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Hopefully I'm right about # of patients. But I think that's right. Very exciting! Just not sure if it will help with insurance appeals because it's not a published journal article. But I think some of these abstracts from NACFC are published as supplements in professional journals.

Aboveallislove, Thanks for the response. I don’t have a lot of time to respond right now. I did want to say that my doctor, along with some other doctors in the area have submitted an abstract (? not sure if this is the right terminology) to be presented at NACFC this October, discussing their...

There is another study that is not currently enrolling, but should be soon. Your son's mutation is included (missense mutation). But you have to be 12 or older to participate. Here's the thread that discusses...

N-of-1 studies make the most sense given the fact that there are 1900 mutations, the vast majority of these being extremely rare. To complicate matters even more, there is great variability between people who share the same mutation combination. Genetic modifiers and environment, not just...

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...

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)...

Kenna2, I’m not sure how severe your liver/lung involvement is and don’t mean to question your doctor’s decision; but I did want to make you aware of recommendations for those with hepatic impairment: Dosage Adjustment for Patients With Hepatic Impairment1 • No dose adjustment is necessary for...

Aboveallislove, no worries- you were clear and I’m in agreement with what you said :) Believe me, I know what an advocate you have been on this forum, doing everything possible to keep people informed and up to date. I was just trying to brainstorm with you about off-label use. I didn’t want...

Aboveallislove- I agree that we need some ideas on expanding off-label use when it's appropriate. The first hurdle is increasing awareness of patients and doctors that off-label use is possible and may be very effective for certain mutations. Just because your mutation is listed under...

Stephen- Thanks for the link. There are 3 arms of the trial: placebo, monotherapy with Ivacaftor, VX-661/ivacaftor combination. I guess they’re trying to determine if combo drug has even more of a benefit than Ivacaftor alone for residual function. Based on Denver proof-of -concept study...

https://tools.cff.org/Display/dsp_ClinicalResearchHTML.cfm?id=406&IDH=9176856&State=&advancedSearchOn=true&ageshow=&status=&FEV1=&PSEUDOMONAS=&BCepacia=&PLT=0&Therapy=&Char=&Keyword=661 See link clinicaltrial.gov site for all participating sites...

On CFF website. Study not enrolling yet, no participating sites listed yet. See below for list of mutations that are eligible. Study Name: Vertex 661 and ivacaftor (Vx-661-107) in people with cystic fibrosis ages 12 and older with one copy of the F508del-CFTR mutation Study Type...

nocode- Just thought of something. I'm not sure what your second mutation is. I'm assuming it DF508?

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...

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...

Sorry I haven’t responded sooner. Just seeing this now. Yes, I continue to do well on Kalydeco. Progress did plateau quickly for me. But in my eyes, stability is progress considering I felt like my health has been faltering for the last 3+ years. The most noticeable differences are decreased...

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."

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...

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...

If you have had an elevated sweat test of 84 and 90, then you should have been diagnosed with CF. Sweat test gives the definitive diagnosis, not genetic testing. From CFF website: "The sweat test has been the “gold standard” for diagnosing cystic fibrosis (CF) for more than 50 years. When it is...