answer your other question, Iíll probably just stay with the Kalydeco. I still have this fear that my insurance covered this inadvertently since there were no questions asked, no appeals necessary. I literally feel nauseas whenever thereís a letter from my insurance company addressed to me. Iím so worried that they are telling me that thereís been an oversight and it should have never been approved. I wouldnít want to bring it to their attention by trying to get VX-809 off label (when it does get approved for ddf508). Iím so thankful for Kalydeco, I don't want to press my luck.

  7. #137
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    Sorry. There are eventually going to be 4 arms. There will be one arm that is for heterozygotes with df508 and another mutation not know to be benefited by Kalydeco. So, to me, it would seem they would qualify any df508 heterozygotes who don't qualify for the residual function arm criteria. And if those who likely would benefit from Kalydeco "qualify" and enroll that could get better results overall and maybe help make the labeling such that all heterozygotes with df508 qualify. Hope that makes more sense.

    Totally understand re the under the radar. I'd do the same!!!!

    Quote Originally Posted by jricci View Post
    Sorry, you lost me on the 661 study. What arm are you talking about? Is it a current study or one that is expected soon?
    To answer your other question, Iíll probably just stay with the Kalydeco. I still have this fear that my insurance covered this inadvertently since there were no questions asked, no appeals necessary. I literally feel nauseas whenever thereís a letter from my insurance company addressed to me. Iím so worried that they are telling me that thereís been an oversight and it should have never been approved. I wouldnít want to bring it to their attention by trying to get VX-809 off label (when it does get approved for ddf508). Iím so thankful for Kalydeco, I don't want to press my luck.

  8. #138
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    I received some more information re: 661 study (108) for residual function https://clinicaltrials.gov/ct2/show/NCT02392234 I spoke with the study nurse coordinator from Tucson AZ CF center (pediatric and adult). She was very helpful and said that although she canít share the mutations that are listed as inclusive for the study, if someone would like to find out if their mutation is listed, you can contact her and she will answer your question directly. The Tucson AZ center and the Tampa FL center are the only 2 centers that are currently accepting participants for this particular study. She said that the general guideline that Vertex has set is that each participating center can accept 3 patients. Because the Tucson center is one of only 2 centers currently enrolling, they have been granted permission to accept more participants beyond the 3 originally stated and they are currently accepting inquires from those patients from other centers. She said that Vertex will be activating 12 more centers in the US sometime in May. She also said that there is an upcoming VX-661 study (109) that will be broader in their inclusion. https://clinicaltrials.gov/ct2/show/NCT02412111
    This isnít real clear to me because it looks like it will be for those patients with gating mutations, but maybe Iím missing something. She also said that Vertexís reimbursement for travel for the 661 studies would be very limited because the need for participants isnít an issue. She thought that they would reimburse patients up to $250.00. Iím not sure if this is a total for the entire span of the study or per study visit. So if anyone is interested in this 661 study for residual function (108 ) and is from the Tucson area and/or is willing to travel there, I would encourage you to contact her. Her contact information (from CFF website) is Molina de Rodriguez: [email protected]
    Her phone number is (520) 891-6767 (also listed from the CFF website) Please note that this phone number is her work cell number so please take the time zone difference into account when you call her.

  9. #139
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    Thanks so much for posting! I'm wondering if she means the more general heterozygote study I mention above. There will be 4 studies in total. This summarizes the different studies. One is the residual function and one the gating ones and then ddf508 and then a heterozygote df508.
    http://investors.vrtx.com/releasedet...leaseID=902790

  10. #140
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    Thank you so much for sharing the information about the Class IV. I am a big fan of this and i am so glad that i finally found it here. You must get college essay writing service which helps to complete your exam. I will also share it with my friends.

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