First, the semantics.
While I have called what I am doing a "clinical trial," it is actually an "investigational study." The difference is that in an investigational study, the doctors and researchers are still very much trying to understand whether or not and how a certain drug works in the treatment of an illness or condition.
In a clinical trial, doctors already believe the drug they are studying is effective in the treatment of an illness or condition, and they trial the drug with a larger group of subjects (and, at least in most cases, controls), to test for efficacy across a bigger group.
Clinical trial is an easy short-hand for me, and for those who know us...but for those who are following along because they or someone they care about is living with Celiac Disease, I want to be very clear. In my mind, I have equated it with that which precedes a Phase 1 clinical trial. At this point, the doctors are just looking to see if there might be value in this intervention.
The purpose of the study is to evaluate the safety and effectiveness of Montelukast (more commonly known as Singulair) when given to people with celiac disease who are allowed to eat gluten."
The information in the consent form is very clear. Montelukast controls the symptoms of asthma and allergic rhinitis, but is not a cure. That said, preliminary research shows that Montelukast blocks the development of the immune cell that stimulates intestinal damage.
Investigational studies tend to be quite small. In my case, there are twelve subjects.
Clinical trials tend to be slightly larger than investigational studies. In Susan's case, since she was in a Phase 1 clinical trial (think next step after an investigational study), the group of subjects was still small, but not quite that small. Susan was one of nine subjects at one of four study sites.
In the case of the investigational study, 2 out of every 3 subjects will receive the study drug. In this case, that means that eight subjects will receive Montelukast while four will receive placebo. All of the subjects will be asked to consume six grams of gluten (in the form of bread) for six weeks.
Unlike the clinical trial that Susan participated in, those subjects who receive placebo will not eventually receive the "study drug."
There is also no promise of any sort of long-term betterment of the quality of one's life.
There is NO reason to believe that I will continue eating gluten in any form once this clinical trial is over.
No.
Gluten is not really in my future.
(at least not in my immediate future...)
"If you agree to take part in this study, there may or may not be direct medical benefit to you. We hope the information learned from this study will benefit other individuals with celiac disease in the future."
This clinical trial is short...if I count from Day 0, which isn't actually the first day of anything (but it is meaningful in clinical trial land), it will last eight weeks.
Today is Day 0.
But how did I get to Day 0?
Well, first there was a (very thorough and careful) Phone Screening -- a phone interview in which I am certain the clinical trial coordinator assessed commitment. I am sure she liked what she heard when I talked about understanding -- based on first-hand experience -- what being the recipient of the placebo in a clinical trial might look and fee like. I am equally certain she liked what she heard when I shared our level of commitment to Susan's clinical trial, PRROTECT, in which she attempted to tolerate ever-increasing doses of peanut without the protection of Xolair, the study drug.
I suspect she did not hear the ambivalence I felt -- the little but very strong voice inside my head that persistently and insistently reminded me that I might not be a good fit for this clinical trial.
Once I "passed' the Phone Screening, there was Screening Visit #1, which involved nearly two hours in the car to the University of Chicago Medical Center one late August morning. It was the the first week all three of my children were back in school, and the kind of day I might have told myself I would work and yet found myself taking the dogs for an extra long walk along the beach...but instead, I sat in traffic. Once at the University of Chicago Medical Center, I underwent a comprehensive medical exam and had a number of tubes of blood drawn.
And then I consented to a pregnancy test...despite my assurances that given the efforts we went to to have our three children and the passage of time since then that it is highly unlikely that we will find ourselves pregnant again.
And then I completed several questionnaires designed to gather information about my adherence to my gluten-free diet (Dietary Adherence Questionnaire) and an index of celiac disease symptoms.
And then I drove home, to wait while the blood work was done, the results examined.
A week later, I got the confirmation e-mail: I had been deemed "healthy enough" to proceed. While a clean bill of health is always reassuring, there was a part of me that very much anticipated (for no real reason, I assure) that I would not be deemed "healthy enough."
And then came Screening Visit #2, which was definitely more invasive. It began with a stool sample that I collected at home and turned in the day of my second screening visit. Screening Visit #2 included a comprehensive medical examination, another pregnancy test, and than the pre-study upper endoscopy (a procedure before which the nurse asked about Living Wills and Advanced Directives -- standard protocol, I know, but still).
The doctor, my husband, and I talked before the upper endoscopy. She explained what I already knew -- that they would sedate me and then examine my esophagus, my stomach and the first part of my small intestines -- the duodenum. She further explained that she would take twelve biopsies of my small intestines.
The doctor did not say anything about how the upper endoscopy would be done, but I had vague memories of swallowing a black snake-like tube during the first upper endoscopy I underwent as part of the original diagnostic process in 2011. When I commented that I remembered having to swallow a black snake-like tube, she paused and said very clearly that that was not something I should remember.
She assured me I would not remember it this time, except that I have this memory of fighting the black snake trying to make its way down my throat. I am hopeful that by the time I undergo the second endoscopy -- the one required toward the end of this study -- that I will have greater confidence in the process and not fight that black tube as it snakes its way down my throat.
The doctor also explained that they would take twelve separate biopsies, and she assured me that I would be fine. She added that they screen their candidates very carefully, looking only for those whose Celiac Disease appears to be very well controlled.
That gave me pause.
I am very careful, but I have accidentally been "glutened."
I remember full well the night in February when I ate the most incredible pizza -- that I believed to be gluten free. A few hours later, I was fairly certain the pizza had been incredible because it was most decidedly not gluten free. (Either that or it was the worst case of food poisoning known to man.)
