STX209 – Phase III Trial

This…this could be the backpack of any young boy…

This, however, could not.

Not normal.

I am grateful for the chance to be here, looking at a box of medicine that may or may not change our lives. It’s still a bitter pill to swallow…and I’m not even the one taking it.

Oh, the Places You’ll Go!
by Dr. Seuss

You will come to a place where the streets are not marked.
Some windows are lighted. But mostly they’re darked.
A place you could sprain both your elbow and chin!
Do you dare to stay out? Do you dare to go in?
How much can you lose? How much can you win?

And IF you go in, should you turn left or right…
or right-and-three-quarters? Or, maybe, not quite?
Or go around back and sneak in from behind?
Simple it’s not, I’m afraid you will find,
for a mind-maker-upper to make up his mind.

You can get so confused
that you’ll start in to race
down long wiggled roads at a break-necking pace
and grind on for miles across weirdish wild space,
headed, I fear, toward a most useless place.
The Waiting Place…

 

Today, my sweet boy, after 6 1/2 years…we finally leave that most useless of places. The place where we could do nothing but hope and wish and dream. Today, we are off on a grand adventure and I’m so honored to be on it with you and your Daddy.

On Friday, Eric took Caleb on the final screening visit for the Phase III STX209 drug trial. We did the phone screening ages ago, September, 2011 to be precise, and I wrote about it at that time (STX209 – You’ve heard of it, right?). That was when we discovered that, in order to do the study, the Celexa would have to go. There are no SSRIs allowed during the study.

We started talking about taking him off the Celexa then…and we continued to talk about it and drag our feet and gnash our teeth until August of this year when finally the stars aligned. The doctors and study people were all ready. Eric was ready. It was just up to me to say, “Let’s do this.”

It wasn’t easy. There was one moment, the night before we started the washout period in August, when Caleb looked at me with such love and trust that I very nearly pulled the plug on the whole idea. How was I going to take away something that I knew was helping him for a chance that we might get something better. I spent a long, sleepless night crying and finally decided to go with what has gotten me through all of the hardest steps on this journey...hope.

Prior to this visit, we had received a long e-mail from the study coordinator so we knew what to expect, we just hadn’t expected Eric to have to do it alone. It broke my heart to miss this, it was so unfair to leave the two of them to go this alone but it couldn’t be helped.

They began by reviewing the study and signing the consent form to go forward with the study. After that there were more forms. Of course there were more forms!! They included a basic demographic form and medical release forms so the study doctors and coordinator could contact and share information with Caleb’s doctors. Eric watched a short video explaining the main questionnaire used throughout the study, the Aberrant Behavior Scale – Community (ABC-C), and then he filled out the actual form. He wrapped it all up by completing the Visual Analog Scale questionnaire.

Then he met with the pediatrician, she reviewed the study criteria, his medical and surgical history, current medications and did a physical examination with Caleb. One of the things she did was an ASD screening. What Eric and I found interesting is that Caleb only met two of the criteria. Interesting since C has a PDD-NOS diagnosis. It’s interesting how many different opinions we have had on this over the years. 1. He doesn’t have it but it’s good for extra services, 2. No, he does not have ASD, 3. Yes, he has ASD, it’s on his IEP, right? and 4. He doesn’t have ASD. OH, wait, that’s only 2 opinions. The doctors (1, 2 and 4) and the school (3) ((Yes, this IS a sore spot with the school, even his super awesome we love them so much school!)).

They ended the day with urinalysis, EKG and bloodwork. Yes, the EKG and bloodwork sucked. I am glad I wasn’t there for these…everyone should be glad I wasn’t there for these. Mama Bear comes out to rip heads off e.v.e.r.y.t.i.m.e…and these smart and helpful people need their heads to keep doing the good work they’re doing. Even though it involves torturing small children…mostly my small child. Grrr.

I am sorry I missed the urinalysis though, it was the first time anyone has asked Caleb to pee in a cup. Eric got to hold the tiny cup. I really, really wish I could have been there taking photos of Eric as he realized Caleb didn’t know how to stop mid-stream to avoid an overflow. Those would have been frameworthy!

I was aware that there was a chance that Caleb wouldn’t be affected enough by his anxiety to qualify. When I asked Eric about this he said there wasn’t even a question that he qualified. Um, yay? I’m glad he qualified, don’t get me wrong, but it just sucks to know how obviously he struggles. It’s not something we think about daily so when we have to face it, it hurts.

The entire visit took 3 hours. Yeah, THREE hours. There was one slight hitch during the visit that added about a 1/2 hour…Caleb is study participant number 9 for this location. This may not seem like a big deal but it is a tremendously exciting milestone. You see, this is a small study location and historically they have struggled to find enough participants to even make being a study site worthwhile. In fact, it’s been so difficult that they believed that they would only be able to find 6 – 8 participants. Obviously, if Caleb is #9, they surpassed that goal which meant they had to get approval from the internal review board to change the study parameters. Permission was given earlier in the week but a slight mix-up resulted in the wrong paperwork being stamped. It took some time to clear up but they did it.

