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Thread: Tezacaftor/Ivacaftor

  1. #1
    Junior Member
    Join Date
    Feb 2017
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    4

    Question Tezacaftor/Ivacaftor

    I recently participated in a double blind clinical drug trial for the medication Tezacaftor/Ivacaftor. The trial ended and I opted to take part in the extended access program and have been taking the actually drug for about a week now. I was wondering if anyone else was taking this drug? If so, what kind of results are you seeing?

    Thanks in advance!
    Lacie

  2. #2
    I was desperate to get my daughter onto one of those clinical trials. I was not successful.
    I’d love to know how you are doing. Are you a heterozygous DF508? You can private message me if you prefer. I’m just very hopeful that the tezacaftor can help my daughter.
    Thanks
    Michelle

  3. #3
    Junior Member
    Join Date
    Feb 2017
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    4
    Hi ToriMom!
    This drug has been a game changer for me. I am a heterozygous DF508. I tried Orkambi when it was new on the market and did not tolerate it at all. It was awful. When my physician offered this trial to me I knew I should try! I knew as soon as a couple days into the trial that I had to have received the drug and not the placebo. After being accepted into the Extended Access Program, it confirmed my initial thought. The biggest difference I have experienced is that I just, over all, feel good. I hadn't felt good in as long as I could remember. My PFTs have always been good; between 77% and 90% depending on whether or not I was fighting a bug, but have progressively been declining. The declines were between .25 and .50 percent every 6 months or so. Between the trial and starting the EAP, I have been on the medication going on 6 months. There has not been any decline in my PFTs, even with coming down the the flu (Influenza B) and common cold. The Influenza did not lead to Pneumonia, when before it most likely would have. I had a mile stone today- I ran about half a mile without feeling like I could keel over. I was so excited. With the medication I have noticed weight gain. Unlike a lot of CFers, I am already slightly overweight so that hasn't been too much welcomed by me. I am learning I need to watch what I eat, when before I was able to eat anything!

    I hope your daughter can get on this medication and has great results! Feel free to reach out to me if you have any questions!

  4. #4
    Thank you ...I’m so glad you responded! It gives me some hope that you are doing well. I really need to believe there is hope for my daughter and her future.
    Thanks again!
    Michelle

  5. #5
    Junior Member
    Join Date
    Jan 2018
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    1

    Bright future

    Hi Michelle,
    I just joined this blog today and saw your post. I just took part in new phase 2 study of a drug combination tailored to DF508 heterozygous and I assume I was not on the placebo because the results were phenomenal. I'm 59yrs 43% fev1 and my score jumped into the mid 50s. My lungs were so clear and my breathing was so easy and full. I was hiking and fly fishing with renewed vigor not seen in a very long time. I believe the future is bright for people diagnosed with CF. I hope this good news finds your daughter well. Be patient and diligent with her care because the new combinations will hopefully be available as early as 2019. We live in miraculous times. Be well, Billy

  6. #6
    Billy,
    Thank you so much for your encouraging reply. It feels like every day since her diagnosis I've lived in fear of losing her, and now...some days...I can have some hope for more time.
    Best wishes,
    Michelle

  7. #7
    jamest
    Guest
    This has been approved by the FDA! https://www.reuters.com/article/vert...-idUSL2N1Q21GD

    It's called Smydeko.

  8. #8
    Junior Member
    Join Date
    Feb 2018
    Posts
    1
    Hello, new to the forum community.I took part in the triple combo phase 2 in june 2017. Just a 2 week study but my base line fev of 72% jumped to 90% after one week. Felt amazing energy off the charts. Eagerly awaiting phase 3 in coming months. Fev down to 70% currently. 43 year male. Heterogeneous. Married 20 years . 2 children adopted.
    Great things to come, even though difficult at times we all have to stay postive and keep on keeping on.

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