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Thread: residual function & Kalydeco news

  1. #1
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    residual function & Kalydeco news

    Hi all,
    Does anyone has any news on the pilot study of Kalydeco and residual function mutations? On clinicaltrials.gov website it says completed and has results, but i have trouble deciphering the actual results. Maybe anyone had beteter luck?

    thanks

  2. #2
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    Here's Vertex's press release: http://investors.vrtx.com/releasedet...leaseID=852372
    It does work for proof of concept but rather than go to Phase 3 with Kalydeco, Vertex is doing a Phase 3 with VX661 & Kalydeco which another study shows works better for those that Kalydeco works for. That trials is currently enrolling and if you are residual function you might try to get in that:
    https://clinicaltrials.gov/ct2/show/...m=vx661&rank=1

  3. #3
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    Oh, thanks, I guess i missed the results of this. so basically they ar not going to do anything specific for residual function anymore from what i gather. My dd has one copy of delta 508 but she is too young. thanks for answering

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    They aren't doing anything for residual function for kalydeco alone but 661 and kalydeco together will be the on label for residual function. If she's two you might try for kalydeco alone off label if her other is residual function...otherwise you'll have to wait for the 661 study approval likely about two years.

  5. #5
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    Interesting information. My children have one residual function mutation and I too have been waiting the outcome of this study. I was not aware that the residual function study was "suspended" and wrapped in with the larger study involving 661. The press release issued following the proof of concept study specifically stated "Vertex plans to initiate a Phase 3 study of ivacaftor in people with residual function mutations, pending discussions with regulatory authorities regarding the design of the study." That being said, where is it stated that there won't be a separate phase III study? Is it simply because there has been no enrollment yet?

    Thanks!

  6. #6
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    This is the Phase 3 study they were referring to in the press release.
    https://clinicaltrials.gov/ct2/show/NCT02392234 They just added the combo drug to one of the arms. I think people are getting confused because there are 3 arms of the trial: placebo, monotherapy with Ivacaftor (Kalydeco), VX-661/ivacaftor combination. They’re trying to determine if combo drug has even more of a benefit than Ivacaftor (Kalydeco) alone for residual function.
    Also note that as of July 8th, clinicaltrials.gov site has been updated and they added more study sites that are actively recruiting. If you have a residual function mutation (pancreatic sufficient OR sweat chloride between 60-80) that is not listed in this study please post. I'm wondering what other mutations (besides mine) are "missing". Thanks


  7. #7
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    Jricci,
    thanks! I totally forgot about the mono arm!

  8. #8
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    Just realized that clinicaltrials.gov site still doesn't include which mutations. If anyone missed previous thread, you must have one df508 mutation and one of the following mutations to participate in the study:

    2789+5G?A, D110E, R352Q, A1067T, 3849+10kbC?T, D110H, A455E, R1070Q,
    3272-26A?G, R117C, D579G, R1070W, 711+3A?G, E193K, S945L, F1074L, E56K,
    L206W, S977F, D1152H, P67L, P205S, F1052V, D1270N, R74W, R347H, K1060T

    https://tools.cff.org/Display/dsp_Cl...r=&Keyword=661

    If you have a residual function mutation (pancreatic sufficient OR sweat chloride between 60-80) that is not listed in this study please post. I'm wondering what other mutations (besides mine) are "missing". Thanks

  9. #9
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    Quote Originally Posted by jricci View Post
    Just realized that clinicaltrials.gov site still doesn't include which mutations. If anyone missed previous thread, the study is inclusive of the following mutations:

    2789+5G?A, D110E, R352Q, A1067T, 3849+10kbC?T, D110H, A455E, R1070Q,
    3272-26A?G, R117C, D579G, R1070W, 711+3A?G, E193K, S945L, F1074L, E56K,
    L206W, S977F, D1152H, P67L, P205S, F1052V, D1270N, R74W, R347H, K1060T

    https://tools.cff.org/Display/dsp_Cl...r=&Keyword=661

    If you have a residual function mutation (pancreatic sufficient OR sweat chloride between 60-80) that is not listed in this study please post. I'm wondering what other mutations (besides mine) are "missing". Thanks

    If they included specific mutations they would have to mention it, right? I would suppose their inclusion parameters are clearly stated and they include every mutation accordingly. My son has R347P which is was not mentioned to respond in the in vitro studies. In real life, my son (11 years) is clearly pancreas sufficient.

  10. #10
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    If he'll be 12 in time, there is another hetero arm that he could try to get in. Or, if he is PS, then discussing off label with his doctor would make sense too.

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