The doctor and my husband talked after the upper endoscopy (I was really, really sleepy after). She assured him that "things looked fine," but was also very clear that I could not be enrolled in the clinical trial until results of the biopsies came back. The doctor told us she should have results in a week.
A week passed.
Then a week and a day passed.
I found myself checking the University of Chicago patient portal, in case I somehow missed the e-mail notification that the results were available.
And then I wondered if maybe these -- like some of the results in Susan's clinical trial -- would never be for my eyes.
A week and TWO days passed.
The study coordinator e-mailed me to tell me not to worry -- the biopsy results were not back yet. I responded gratefully, for I had been worrying. That's just my nature.
A week and THREE days passed.
And then the e-mail came from the study coordinator.
I was cleared for enrollment in the study (after all that!). She wanted to schedule Day 0, the gluten challenge.
We e-mailed back and forth a bit about my availability -- I needed a day where I could be at the University of Chicago Medical Center by 10:30 am, and I needed to be sure I could clear myself of any responsibilities for the remainder of the day -- just in case.
We set Day 0 as Tuesday, September 13, 2016.
It is incredible to me all that came before Day 0.
Day 0 is a big day -- it is the gluten challenge. As with Screening Visit #2, there were things to be done at home prior to the visit, although easier than collecting a stool sample. I took my first dose of the "study drug" at home on the morning of Sunday, September 11, 2016. I took my second dose of the "study drug" at home on the morning of Monday, September 12, 2016.
I have been instructed to return the empty prescription bottle when I go to my Day 0 appointment, and it is already in my purse, just to be sure I remember it.
Prior to beginning the gluten challenge, I will undergo a complete medical examination and I will be given another dose of the "study drug." After a few minutes, I will consume somewhere between two and four slices of bread (dosage is determined by weight) that contain a total of exactly six grams of gluten.
And then I will wait.
After four hours, my blood will be drawn, and if I am unaffected, I will be free to go home.
I have been thinking about that bread. While I certainly miss being able to eat gluten at times, I do not miss sliced bread. The truth is, I never really even liked sliced bread, unless it was toasted and smeared with peanut butter...but due to Susan's peanut allergy, until very recently, we hadn't even had peanut butter in the house. The truth of the matter is that I had pretty much stopped eating sliced bread long before I was diagnosed with Celiac Disease.
After Day 0, Week 1 will begin. I will be expected to take the "study drug" every morning and to consume my gluten doses daily. It is my understanding that there is a lot of freedom in how one consumes the daily gluten dose, but not too much freedom. The daily gluten doses will be provided by the University of Chicago Medical Center. Each dose will be pre-measured, individually bagged and frozen.
Once each week for Weeks 1, 2 and 3, I will participate in a Phone Call Follow-Up in which I will complete the Dietary Adherence Questionnaire and answer questions regarding any symptoms I might be having. The investigational study protocol stresses repeatedly the importance of my reporting any symptoms I might have, even those I might deem unrelated to my consumption of gluten. I will also be asked to count how many doses of the study drug I have and how many gluten doses I have in order to help ensure study compliance.
In Week 4, I will have another visit to the University of Chicago Medical Center. I will be asked to return any unused study drug and any gluten doses I have not consumed, in order to ensure compliance.
I will undergo a comprehensive medical examination and I will be required to submit to a pregnancy test. (Should any subject become pregnant during the investigational study, she will have to withdraw. For some reason, I find this fascinating, and wonder if that is simply typical language or if there is a real concern about what might happen if a subject in this particular trial were to become pregnant.) I will have blood drawn.
I will complete a Dietary Adherence Questionnaire, answer questions about any symptoms I might be having and I will schedule the second upper endoscopy, which will be done during Week 6 of the investigational study.
Before I leave, I will receive a stool sample collection kit (I fully intend to put this particular brown bag away with far greater care than I did the first one -- for anyone who has ever done this, imagine my daughter's surprise when she opened it, intending to help me put the groceries away. I will also receive additional study drugs and enough doses of gluten for another two weeks.
In Week 5, I will participate in a Phone Call Follow-Up in which I will complete the Dietary Adherence Questionnaire and answer questions regarding any symptoms I might be having. The investigational study protocol stresses repeatedly the importance of my reporting any symptoms I might have, even those I might deem unrelated to my consumption of gluten. I will also be asked to count how many doses of the study drug I have and how many gluten doses I have in order to help ensure study compliance.
In Week 6, I will return to the University of Chicago Medical Center. I will submit my stool sample and I will be asked to return any unused study drug and any gluten doses I have not consumed, in order to ensure compliance.
I will undergo a comprehensive medical examination and submit to a blood draw before undergoing an upper endoscopy. I will be sedated and the doctor will examine my esophagus, my stomach and the first part of my small intestines -- the duodenum. Twelve biopsies of my small intestines will be taken.
I will complete a Dietary Adherence Questionnaire, answer questions about any symptoms I might be having and I will schedule the final study visit. This will mark the end of my eating gluten!
In Week 8, I will return to the University of Chicago Medical Center. I will undergo a comprehensive medical examination and submit to a blood draw. I will complete a Dietary Adherence Questionnaire and answer questions about any symptoms I might be having.
If I have been fully compliant, upon discharge from the clinical trial, I will receive a check for $160.00 (yes, I assure, the decimal IS in the right place!).
I am nervous, because I do not have to take the risks associated with this clinical trial (and they are laid out very clearly), but I have decided I am going to -- for Susan (who does not have Celiac Disease, but who forged her own brave course for the good of others), and for everyone in this world who has to think about absolutely everything they put in their mouth.
Some things are important to do just because someone needs to do them.
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