That night Eric fell asleep an hour before Caleb’s bedtime and didn’t move, except to pull the covers over himself, until the next morning. It was emotionally and physically draining. Caleb, on the other hand, was fine after he got his reward…a footlong meatball sub from Subway ((Subway! Subway! Eat fresh!)). We thought he would eat half of it at dinner and have half for lunch the following day. Saturday at lunch he at the 2nd half of my meatball sub because he finished his entire sandwich the night before. Holy moly!

So, what lies ahead for us on this new adventure?

• Visit 1 – Start medication, complete the Vineland Assessment & behavior forms  ~2.5hrs
• Visit 2 – Labs (bloodwork) & behavior forms  ~1hr
• Visit 3 – Labs (urinalysis & bloodwork) & behavior forms  ~1.5 hrs
• Visit 4 – Labs (urinalysis, bloodwork & EKG), Vineland Assessment & behavior forms  ~2.5 hrs
• Visit 5 – Visit 5 is short if we decide to not enter the open label extension study.  Otherwise it will also be Visit 1 of the open label (treatment with arbaclofen for 2 years, visits average every 3 months)  ~2.5hrs

I think the only way we don’t do the the Open Label extension is if we have to stop this study before it is completed. We haven’t really questioned that part, it was the potential of getting the placebo that we struggled with.

Stay tuned…

 

When we decided to take Monkey off Celexa, hoping that he might qualify for the STX 209 trial, you might remember I was a little apprehensive. OK, maybe more than a “little.”

Hope, it isn’t always pretty.

I’ll be totally honest, I’m still waiting for the $hit to hit the fan but so far, so good ((And, yes, I did probably just curse us! Thank you for noticing and please send wine. Lots and LOTS of wine. Just in case.)).

We’ve tried to continue pushing Monkey a bit, while also keeping in mind that he’s dealing with a cwap load more anxiety than he has been for the last 18+ months, and he continues to impress us. We took him to the zoo this past weekend with his Grammy & Grampy Welin and he did awesome.

The Franklin Park Zoo is pretty large and open and it’s familiar so it was a good choice but it was exceptionally crowded for a few reasons:

1. It was a beautiful sunny day.
2. It was a beautiful sunny SATURDAY.
3. The 39th Caribbean Festival was taking place (literally THOUSANDS of attendees, loud music (the bass could be heard very clearly at all times) and very slow traffic/weird traffic patterns.)

There were moments when he was less than thrilled but it was a typical toddler who won the award for going into “Bat$hit Crazy Meltdown” mode ((She was tiny and she had both parents struggling and racing for an exit. They could not even physically get her into her stroller. I felt really bad for them and really, REALLY glad it was not my kid!)).

He walked through a “jungle” looking at animatronic dinosaurs (moving and randomly making loud roars) very, very happily…he was enthralled, even. He visited just about every exhibit in the zoo. He played on the ridiculously huge and very crowded playground. He even went into the Tropical Forest exhibit which is inside, dark, humid and a little stinky which he never does ((We did bribe him with Ms but still, you canNOT bribe a child with FX into doing anything if they are struggling. Cold hard truth right there that so many people do not grasp.))!

Overall it was a great day and he ended his day by coming home and cuddling on the couch with Grampy. Does it get any better? Grampy, for sure, would say no 😉

The real test, most likely, will be school. Which starts next Tuesday…which also just happens to be his first day with no Celexa at all. Perfect planning, eh?

Last night, as I was getting Monkey ready for bed, he looked me right in the eye and his face lit up. It was one of those perfect moments when I just knew that he loves and trusts me implicitly. Naturally, I burst into tears and after putting him down for the night I spent hours crying. Why?

Today, after much agonizing, we are starting C’s Celexa washout period. And so it begins…3 weeks of gradually reduced anxiety medication, 4 weeks of no anxiety medication and then we see if we qualify for the STX209 trial. There is no guarantee that he will qualify. If he doesn’t, that is 7 weeks of heightened anxiety for no benefit to anyone. If he does qualify, it will mean 4 1/2 months of … well … we don’t know. Maybe placebo, maybe partial doses of the study drug, maybe a full dose.

Why are we doing this? Hope. Hope that this will help him more than the Celexa has. Hope that this will help a whole lot of other kids someday. Sometimes hope is a beautiful thing and sometimes hope is swollen eyes and an aching heart. Sometimes hope is a desperate decision to just do it, even when you aren’t sure you want to.

Right now the only thing I am sure of is that I am so fucking tired of having to make decisions like this.

If you want to read more details about what is involved in the STX209 trial, read this post from last September:

STX209 – You’ve heard of it, right?

