Hey Everyone!
I know there were a few people interested, so I decided to start my own thread about my Denver experience.
Sooo.... I made it to Denver on Tuesday to be screened for the Kalydeco trial. For those who don't know, it is the trial for residual function mutations. In order to get in, one must meet at least one of 3 criteria: A sweat test under 80, pancreatic sufficient, or diagnosed at or after age 12. We know that I don't meet the second two, so I can only get in if my sweat test comes in at or under 80. It was 83 when I was diagnosed in 1981, so it's really questionable. However, they seemed optimistic that I would get in.
So my sweat test was an adventure in and of itself. After stimulating the sweat and waiting the 30 mins, I did not have enough sweat for the test! So the research coordinator told me she was going to have me run/walk a few laps in the hall to sweat more. I honestly thought she was kidding and laughed. Nope, she was serious as a heart attack. So off I went. Upon returning, I had sweat a little more, but still not enough. She collected that sweat, and then had to do a second test to try to get more. This time, she and the doctor had me go outside and make a lap around the hospital in the 90 degree heat. Still didn't sweat much, but in the end she thinks it is probably enough for them to test. If we went through all that only to find out it's not enough sweat to be tested I will crap! My feet are blistered and sore from jogging in sandals! Who would of known I needed tennis shoes and a sports bra for the screening!!! LOL.
The trial has already be underway for a while, in fact, some participants are already at or nearing the end. I'm not sure the reason, but they decided to open it up to a handful more participants. This trial is more involved than any trials I have done in the past. I do PFTs and pulse oximetry at home every morning and every night. The results are sent to Vertex through a smart phone supplied by them. I also wear a pedometer type thing 24 hours a day. Interesting stuff. So they've already got me started with those things, even though we don't yet know whether or not I am in.
Well, I guess that's about it! I will update you all as to whether or not I get in!
Take Care,
Autumn 32 w/CF
PS. A few points in case anyone is interested... my residual function mutation is S945L. Also, I have mentioned in the past that I am mostly pancreatic sufficient, so you may be wondering why I say that we know I don't get in on that criteria. Well, my fecal elastase number is very low, which does not reflect my need for few to no enzymes. They think that the low elastase numbers are a result of having pancreatitis many times. They said that for whatever reason they are seeing low numbers in people who have had pancreatitis. Interesting.
I know there were a few people interested, so I decided to start my own thread about my Denver experience.
Sooo.... I made it to Denver on Tuesday to be screened for the Kalydeco trial. For those who don't know, it is the trial for residual function mutations. In order to get in, one must meet at least one of 3 criteria: A sweat test under 80, pancreatic sufficient, or diagnosed at or after age 12. We know that I don't meet the second two, so I can only get in if my sweat test comes in at or under 80. It was 83 when I was diagnosed in 1981, so it's really questionable. However, they seemed optimistic that I would get in.
So my sweat test was an adventure in and of itself.
After stimulating the sweat and waiting the 30 mins, I did not have enough sweat for the test! So the research coordinator told me she was going to have me run/walk a few laps in the hall to sweat more. I honestly thought she was kidding and laughed. Nope, she was serious as a heart attack. So off I went. Upon returning, I had sweat a little more, but still not enough. She collected that sweat, and then had to do a second test to try to get more. This time, she and the doctor had me go outside and make a lap around the hospital in the 90 degree heat. Still didn't sweat much, but in the end she thinks it is probably enough for them to test. If we went through all that only to find out it's not enough sweat to be tested I will crap! My feet are blistered and sore from jogging in sandals! Who would of known I needed tennis shoes and a sports bra for the screening!!! LOL. The trial has already be underway for a while, in fact, some participants are already at or nearing the end. I'm not sure the reason, but they decided to open it up to a handful more participants. This trial is more involved than any trials I have done in the past. I do PFTs and pulse oximetry at home every morning and every night. The results are sent to Vertex through a smart phone supplied by them. I also wear a pedometer type thing 24 hours a day. Interesting stuff. So they've already got me started with those things, even though we don't yet know whether or not I am in.
Well, I guess that's about it! I will update you all as to whether or not I get in!
Take Care,
Autumn 32 w/CF
PS. A few points in case anyone is interested... my residual function mutation is S945L. Also, I have mentioned in the past that I am mostly pancreatic sufficient, so you may be wondering why I say that we know I don't get in on that criteria. Well, my fecal elastase number is very low, which does not reflect my need for few to no enzymes. They think that the low elastase numbers are a result of having pancreatitis many times. They said that for whatever reason they are seeing low numbers in people who have had pancreatitis. Interesting.