First Visit to Duke

Well, here we go.  The first real post about the study at Duke!

First of all, a disclaimer.  Anything that I post is written from my perspective as a parent.  These are my observations and feelings, nothing is official from the people running the study.  I am doing this blog because I want to share these events with people who have an interest in Ian and in Fragile X research.  I am not endorsing the study in any way, though I hope this may help other people who are considering participation as other blogs helped me.

The official name of the study is "A Randomized, Double-Blind, Placebo-Controlled, Fixed Dose Study of the Efficacy, Safety and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Withdrawal in Children with Fragile X Syndrome."  Phew, that's a mouthful!

Not only is it a mouthful, but this is a big deal.  When it is done, this study will have had 200 participants at 30 locations around North America.  I found out yesterday that Ian is probably one of the last ones to be seen at Duke.  This is funded by Seaside Therapeutics which discovered the drug, and they are paying Duke (and the other locations) to conduct the study.

I first heard about this from other parents through one of the Fragile X (FX for short) Facebook pages.  Last year we explored doing this at Kennedy Krieger in Baltimore, but for various reasons it didn't work out.  Then I saw that Duke was doing it and tried again.  Duke is much easier for us since my sister Mary lives in nearby Chapel Hill and I'm very familiar with the area.  I was also impressed with the people I spoke with at Duke; overall, they seemed much more organized than the people in Baltimore.

Participation in this is quite a commitment of time and energy.  We have 6 visits between yesterday and May 21st and there will be 7 scheduled phone calls.  Since I do not have a job at the moment, this was the perfect time to do this since the visits are scheduled for Tuesdays.  Durham is about a 2 and 1/2 hour drive from Richmond, so we are planning to drive down on the Mondays preceding the appointment and stay with Mary and Jim, then return home after the appointment on Tuesdays.  This way Ian only has to miss one day of school.

So, our first trip......

We left on Monday after Ian got home from school around 3.  I had spent the morning getting the car packed and trying to anticipate every need that the little man might have.  Charging the all-important iPad, packing snacks, making sure we have things like his sound machine so he can have his "beach" for going to sleep.  The good thing is, I took notes and next time will be much easier and faster!

I am lucky because Ian is a good traveler.  We have gone on some real adventures, and he can mostly roll with it.  He gets in his seat, puts on the head phones and gets the iPad on and he is set.  I put his snacks and juice boxes in a bag on the back seat and he could help himself.  We drove straight through, and he was a champ.

At Mary and Jim's he ate dinner (chicken and fries from Wendy's) and then took off with the iPad to our room.  He went to bed early and slept well.  I got to have a beautiful dinner and a glass of wine and spend some time with my sister....and watch the Downton Abbey episode I missed on Sunday.  Perfect!

On Tuesday morning, we got up around 7.  Ian ate breakfast, watched PBS Kids and then we got dressed and packed up to go.  Our appointment was for 10:30, so we left Chapel Hill at 10.  Our instructions were to use Valet Parking, but when we got there that was full so we had to shift gears and go to the parking garage.  Ian did fine, even with the elevator.  We met Deby, who is one of the coordinators, and she took us to the clinic.  Ian had some problems going through the doors the first time, but otherwise he was good.

We met Wan, the clinical research coordinator, and a few of the nurses.  The first hour or so was going over the consent forms and getting more information.  Deby had warned me that Wan was very detailed about this, but I understand.  There is a lot to explain and understand.  I had received a copy attached to an email so I had read it before, but I get why they go over it.

After that I had to watch a video about filling out a questionnaire called the ABC.  I could almost swear I've done this one before.  It's basically where you compare your child to typically developing peers.  This is a crucial step because if your child does not have "issues" there is no need to be there, right?  Well, I can see where it would be hard, but I have been dealing with this for 11 years and I've also worked with children Ian's age in a school setting so I have no illusions about his "differentness."  After this, I met a psychologist  and answered her questions.  Then we had lunch, which the study paid for.

Through all this, Ian was glued to either the iPad, or the DVD player they had brought in.  Here he is after lunch.

 Well, then the real "fun" began.  It started with blood pressure.  Ian fought the cuff, but we got it done.  Then his temperature was taken.  He fought the thermometer going in his arm pit, be we got it done.  He was weighed and his height was taken.  Not too bad.  Okay, then it was time for an ECG to make sure his heart was okay.  Oh boy.  They wanted him to lay down on the bed.  Oh no, he hates doing that.  We finally got it done.  Phew.

Dr. L. came in and did a brief physical.  Even this was met with a lot of resistance.  I felt so bad for him, but we had to get it done and we did.  The worst part of the day, no surprise, was doing the blood draw.  A wonderful nurse named Stephanie came and did it, but we had to do it twice because the vein collapsed in the first arm.  But, we got it done.  Probably the easiest thing we did all day was the urine collection!

And then, mercifully, we were done.  We said our goodbyes and were told to return on March 5th.  We were in!  We walked to the parking garage and saw that it was a beautiful day.  We got in the car and headed for home.  As we were crossing Lake Gaston and arriving back in Virginia, we had the most gorgeous sunset to end the day.  We stopped for gas, and kept going.  Even when the iPad died, Ian was a good boy.  He was exhausted and fell into bed when we got home.  I can't remember when I've been prouder of him.