 

We here at Chez Monkey are very excited that there will be a nearby clinic for Seaside Therapeutics’ Phase III STX209 trial. We have spoken to the researcher at UMASS in Worcester and he’s given us the OK to share with you what he told us in our screening call. Duhdee and I are still discussing our participation, that’s a separate post though.

The screening call took about 30 minutes, it could take longer if you have more questions or if your child has more health concerns, Monkey is a healthy boy so we skimmed past most of those pretty quickly.

This first call is to establish preliminary eligibility. If you make it past this call, you will go to the study site for additional screening. So, what are they looking for in this first call?

1. Your contact info.
2. What language your child speaks and/or understands.
3. If the fragile X syndrome diagnosis is confirmed. (If yes, they will want a copy of the report. If no, they can do the testing as part of the screening process.)
4. What medications your child is currently on. There are some that are not being allowed, SSRIs are a biggie ((This is where Duhdee and I stumbled.)). Don’t assume you won’t be able to work around this though, the study Dr’s are willing to talk to your child’s prescribing physician if you are willing.
5. If your child is on a medication that is not allowed, and you and your child’s doctor decide to remove that medication…and this is VERY IMPORTANT…YOU MUST TALK TO YOUR CHILD’S DR. MUST! MUST! MUST! The period your child needs to be off the medication will vary depending on what it is. Generally, between 4 and 8 weeks.
6. What educational interventions your child receives, if any, and when that started (PT, OT, ST, Special Education, etc.)
7. What behavioral interventions your child receives, if any.
8. What dietary interventions your child receives, if any.
9. How your child does with blood draws, there are 3 during the study period.
10. If your child ever participated in a clinical trial and how that went.
11. Any questions or concerns you might have about any of what you’d discussed.

If you make it through the screening call you will have to go to the study site for additional screening. The additional screening will involve…

1. A physical exam.
2. A medical history.
3. A psychiatric history.
4. An EKG. He said it was a simple EKG, non-invasive, and only takes a couple minutes ((He said it was not painful, I could not help myself and replied that Monkey has a different definition of painful than he does, lol.)).
5. Filling out behavioral questionnaires ((Of course, do any of us ever go to any appointment and NOT have to do this??)).
6. A blood draw. He assured us the nurses they use are very nice and very patient.

So…you made it all the way through and you are enrolled in the clinical trial. Now what?

First, there are two age groups being included in this trial at the UMASS Worcester site. Ages 12-25, which is, as of August 30, underway and ages 5-11, which should be getting underway in the next two weeks. They have the trial drug on hand and they’ve cleared their ethics requirements, it’s just down to the nitty, gritty, administrative stuff.

The trial will last about 4 1/2 months. During that time there will be 6 study visits to the Worcester location and 11 phone calls with one of the Dr’s. The phone calls will be every 4 days for the first two weeks then dropping down to once a week and eventually once every two weeks. The visits will vary in length, the phone calls should be 20 minutes or less. So, there is a time commitment required here and you will have to stay on their visit schedule.

In the ages 5-11 group, called the HARBOR-C trial, there will be 4 dosing groups:

1. placebo
2. 5 mg dose
3. 10 mg dose
4. 15 mg dose

That’s a 1 in 4 chance you will not get the study drug. Once the trial is complete, however, you will be given the option of receiving the full dose of the study drug until the FDA approves the drug for marketing.

In the ages 12-25 group, called the HARBOR-A trial, there will just 2 dosing group:

1. placebo
2. dose adjusted by doctor during a titration period

As with the HARBOR-C trial, once completed you will be given the option of receiving the full dose of the study drug.

That is about it. It’s not an easy process but it wasn’t terribly painful either (so far, at least.) Duhdee and I are still talking it over and will be sending a follow up e-mail with some additional questions. There is room for 6-8 patients in each HARBOR trial, they’ve had a fair amount of interest but the slots are not yet full so I encourage you to make the call and talk it over.

Michael Hill is stepping aside, the new contact is below!

You can reach Mike Kara at:

Michael Hill Kara Manchester
Research Coordinator
University of Massachusetts Medical School
Biotech I, Suite 100
365 Plantation St. Worcester, MA 01605
Phone: 508-856-2041 508-856-2789
Fax: 508-856-8211
Research Line: 508-856-5896
Email: Michael.Hill@umassmed.edu Kara.Manchester@umassmed.edu

You can find out more information about the Worcester research group at http://www.umassmed.edu/psychiatry/candi/index.aspx.

Check out ClinicalTrials.gov for more information too. If you aren’t local to us, there are 19 other study locations, check here for a list: Clinical Trial Sites.

And, I don’t really think this needs to be said but, Duhdee and I have no affiliation with Seaside Therapeutics. We are vocal supporters but we are not, in any way, compensated by them or anyone else for talking about this process. We encourage you to reach out to them and think about it but we still may very well come down on the “No, thanks” side ourselves. If we do, though, it will be a pretty big disappointment for us…we really want to do this but it’s all about Monkey’s best interests. Obviously.