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Clinical Research Study Now Enrolling Participants with Celiac Disease

Dr. Dan LefflerThe National Foundation for Celiac Awareness (NFCA) is all about educating our community on clinical research opportunities.  After all, research is the only way to advance potential pharmaceutical treatments for celiac disease (or any condition) forward. 

To help get you all the details you need to make an informed decision about clinical research study participation, we talked to Dr. Dan Leffler, Director of Research for the Celiac Center at Beth Israel Deaconess Medical Center, NFCA Scientific/Medical Advisory Council Member and a principal investigator involved in the CeliAction Study.  We tossed him the most commonly asked questions about research study participation.

Skip to the first question or use the links below to select which answer you'd most like to read.  You can also visit the CeliAction Study website to learn more and to see if you qualify to participate.

For some celiac disease patients, it’s hard to understand why a pharmaceutical treatment is needed when we have the gluten-free diet. Many are hesitant to give up a treatment that, in theory, can be managed starting with a trip to the grocery store. Can you explain the need for a pharmaceutical treatment?

vast majority quote 2
For a minority of people with celiac disease, it is probably true that there is little to gain from a pharmaceutical therapy.  However, we know that the vast majority of people with celiac disease are not doing as well as they, or their doctors, would like. They have ongoing intestinal damage, ongoing symptoms and reduced quality of life associated with the burden of following the gluten-free diet. These are all areas that we hope a medication will improve.
 

Many adult celiac disease patients have ongoing intestinal damage despite being on the gluten-free diet. How much gluten does it take to trigger the autoimmune response in a person with celiac disease that can lead to intestinal damage? Can the CeliAction Study potentially help to address this problem?
two years, five years quote 2

Studies have shown that less than 50 mg of gluten per day, really just a few crumbs of bread, is enough to cause intestinal damage.  We used to think that the majority of patients had complete intestinal healing after a short time on a gluten-free diet. However, we now know that only one-third of patients heal their intestine after two years on a gluten-free diet and less than two-thirds are healed after five years.  Even in children, growing evidence suggests that around 25% still have ongoing intestinal damage, so it is clear that we have a great deal of room for improvement at all ages.   
 

NFCA knows that the community wants to know more about how this medication may help them better manage their gluten-free diets. Can you give an example of an everyday scenario where this medication might improve their lives?alv-003 quote 3


I think the most common scenario will be the person who is doing their best at a gluten-free diet but are still having ongoing celiac disease-related symptoms on a regular basis.  Their tTG may be normal or a bit elevated and yet, if they had a repeat endoscopy, there would be some mild or moderate intestinal damage.  A medication like ALV-003, if approved, would allow this type of person to improve their symptoms, speed up healing of the intestine while at the same time providing a "safety net" against accidental gluten contamination, such as what occurs regularly when dining out or eating processed foods.

Can you tell us about how a person would take this medication? If approved, would it be taken everyday or only in certain situations?

I think both are reasonable possibilities.  There are some people who are either highly sensitive to gluten or have diets that are difficult to fully control, for instance college students on a meal plan or frequent business travelers.  For these individuals, this type of a medication would be most effective if taken routinely.  On the other hand, there are people who can achieve adequate dietary control most of the time, but will need some extra protection on weekends when they eat out or on vacation, for example. 

We know that celiac disease affects people differently and some experience no symptoms at all when accidentally exposed to gluten. We want to learn more about the type of people that could participate in the CeliAction Study. What do study participants have in common with one another?

To be eligible to participate in the CeliAction study, individuals must be adults who have been diagnosed with celiac disease based on serology and/or biopsy and have been on a gluten-free diet for at least 11 months and have ongoing symptoms.  There are a few other criteria, but these are really the major ones.  I would encourage anyone who is interested, even if they are not sure they qualify, to visit the study website and call their local participating center. 

Can you briefly walk us through the clinical trial sign up process for the CeliAction Study? For example:

  • What happens after a person completes the online survey to see if they can participate?
  • How long will a person participate for?
  • How many visits will they make to the study center?
  1. If you complete the online survey and meet study criteria, your information will be forwarded to the study site closest to you.  The study investigators will then reach out to you to discuss the study in more detail and arrange a screening visit. 
     
  2. At the screening visit you will review the informed consent for the study and have any questions about the study answered by the study team.  If you agree to participate, blood work will be performed and you will be scheduled to come back in a few weeks. 
     
  3. At that next visit you will be instructed in the use of an automated telephone survey. You will complete this daily for four weeks and if you have sufficient symptoms, you will be scheduled for your third visit, when you will have an upper endoscopy with small intestinal biopsy for evaluation of celiac disease activity. 
     
  4. If you have both sufficient symptoms and ongoing celiac disease-related intestinal damage, you will be randomized to get the study drug or placebo which you will take for the next 12 weeks.
     
  5. At the end of the treatment phase, you will come back for a repeat upper endoscopy with a small intestinal biopsy for evaluation of celiac disease activity. 
     
  6. You will then have a final, seventh study visit a few weeks after the endoscopy. 

To summarize, the study overall lasts about 18 weeks, which includes 12 weeks of treatment and two upper endoscopies.  Additionally, there is an option to continue treatment for an additional 12 weeks and have a third upper endoscopy. However, this is completely optional and does not affect your ability to complete the initial part of the study as I’ve outlined above.

If a person does participate in the study, what happens after the trial is finished? For example, will they find out if they received the investigational drug or the placebo? Will they find out if the drug worked?

As is standard for clinical trials, neither you nor any of the study doctors will find out which treatment you received.  You will be informed of study-related results including your celiac disease blood test levels and your biopsy results, so you will know if you had any change in these tests, but not whether this was due to the drug being studied.  Once the study is completed, the information will be very carefully analyzed and eventually released for review and publication.  This process often takes a year or more to complete after the study is finished.  A good way to learn overall if the study supported the efficacy of the drug is to be in contact with your local celiac disease center. 

How does community participation help themselves and others with celiac disease?participation quote 4


It is important that anyone participating in this type of research study understands that there is no promise of clinical or therapeutic benefit. We hope that the types of drugs tested in these trials will be safe and effective, but in many cases we will not know that for years to come.  What we can say with certainty is that if people do not participate in clinical trials, we will be unable to develop and study new therapies for celiac disease or for any other disorder. If you care about celiac disease and want to help make the future a better and healthier place for yourself and others, participating in research is one of the most important things you can do. 

Learn more about the CeliAction Study and see if you qualify:
www.CeliActionStudy.com

 
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